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Allergies


Study Title: A Phase III, Randomized, Placebo-Controlled Clinical Trial to Study the Efficacy and Safety of MK-3641, a Ragweed (Ambrosia artemisiifolia) Sublingual Immunotherapy Tablet, in Children With a History of Ragweed-Induced Rhinoconjunctivitis With or Without Asthma

The purpose of this study is to assess the efficacy and safety of MK-3641 (short ragweed [Ambrosia artemisiifolia] extract, MK-3641, SCH 039641, RAGWITEK™) sublingual immunotherapy tablets in children aged 5 to 17 years with ragweed-induced rhinoconjunctivitis with or without asthma. The primary hypothesis of this study is that administration of MK-3641 sublingual immunotherapy tablets to children 5 to 17 years of age, compared with placebo, will result in a significant reduction in rhinoconjunctivitis symtoms and medication use over the peak ragweed season (RS).

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Amyloidosis


AL Amyloidosis - Assessing Treatments for Patients with Relapsed or Refractory Systemic Light Chain AL Amyloidosis

This is a phase 3, randomized, controlled, open-label, multicenter study of the oral formulation of dexamethasone plus MLN9708 (investigational study drug) compared with treatment chosen by the investigator from a prespecified list of regimens available in clinical practice. Treatment options will include: dexamethasone alone, dexamethasone plus an alkylating agent (melphalan or cyclophosphamide), or dexamethasone plus an immunomodulatory drug ([IMiD], thalidomide or lenalidomide) in patients with relapsed or refractory AL amyloidosis. The sponsor is Millennium Pharmaceuticals Inc. For any questions, please call the number listed above. Participants may also view clinicaltrials.gov for any additional information.

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Aneurysms


Pivotal Study of the MicroVention Flow Re-Direction (FRED) Endoluminal Device Stent System in the Treatment of Intracranial Aneurysms

The purpose of the study is to evaluate the safety and effectiveness of the FRED device, which is intended for use in treating wide-neck brain aneurysms. The FRED device is a small metallic mesh tube (or “flow-diverting stent”) that will be placed across the neck of the aneurysm and is intended to redirect blood flow away from the aneurysm. By blocking blood flow into the aneurysm, it is hoped the aneurysm will thrombose (clot) to decrease the chance for rupture.

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Bone Marrow Transplant


A Single-arm, Prospective Study of Remestemcel-L, Ex-vivo Cultured Adult Human Mesenchymal Stromal Cells, for the Treatment of Pediatric Patients who Have Failed to Respond to Steroid Treatment for Acute GVHD

The main purpose of the study is to find out about whether the study drug, Remestemcel-L, is safe in humans and an effective product in pediatric patients with acute Graft versus Host Disease (GVHD). GVHD may occur after a bone marrow or stem cell transplant in which someone receives bone marrow tissue or cells from a donor (called an allogeneic transplant). The new, transplanted cells attack the recipient's body (the host) because they regard it as foreign.

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Brain Tumors


A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma

This study is being done to test the effect of a drug, nifurtimox, against neuroblastoma and medulloblastoma in children. Nifurtimox is a drug that has been used in South America for many years to treat a parasitic disease known as Chagas Disease. It is not approved by the Food and Drug Administration for routine use in neuroblastoma or medulloblastoma in the United States, but the FDA is allowing it to be used in research studies like this one. Limited early observations, suggest that nifurtimox may have anti-tumor activity for neuroblastoma and medulloblastoma. We do not know whether nifurtimox will shrink/kill tumor cells effectively in children. Therefore, the major goal of the study is to learn if nifurtimox in combination with other common chemotherapy drugs is effective in shrinking/killing neuroblastoma and medulloblastoma cells. We will also be collecting information about any side effects that the drug may have.

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A Randomized, Phase III Trial Of Memantine and Whole-Brain Radiotherapy With or Without Hippocampal Avoidance in Patients With Brain Metastases

The purpose of this study is to compare any good and bad effects of avoiding the hippocampus during whole-brain radiation plus memantine to using the usual whole-brain radiation plus memantine. The hippocampus is a brain structure that is important for memory. The addition of the hippocampal avoidance technique to the usual whole-brain radiation plus memantine will decrease the dose of radiation to your hippocampus. It is hoped hippocampal avoidance technique will decrease the chance of cognitive side effects, however it is possible hippocampal avoidance could have no impact on cognitive side effects and could even cause side effects. This study will allow the researchers to know whether this different approach is better, the same, or worse than the usual approach. Memantine is already FDA-approved for use in patients with dementia and is commonly used off-label (that is, for a purpose for which it is not FDA approved) for patients receiving whole-brain radiation therapy for cancer that has spread to the brain.

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A071101: A Phase II Randomized Trial Comparing the Efficacy of Heat Shock Protein-Peptide Complex-96 (HSPPC-96) (NSC #725085, Alliance IND# 15380) Vaccine Given with Bevacizumab Versus Bevacizumab Alone in the Treatment of Surgically Resectable Recurrent Glioblastoma Multiforme (GBM)

The purpose of this study is to compare the effects of a vaccine called heat shock protein peptide complex 96 (HSPPC-96) given with a drug called bevacizumab to bevacizumab alone. HSPPC-96 is experimental and is made from tissue taken from the brain tumor. Specifically, HSPPC-96 is a protein (a naturally occurring substance) that is taken from a person's brain tumor tissue and given back to them in the form of a preparation called a vaccine that may work to help their body have a response against remaining brain tumor cells. Bevacizumab blocks a protein called VEGF (Vascular Endothelial Growth Factor), which is produced by normal cells but over produced by cancer cells. Bevacizumab has been approved by the Food and Drug administration for treating brain tumors that grow back. In this study, subjects will either get HSPPC-96 vaccine at the same time as bevacizumab, HSPPC vaccine first and then bevacizumab if their tumor comes back, or bevacizumab alone. The use of HSPPC-96 in combination with bevacizumab is also investigational

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A221101: A Phase III Randomized, Double-Blind Placebo Controlled Study of Armodafinil (Nuvigil®) To Reduce Cancer-Related Fatigue in Patients with High Grade Glioma

The purpose of this study is to:  1) see if taking the study agent, armodafinil, at a dose of 150mg or 250mg, will improve problems with fatigue in subjects who have been diagnosed with cancer and are experiencing fatigue; and 2) see the effects (good and bad) of taking Armodafinil compared to placebo (an inactive agent) on cancer related fatigue. In this study, subjects will take either the study agent, armodafinil, or the placebo (inactive agent).  Subjects will not take both. Armodafinil (Nuvigil®) is a medicine that is currently FDA approved to promote wakefulness in people who have sleep disorders.   However, it is not been studied in people with cancer related fatigue.

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Phase I/II Dose Escalation Trial to Assess Safety of Intrathecal Trastuzumab for the Treatment of Leptomeningeal Metastases in HER2 Positive Cancer

The purpose of this the study that you are now being asked to participate in is to determine how safe and effective the study drug, trastuzumab, is in patients with HER-2 positive cancer that has spread to the fluid around the brain and spinal cord.

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Phase II Trial Of SMO/AKT/NF2 Inhibitors In Progressive Meningiomas With SMO/AKT/ NF2 Mutations

The purpose of this study is to test good and bad effects of two different drugs against meningioma tumors with altered genes. Today, therapy for meningioma is the same for all patients, and is not based on tumor genetic testing. This trial is trying to see if tumor genetic testing would be helpful at guiding treatment in meningioma patients. Researchers have looked at the DNA material (genes) that can be affected in meningioma and have found several genes that are altered, or mutated. These include the genes called SMO and NF2. When the SMO or NF2 genes are altered, it can cause a tumor to grow. There are drugs that target these 2 genes.

The study drug, vismodegib, blocks the SMO receptor. Vismodegib has already been FDA-approved to treat basal cell cancer, which is a type of skin cancer. Vismodegib could shrink cancer, but it could also cause side effects. The study drug, GSK2256098, blocks FAK, and seems to work better in tumors that have NF2-mutations. GSK2256098 has been tested in other cancers. Researchers hope to learn if either of the study drugs will shrink cancer.

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Randomized Phase II Trial of Hypofractionated Dose-Escalated Photon IMRT or Proton Beam Therapy Versus Conventional Photon Irradiation With Concomitant and Adjuvant Temozolomide in Patients With Newly Diagnosed Glioblastoma

The purpose of this study is to compare a different radiation schedule and higher radiation dose [higher dose group] to the standard dose of radiation [standard dose group]. Both groups will receive usual chemotherapy, temozolomide. The higher radiation dose could shrink brain cancer, but it could also cause side effects. This study will allow the researchers to know whether this higher dose is better, the same, or worse than the usual approach.

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Breast Cancer


Breast Cancer - Determining Effect of MGAH22 in Breast Cancer Patients

To determine if MGAH22 has sufficient activity in the population of breast cancer participants whose tumors exhibit 2+ HER2 oncoprotein expression and lacks HER2 gene amplification.

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A Cohort Study to Evaluate Genetic Predictors of Aromatase Inhibitor Musculoskeletal Symptoms (AIMSS)

This research is being done to find out what effects, good and/or bad, that anastrozole has on women and whether their genes can help explain how anastrozole affects them. Specifically, many women who take anastrozole, or one of the other aromatase inhibitors (letrozole or exemestane) during treatment for breast cancer report muscle and joint aches; however, the reasons that lead to these symptoms are not known. These symptoms have been called “aromatase inhibitor-associated musculoskeletal syndrome” (or AIMSS). Small studies have suggested that a person’s genetic information may help us to develop a way to predict who will develop side effects and how best to treat them, and we hope to collect information in this study to look at this more closely. In addition, we hope to look at how the side effects from hormone therapy influence quality of life and a patient’s willingness to continue hormonal treatment.

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A Phase 1, Open Label, Dose Escalation Study of MGA271 (Fcoptimized Humanized Anti-B7-H3 Monoclonal Antibody) in Patients with Refractory B7-H3-Expressing Neoplasms or Neoplasms Whose Vasculature Expresses B7-H3

The purpose of this study is to evaluate the safety of MGA271 when given by intravenous (IV) infusion to patients with refractory cancer. The study will also evaluate how long MGA271 stays in the blood and how long it takes for it to leave the body, what is the highest dose that can safely be given, and whether it may have an effect on tumors.

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A Phase II Randomized Controlled Trial of Genomically Directed Therapy After Preoperative Chemotherapy in Patients with Triple Negative Breast Cancer

The purpose of this study is to test the theory that therapy designed for each individual’s tumor will improve outcomes over standard of care in a population that needs a better standard. Using tumor tissue samples from a prior surgery, treatment reccomendations will be made based on DNA sequencing of the tumor cells by a Cancer Genomics Tumor Board facilitated by the Hoosier Cancer Research Network. Subjects will be randomized to one of several treatment options based on their specific tumor genetic make-up, prior treatment history and tolerance, and medical history.

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A phase III, randomized, open label, multicenter, controlled trial of niraparib versus physician’s choice in previously-treated, HER2 negative, germline BRCA mutation-positive breast cancer patients.

The purpose of this study is to compare the effects, both good and/or bad, of giving participants either niraparib or the standard chemotherapy for HER2-negative , BRCA mutation positive breast cancer to find out which is better

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A Randomized Phase III Trial of Adjuvant Therapy Comparing Doxorubicin Plus Cyclophosphamide Followed by Weekly Paclitaxel with or without Carboplatin for Node-Positive or High-Risk Node-Negative Triple-Negative Invasive Breast Cancer

This study is looking to determine whether the addition of carboplatin to an adjuvant chemotherapy regimen of doxorubicin/cyclophosphamide followed by paclitaxel will improve invasive disease-free survival compared to treatment without carboplatin.

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Bronchopulmonary Dysplasia


Efficacy of Recombinant Human Clara Cell 10 Protein (rhCC10) Administered to Premature Neonates With Respiratory Distress Syndrome

Recombinant human CC10 protein (rhCC10) is a novel therapeutic agent used to prevent the development of chronic respiratory morbidity (CRM; repeated respiratory infections, asthma, re-hospitalizations) in preterm infants. Native CC10 is a natural anti-inflammatory and immunomodulatory factor produced by Clara Cells in the lung and is the most abundant protein in respiratory mucosa. Animal data demonstrate that a single intratracheal dose of rhCC10 administered shortly after birth reduces lung inflammation (important biomarkers linked to lung injury in preterm infants), promotes normal lung development, preserves lung architecture, improves pulmonary function, suppresses the response to endotoxin and enhances resistance to pulmonary infections. In preterm infants who die or develop lung inflammation and subsequent bronchopulmonary dysplasia (BPD), both the concentration and activity of CC10 are significantly reduced indicating that CC10 is essential for preventing lung injury and promoting normal lung development. In a small phase I study, rhCC10 significantly decreased several indices of pulmonary inflammation in the lungs of premature infants who were at risk of developing BPD and associated CRM. The drug appeared to be safe, well-tolerated, and reduce risk of re-hospitalization due to respiratory illness for 9-10 months after a single intratracheal dose at the time of birth (0/11 rhCC10-treated infants vs. 3/6 placebo-treated). This supports the protective role of rhCC10 against damage from hyperoxia, mechanical ventilation, inflammation, and infection in the immature lung. A more normal airway epithelium will produce significantly more endogenous CC10, with both factors contributing to enhanced resistance to infections, less asthma, and improved long-term respiratory outcome. We propose to conduct a Phase 2 clinical trial to evaluate rhCC10 in extremely premature infants (<29 weeks gestation) for the prevention of BPD and CRM. This will be a randomized, double-blind, placebo-controlled dose escalation study in 88 premature infants. A single intratracheal dose of study drug (rhCC10 or placebo) will be administered to preterm infants receiving surfactant and mechanical ventilation for treatment of RDS. Infants will be followed to evaluate safety, pharmacokinetics, and short and long term efficacy of this approach. Safety will be evaluated through serious adverse event (SAE) and adverse event monitoring and by Bayley neurodevelopmental assessments at 18 months corrected gestational age (CGA). Efficacy measurements will include the primary combined endpoint of alive without evidence of CRM at 12 months CGA (defined by parental diaries and pulmonary questionnaires) comparing rhCC10 treated to placebo controls. The availability of a therapy which prevents lung injury, promotes lung development, and prevents serious respiratory infections and asthma in high risk preterm infants would be a highly significant advancement in care.

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Diabetes Type 2


Assessment of Coagulation Factors in Nonalcoholic Fatty Liver Disease (NAFLD) and Related Conditions

This non-interventional study seeks to determine the validity of the hypothesis that individuals with Nonalcoholic Fatty Liver Disease and/or its related conditions such as Type 2 Diabetes and obesity have elevated levels of two related coagulation proteins on their circulating blood cells, which are believed to play an important role in the development and progression of liver disease.

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Diffuse Large B-Cell Lymphoma


Lenalidomide in Patients with Newly Diagnosed Diffuse Large B Cell Lymphoma

The purpose of this study is to find out what effects, good and/or bad, the addition of lenalidomide to standard chemotherapy (RCHOP) has on you and your cancer. Everybody in this study will receive standard chemotherapy. In addition to standard chemotherapy randomly chosen half of the subjects will receive a medication called lenalidomide. Adding lenalidomide to the standard chemotherapy RCHOP is considered experimental in diffuse large B cell lymphoma, and is not FDA approved. There is NO placebo (“sugar pill”) in this study, and everybody selected will receive an active medication.

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Dry Eye


Dry Eye Assessment and Management Trial

Dry Eye Disease, phase III double masked, 1 year primary study and 2 year extension study for patients to be treated with Omega 3 fatty acids or placebo.

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Eosinophilic Esophagitis


A Randomized, Double-Blind, Parallel, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Dupilumab in Adult Patients with Active Eosinophilic Esophagitis

The primary objective of the study is to assess the clinical efficacy of repeat subcutaneous (SC) doses of dupilumab, compared with placebo, to relieve symptoms in adult patients with active, moderate to severe eosinophilic esophagitis (EoE).

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Ependymoma


ACNS0831: Phase III Randomized Trial of Post-Radiation Chemotherapy in Patients with Newly Diagnosed Ependymoma Ages 1 to 21 years

This study is being done to evaluate what effects, if any, chemotherapy can have when added to the standard of care for this type of cancer. The current standard of care for this type of cancer is a surgery, to remove as much of the tumor as possible, followed by radiation. It will look at if chemotherapy, when added to the standard of care, affects the cancer from coming back.

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Fibromyalgia


A Randomized, Double-Blind, Placebo-And Active-Controlled Study Of DS-5565 In Subjects With Pain Associated With Fibromyalgia

This is a phase 3 trial evaluating microgabalin for the treatment of pain associated with fibromyalgia. The primary objective of the study is to compare change in weekly ADPS (average daily pain score) from baseline to Week 13 in patients receiving a total daily dose of either 15 mg or 30 mg of microgabalin versus placebo. Patients that complete the initial study are eligible to enroll in an extension study which aims to assess the long-term safety of a total daily dose of mirogabalin 15 mg or mirogabalin 30 mg in patients with pain associated with fibromyalgia. Eligible subjects will be randomized in the ratio of 1:1:1:1 to receive 13 weeks of treatment with DS-5565 15 mg once daily (QD), DS-5565 15 mg twice daily (BID), placebo, or pregabalin 150 mg BID.

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Glioblastoma


Randomized Phase II Trial of Hypofractionated Dose-Escalated Photon IMRT or Proton Beam Therapy Versus Conventional Photon Irradiation With Concomitant and Adjuvant Temozolomide in Patients With Newly Diagnosed Glioblastoma

The purpose of this study is to compare a different radiation schedule and higher radiation dose [higher dose group] to the standard dose of radiation [standard dose group]. Both groups will receive usual chemotherapy, temozolomide. The higher radiation dose could shrink brain cancer, but it could also cause side effects. This study will allow the researchers to know whether this higher dose is better, the same, or worse than the usual approach.

More

Glioma


A221101: A Phase III Randomized, Double-Blind Placebo Controlled Study of Armodafinil (Nuvigil®) To Reduce Cancer-Related Fatigue in Patients with High Grade Glioma

The purpose of this study is to:  1) see if taking the study agent, armodafinil, at a dose of 150mg or 250mg, will improve problems with fatigue in subjects who have been diagnosed with cancer and are experiencing fatigue; and 2) see the effects (good and bad) of taking Armodafinil compared to placebo (an inactive agent) on cancer related fatigue. In this study, subjects will take either the study agent, armodafinil, or the placebo (inactive agent).  Subjects will not take both. Armodafinil (Nuvigil®) is a medicine that is currently FDA approved to promote wakefulness in people who have sleep disorders.   However, it is not been studied in people with cancer related fatigue.

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GVHD


A Single-arm, Prospective Study of Remestemcel-L, Ex-vivo Cultured Adult Human Mesenchymal Stromal Cells, for the Treatment of Pediatric Patients who Have Failed to Respond to Steroid Treatment for Acute GVHD

The main purpose of the study is to find out about whether the study drug, Remestemcel-L, is safe in humans and an effective product in pediatric patients with acute Graft versus Host Disease (GVHD). GVHD may occur after a bone marrow or stem cell transplant in which someone receives bone marrow tissue or cells from a donor (called an allogeneic transplant). The new, transplanted cells attack the recipient's body (the host) because they regard it as foreign.

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Healthy Volunteers


Assessment of Coagulation Factors in Nonalcoholic Fatty Liver Disease (NAFLD) and Related Conditions

This non-interventional study seeks to determine the validity of the hypothesis that individuals with Nonalcoholic Fatty Liver Disease and/or its related conditions such as Type 2 Diabetes and obesity have elevated levels of two related coagulation proteins on their circulating blood cells, which are believed to play an important role in the development and progression of liver disease.

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Hepatitis B


A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multi center Study to Evaluate the Safety and Efficacy of GS-9620 in combination with Tenofovir Disoproxil Fumarate (TDF) for the Treatment of Subjects with Chronic Hepatitis B and who are currently not on Treatment

This is a randomized, double-blind, placebo-controlled, multicenter Phase 2 study to evaluate the safety and efficacy of administering either 1 mg, 2 mg or 4 mg of GS-9620 orally once a week (every 7 days) both in-clinic and at home for 12 doses (Weeks 0-11) in adult CHB subjects who are not currently on treatment for CHB.

Approximately 175 subjects will be randomized in a 1:2:2:2 ratio to one of the four treatment arms (A:B: C: D) for weekly dosing for a total of 12 doses. All subjects will also be treated with Tenofovir

Disoproxil Fumarate (TDF) 300 mg oral daily for 48 weeks. Randomization will be stratified by HBeAg status (positive vs. negative) and ALT (> vs. <19 IU/L for female; > vs. < 30 IU/L for male).

  • Treatment Arm A: 25 subjects administered TDF 300 mg oral daily + placebo orally once a week (every 7 days) for 12 doses
  • Treatment Arm B: 50 subjects administered TDF 300 mg oral daily + GS-9620 1 mg orally once a week every 7 days) for 12 doses
  • Treatment Arm C: 50 subjects administered TDF 300 mg oral daily +GS- 9620 2 mg orally once a week every 7 days) for 12 doses
  • Treatment Arm D: 50 subjects administered TDF 300 mg oral daily + GS-9620 4 mg orally once a week (every 7 days) for 12 doses

After Week 11, GS-9620/placebo will be discontinued. All subjects will continue receiving TDF and will be followed for 37 weeks (Week 48).

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High Blood Pressure


Hypertension - SPRINT

About two-thirds of those over age 60 have high blood pressure. Lowering blood pressure further than current practice might help prevent heart disease, stroke, and kidney disease. In addition, high blood pressure can cause changes in memory and thinking as people get older. The purpose of this study is to determine whether lowering blood pressure will reduce the rate of heart disease and stroke, memory decline or worsening of kidney disease in adults over the age of 55 who already have high blood pressure. Specifically, the SPRINT trial will test whether keeping systolic blood pressure (top number in your blood pressure reading) less than 120 mmHg is better than the current recommendation of keeping it less than 140 mmHg. This study does not involve experimental drugs. The trial is approximately 5 years long and participants receive free blood pressure medications for the duration of the study.

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Hodgkin Lymphoma


A Phase I-II Trial of Brentuximab Vedotin Plus Rituximab as Frontline Therapy for Patients with CD30+ and/or EBV+ Lymphomas

The primary purpose of this study is to evaluate the safety of the combination of two different drugs (brentuximab vedotin and rituximab) in patients with certain types of lymphoma. This study is for patients who have a type of lymphoma that expresses a tumor marker called CD30 and/or a type that is associated with the Epstein-Barr virus (EBV-related lymphoma) and who have not yet received any treatment for their cancer, except for dose-reduction or discontinuation (stoppage) of medications used to prevent rejection of transplanted organs (for those patients who have undergone transplantation). These include, but are not limited to, diffuse large B-cell lymphoma (DLBCL), post-transplant lymphoproliferative disorder (PTLD), non-Hodgkin’s lymphoma (NHL), Hodgkin’s lymphoma (HL), and peripheral T-cell lymphoma (PTCL).  In addition, this study will attempt to evaluate how effective this combination of study drugs may be in addressing these types of lymphoma.

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Sequential SGN-35 Therapy (S-AVD) for Untreated Hodgkin Lymphoma

This study involves administration of an investigational drug called SGN35 (brentuximab vedotin) in combination with standard chemotherapy (adriamycin, vinblastine, and dacarbazine, or AVD) for participants 60 years old or older with untreated Hodgkin Lympoma     

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Hypertension


Hypertension - SPRINT

About two-thirds of those over age 60 have high blood pressure. Lowering blood pressure further than current practice might help prevent heart disease, stroke, and kidney disease. In addition, high blood pressure can cause changes in memory and thinking as people get older. The purpose of this study is to determine whether lowering blood pressure will reduce the rate of heart disease and stroke, memory decline or worsening of kidney disease in adults over the age of 55 who already have high blood pressure. Specifically, the SPRINT trial will test whether keeping systolic blood pressure (top number in your blood pressure reading) less than 120 mmHg is better than the current recommendation of keeping it less than 140 mmHg. This study does not involve experimental drugs. The trial is approximately 5 years long and participants receive free blood pressure medications for the duration of the study.

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Leukemia


A Randomized Trial of Levofloxacin to Prevent Bacteremia in Children Being Treated for Acute Leukemia (AL) or Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

This study looks at how well an antibiotic prophylaxis, called levofloxacin, can prevent bacterial infection from developing in children and young adults receiving intensive cancer treatment or a stem cell transplant. The levofloxacin drug which prevents bacterial infection is experimental.

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AALL0932:  Treatment of Patients with Newly Diagnosed Standard Risk B-Precursor Acute Lymphoblastic Leukemia (ALL)

This is a protocol for people with newly diagnosed Standard Risk Acute Lymphoblastic Leukemia. The study is broken up into parts. Each part of the study will be exploring different phases of chemotherapy such as Induction, Post-Induction, and Maintenance. In each phase of the study, researchers will be testing different experimental drug regimens versus standard treatments to see which one works better. Additionally, participants will be grouped based on their risk level and may receive different  chemotherapy regimens based on their risk.

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AALL1131: A Phase III Randomized Trial for Newly Diagnosed High Risk B-Lymphoblastic Leukemia (ALL) Testing Clofarabine (IND# 73789, NSC# 606869) in the Very High Risk Stratum

This study will look at how effective different combinations of chemotherapy are a preventing acute lymphoblastic leukemia from coming back.

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AALL1231: A Phase III Randomized Trial Investigating Bortezomib (NSC# 681239; IND# 58443) on a Modified Augmented BFM (ABFM) Backbone in Newly Diagnosed T- Lymphoblastic Leukemia (T-ALL) and T- Lymphoblastic Lymphoma (T-LLy)

The aims of this study are to improve treatment for T-Lymphoblastic Leukemia (T-ALL) and T-Lymphoblastic Lymphoma (T-LLy). We want to see if adding bortezomib to the standard treatment reduces the chance that the cancer will come back.

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Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome

  1. To find out if subjects with down syndrome and standard risk acute myeloid leukemia can be treated with less treatment and still have successful outcomes.
  2. To find out if subjects with down syndrome and high risk acute myeloid leukemia can be successfully treated with stronger chemotherapy.

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Risk-Stratified Randomized Phase III Testing of Blinatumomab (IND#117467, NSC#765986) in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL)

This randomized phase III trial compares how well blinatumomab works compared with standard combination chemotherapy in treating patients with B-cell acute lymphoblastic leukemia that has returned after a period of improvement (relapsed). Monoclonal antibodies, such as blinatumomab, can block cancer growth by finding cancer cells and helping to kill them or carrying cancer-killing substances to them. It is not yet known whether standard combination chemotherapy is more effective than blinatumomab in treating relapsed B-cell acute lymphoblastic leukemia.

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Liver Diseases


Assessment of Coagulation Factors in Nonalcoholic Fatty Liver Disease (NAFLD) and Related Conditions

This non-interventional study seeks to determine the validity of the hypothesis that individuals with Nonalcoholic Fatty Liver Disease and/or its related conditions such as Type 2 Diabetes and obesity have elevated levels of two related coagulation proteins on their circulating blood cells, which are believed to play an important role in the development and progression of liver disease.

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Lung Cancer


RTOG 1306: A Randomized Phase II Study of Individualized Combined Modality Therapy for Stage III Non-small Cell Lung Cancer (NSCLC)

Cancer is a disease caused by alterations in the functions of genes within the cells. In approximately 15% of patients with non-small cell lung cancer, one of two genes is altered significantly (known as EGFR mutation or ALK rearrangement). Erlotinib, a pill, taken on a daily basis produces significant decrease in the size of tumors in patients with advanced stage lung cancer with EGFR mutation. Similarly, another pill, crizotinib, produces significant decrease in the size of tumors in patients with advanced stage lung cancer with ALK rearrangement. However it is not clear how useful these two drugs will be in patients with a less advanced form of lung cancer in the presence of these gene alterations.  The purpose of this study is to find out what effects, good and/or bad, one of the following treatments has on locally advanced, non-operable lung cancer with specific gene alterations (EGFR mutation or ALK rearrangement).

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Lyme Disease


Xenodiagnosis After Antibiotic Treatment for Lyme Disease – Phase 2 study

A substantial proportion of patients with Lyme disease continue to have symptoms after antibiotic treatment but the cause of these symptoms is controversial.  Recent animal studies have suggested that the Lyme disease bacteria, or their components, may persist even after antibiotic treatment. 

 

The goal of this study is to determine whether the symptoms of Lyme disease after treatment are associated with xenodiagnostic detection of the Lyme disease bacteria. Xenodiagnosis is a method used to detect an infectious organism by using that organism’s natural vector.  Because the bacteria are highly adapted to find their natural host, studies have shown that xenodiagnosis can be a very sensitive method for detecting continued presence of infections, including Lyme disease. 

 

This study will involve placing laboratory reared ticks on patients who have early or late stage Lyme disease. All ticks will then be removed from the patient and tested for evidence of the Lyme bacteria.  Participants will complete a series of questionnaires to assess their level of function and symptomology at different time points after treatment.  The study will look for correlations between levels of function/improvement in symptoms and the continued presence of infection as determined by xenodiagnosis. 

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Lymphomas


A Phase I-II Trial of Brentuximab Vedotin Plus Rituximab as Frontline Therapy for Patients with CD30+ and/or EBV+ Lymphomas

The primary purpose of this study is to evaluate the safety of the combination of two different drugs (brentuximab vedotin and rituximab) in patients with certain types of lymphoma. This study is for patients who have a type of lymphoma that expresses a tumor marker called CD30 and/or a type that is associated with the Epstein-Barr virus (EBV-related lymphoma) and who have not yet received any treatment for their cancer, except for dose-reduction or discontinuation (stoppage) of medications used to prevent rejection of transplanted organs (for those patients who have undergone transplantation). These include, but are not limited to, diffuse large B-cell lymphoma (DLBCL), post-transplant lymphoproliferative disorder (PTLD), non-Hodgkin’s lymphoma (NHL), Hodgkin’s lymphoma (HL), and peripheral T-cell lymphoma (PTCL).  In addition, this study will attempt to evaluate how effective this combination of study drugs may be in addressing these types of lymphoma.

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AALL1231: A Phase III Randomized Trial Investigating Bortezomib (NSC# 681239; IND# 58443) on a Modified Augmented BFM (ABFM) Backbone in Newly Diagnosed T- Lymphoblastic Leukemia (T-ALL) and T- Lymphoblastic Lymphoma (T-LLy)

The aims of this study are to improve treatment for T-Lymphoblastic Leukemia (T-ALL) and T-Lymphoblastic Lymphoma (T-LLy). We want to see if adding bortezomib to the standard treatment reduces the chance that the cancer will come back.

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Intergroup Randomized Phase II Four Arm Study In Patients ≥ 60 With Previously Untreated Mantle Cell Lymphoma Of Therapy With: Arm A = Rituximab+ Bendamustine Followed By Rituximab Consolidation (RB → R); Arm B = Rituximab + Bendamustine + Bortezomib Followed By Rituximab Consolidation (RBV→ R), Arm C = Rituximab + Bendamustine Followed By Lenalidomide + Rituximab Consolidation (RB → LR) or Arm D = Rituximab + Bendamustine + Bortezomib Followed By Lenalidomide + Rituximab Consolidation (RBV → LR)  

In step 1, to find out what effects, good and bad, the addition of bortezomib to rituximab plus bendamustine has on  lymphoma compared to rituximab plus bendamustine alone.

 

In step 2, to find out what effects, good and bad, continuing chemotherapy after step 1 with lenalidomide plus rituximab has on  lymphoma compared to continuing chemotherapy with rituximab alone.

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Lenalidomide in Patients with Newly Diagnosed Diffuse Large B Cell Lymphoma

The purpose of this study is to find out what effects, good and/or bad, the addition of lenalidomide to standard chemotherapy (RCHOP) has on you and your cancer. Everybody in this study will receive standard chemotherapy. In addition to standard chemotherapy randomly chosen half of the subjects will receive a medication called lenalidomide. Adding lenalidomide to the standard chemotherapy RCHOP is considered experimental in diffuse large B cell lymphoma, and is not FDA approved. There is NO placebo (“sugar pill”) in this study, and everybody selected will receive an active medication.

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Sequential SGN-35 Therapy (S-AVD) for Untreated Hodgkin Lymphoma

This study involves administration of an investigational drug called SGN35 (brentuximab vedotin) in combination with standard chemotherapy (adriamycin, vinblastine, and dacarbazine, or AVD) for participants 60 years old or older with untreated Hodgkin Lympoma     

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Mantle Cell Lymphoma


Intergroup Randomized Phase II Four Arm Study In Patients ≥ 60 With Previously Untreated Mantle Cell Lymphoma Of Therapy With: Arm A = Rituximab+ Bendamustine Followed By Rituximab Consolidation (RB → R); Arm B = Rituximab + Bendamustine + Bortezomib Followed By Rituximab Consolidation (RBV→ R), Arm C = Rituximab + Bendamustine Followed By Lenalidomide + Rituximab Consolidation (RB → LR) or Arm D = Rituximab + Bendamustine + Bortezomib Followed By Lenalidomide + Rituximab Consolidation (RBV → LR)  

In step 1, to find out what effects, good and bad, the addition of bortezomib to rituximab plus bendamustine has on  lymphoma compared to rituximab plus bendamustine alone.

 

In step 2, to find out what effects, good and bad, continuing chemotherapy after step 1 with lenalidomide plus rituximab has on  lymphoma compared to continuing chemotherapy with rituximab alone.

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Medulloblastoma


A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma

This study is being done to test the effect of a drug, nifurtimox, against neuroblastoma and medulloblastoma in children. Nifurtimox is a drug that has been used in South America for many years to treat a parasitic disease known as Chagas Disease. It is not approved by the Food and Drug Administration for routine use in neuroblastoma or medulloblastoma in the United States, but the FDA is allowing it to be used in research studies like this one. Limited early observations, suggest that nifurtimox may have anti-tumor activity for neuroblastoma and medulloblastoma. We do not know whether nifurtimox will shrink/kill tumor cells effectively in children. Therefore, the major goal of the study is to learn if nifurtimox in combination with other common chemotherapy drugs is effective in shrinking/killing neuroblastoma and medulloblastoma cells. We will also be collecting information about any side effects that the drug may have.

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Multiple Myeloma


A Phase 3, Randomized, Placebo-Controlled, Double-Blind Study of Oral Ixazomib Maintenance Therapy After Initial Therapy in Patients With Newly Diagnosed Multiple Myeloma Not Treated With Stem Cell Transplantation

This study will assess whether taking ixazomib as maintenance therapy after standard treatments extends the period of time that a patient’s cancer stays inactive.

Maintenance therapy means that a drug is taken for a relatively long period (in this study, up to 24 months) to prolong the time that the myeloma remains under control after a prior therapy; in this study the prior therapy must not include autologous stem cell transplant.  It is not yet proven whether maintenance therapy given to people with multiple meyloma is better than waiting until the myeloma comes back to receive additional treatment.  The study will assess whether taking ixazomib immediately after responding to a prior therapy allows people with multiple myeloma to live longer by preventing or delaying the return of their disease.

This study is also being performed for these additional research purposes:

  • To evaluate the safety of ixazomib and to learn about the side effects associated with the use of this drug when it is used as a maintenance therapy.
  • To find out whether the presence of certain genes and proteins in the cancer cells of patients with multiple myeloma can predict whether ixazomib will work on cancers similar to yours.
  • To evaluate the health and overall well-being of patients while they are taking ixazomib.

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    Randomized Phase III Trial of Lenalidomide Versus Observation Alone in Patients with Asymptomatic High-Risk Smoldering Multiple Myeloma

    This study is being done to evaluate what effects, if any, the study drug Lenalidomide has on patients with Smoldering Multiple Myeloma as compared to observation alone, the current standard of care. It will look at how the study drug compares to observation alone in keeping the cancer from coming back.

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    Multiple Sclerosis


    A Multicenter, Global, Observational Study to Collect Information on Safety and to Document the Drug Utilization of Tecfidera™ (Dimethyl Fumarate) When Used in Routine Medical Practice in the Treatment of Multiple Sclerosis (ESTEEM)

    The purpose of this study is to better characterize the long-term benefit-risk profile of Tecfidera™ (Dimethyl Fumarate (DMF)) in patients with multiple sclerosis (MS) who are prescribed Tecfidera under routine clinical care.

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    Neuroblastoma


    A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma

    This study is being done to test the effect of a drug, nifurtimox, against neuroblastoma and medulloblastoma in children. Nifurtimox is a drug that has been used in South America for many years to treat a parasitic disease known as Chagas Disease. It is not approved by the Food and Drug Administration for routine use in neuroblastoma or medulloblastoma in the United States, but the FDA is allowing it to be used in research studies like this one. Limited early observations, suggest that nifurtimox may have anti-tumor activity for neuroblastoma and medulloblastoma. We do not know whether nifurtimox will shrink/kill tumor cells effectively in children. Therefore, the major goal of the study is to learn if nifurtimox in combination with other common chemotherapy drugs is effective in shrinking/killing neuroblastoma and medulloblastoma cells. We will also be collecting information about any side effects that the drug may have.

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    ANBL1232: Utilizing Response and Biology-Based Risk Factors to Guide Therapy in Patients with Non-High-Risk Neuroblastoma: A Groupwide Historically Controlled Phase III Study

    In this study we are using new biologic features, in addition to the standard risk factors, to help decide which subjects need treatment and what the best treatment is. People with non-high-risk NBL generally respond well to treatment and the majority of patients can be treated successfully with standard therapy. But standard therapy can include risks from surgery and/or side effects from chemotherapy.Based on the evaluation of non-high-risk NBL patients treated in the past, we think that we can change the amount of therapy for subjects and still treat the cancer successfully. Small studies have shown that some subjects can be watched without having surgery or getting chemotherapy and still have an excellent outcome. By not having surgery or lowering the amount of chemotherapy we hope to prevent complications and harmful side effects. For other subjects we think a change in therapy will treat the cancer more successfully.

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    NMTT: Neuroblastoma Maintenance Therapy Trial Using Difluoromethylornithine (DFMO)

    The purpose of this research study is to evaluate an investigational drug (DFMO) for Neuroblastoma that is in remission. Remission means that there are no current signs of active cancer. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the ability of this study drug to keep neuroblastoma in remission and will also look at the safety and tolerability of DFMO.

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    Non-Hodgkin Lymphoma


    A Phase I-II Trial of Brentuximab Vedotin Plus Rituximab as Frontline Therapy for Patients with CD30+ and/or EBV+ Lymphomas

    The primary purpose of this study is to evaluate the safety of the combination of two different drugs (brentuximab vedotin and rituximab) in patients with certain types of lymphoma. This study is for patients who have a type of lymphoma that expresses a tumor marker called CD30 and/or a type that is associated with the Epstein-Barr virus (EBV-related lymphoma) and who have not yet received any treatment for their cancer, except for dose-reduction or discontinuation (stoppage) of medications used to prevent rejection of transplanted organs (for those patients who have undergone transplantation). These include, but are not limited to, diffuse large B-cell lymphoma (DLBCL), post-transplant lymphoproliferative disorder (PTLD), non-Hodgkin’s lymphoma (NHL), Hodgkin’s lymphoma (HL), and peripheral T-cell lymphoma (PTCL).  In addition, this study will attempt to evaluate how effective this combination of study drugs may be in addressing these types of lymphoma.

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    Ophthalmology


    Dry Eye Assessment and Management Trial

    Dry Eye Disease, phase III double masked, 1 year primary study and 2 year extension study for patients to be treated with Omega 3 fatty acids or placebo.

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    Osteoarthritis


    A multi-center, parallel, double-blind, randomized, placebo-controlled study to evaluate the effectiveness of HYMOVIS, a new viscoelastic hydrogel, for the treatment of osteoarthritis of the knee

    This is a multi-center, parallel, double-blind, randomized, placebo-controlled study to evaluate the safety and effectiveness of HYMOVIS, a new viscoelastic hydrogel, for the treatment of osteoarthritis of the knee.  A large number of experimental and clinical studies in OA have demonstrated that intra-articular (IA) administration of hyaluranan (HA) may improve the altered rheological properties of synovial fluid, improve signs and symptoms of patients, and in some studies exert protective and reparative effects on the joint structure.

    Subjects will undergo intra-articular injections of the study treatment (HYMOVIS or placebo), and subjects will receive a second IA injection 7 days following the first injection and will be followed for up to 180 days after the first treatment. Other study procedures include X-ray, physician assessment, urine pregnancy test, urine drug test, saliva alcohol test and subject questionnaires.

    The primary outcome is to evaluate the effectiveness of two 3 mL intra-articular (IA) injections of HYMOVIS in comparison with 3 mL IA injections of Placebo in providing superior pain relief for the treatment of subjects with symptomatic primary osteoarthritis of the knee measured by the Western Ontario and McMaster Universities Arthritis (WOMAC) Pain Subscale.

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    A Phase 2a Study Evaluating the Safety, Efficacy,and Pharmacodynamic Effects of ABT-981 in Patients with Knee Osteoarthritis

    This is a Phase 2a, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the safety, tolerability and efficacy of ABT-981, a novel biologic drug that targets IL-1α and IL-1β, in male and female subjects ages 35 through 74, inclusive, with symptomatic and radiographic knee osteoarthritis. The main objective of the study is to assess the effect of ABT-981 on OA knee pain using the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) at Week 16 and synovitis/effusion volume of the index knee using quantitative measures and semi-quantitative MRI scoring at Week 26.

    Eligible subjects will be randomized in the ratio of 1:1:1:1 to receive study drug every 2 weeks for  50 weeks of treatment with  25 mg ABT-981 SC, 100 mg ABT-981 SC, 200 mg ABT-981 SC or Matching placebo SC

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    Ovarian Cancer


    A Randomized, Double-blind, Placebo-Controlled, Phase 2 Study to Assess the Efficacy and Safety of Farletuzumab (MORAb-003) in Combination with Carboplatin plus Paclitaxel or Carboplatin plus Pegylated Liposomal Doxorubicin (PLD) in Subjects with Low CA125 Platinum-Sensitive Ovarian Cancer

    This research is being done to find out if carboplatin plus paclitaxel or carboplatin plus Pegylated Liposomal Doxorubicin (PLD), chemotherapies (anticancer drugs) that are used to treat ovarian cancer, work better alone or when given with an investigational drug called farletuzumab.

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    Can diet and physical activity modulate ovarian, fallopian tube and primary peritoneal cancer progression-free survival?

    The purpose of this study is to find out if a change in diet and exercise in women with ovarian, fallopian tube, or primary peritoneal cancer has an effect on the length of time someone is cancer free following initial treatment. Some studies suggest diet and exercise may improve survival for cancer patients, but no studies have been done to show if changes in diet and exercise can have an effect on cancer returning in women treated for ovarian, fallopian tube, or primary peritoneal cancer. Other goals include finding out if the changes in diet and exercise will improve your overall quality of life and your ability to be physically active. In addition, you may be asked if researchers can test some of your blood to measure carotenoid levels, which will tell them about the kind of foods you are eating, and examine your genes (DNA).

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    Polycystic Kidney Disease


    A Phase 3b, Multi-center, Open-label Trial to Evaluate the Long Term Safety of Immediate-release Tolvaptan (OPC 41061, 30 mg to 120 mg/day, Split dose) in Subjects with Autosomal Dominant Polycystic Kidney Disease

    Living with the pain, high blood pressure, and other symptoms of autosomal dominant polycystic kidney disease (ADPKD) can be overwhelming. And because ADPKD may be passed from one generation to the next, you may have seen how it has affected your family and wonder how the disease will affect you.

    If you have ADPKD, and have participated in a previous Tolvaptan research study, you may want to learn more about the Open Label clinical research study.

    What is the Open Label study?

    The purpose of the Open Label study is to evaluate the long-term safety and effectiveness of an investigational medication called tolvaptan in patients with ADPKD.

    Who can participate?

    You may be able to join the study if you:

    • Have participated in a previous Tolvaptan research study
    • Are diagnosed with ADPKD

    What is the investigational medication?

    Tolvaptan is being investigated in this study as a potential treatment to slow the progression of cyst growth in the kidneys of adults with ADPKD.

    What are the potential risks and benefits?

    Everyone who participates in a research study must first complete a screening visit. During the screening visit, the study doctor will explain to you the study details and the potential risks and benefits of participation in the study.

    The information learned from the study and your participation may help us learn about how to develop future treatment options for people with ADPKD.

    Study participation is voluntary. If you choose to participate, you may leave the study at any time for any reason. The study doctor and staff will be available to help you throughout the study and to address any questions you may have. Your health is very important to them.

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    A Phase 3b, Multi-center, Randomized-withdrawal, Placebo-controlled, Double-blind, Parallel-group Trial to Compare the Efficacy and Safety of Tolvaptan (45 to 120 mg/day, Split-dose) in Subjects with Chronic Kidney Disease Between Late Stage 2 to Early Stage 4 Due to Autosomal Dominant Polycystic Kidney Disease

    Living with the pain, high blood pressure, and other symptoms of autosomal dominant polycystic kidney disease (ADPKD) can be overwhelming. And because ADPKD may be passed from one generation to the next, you may have seen how it has affected your family and wonder how the disease will affect you.

    If you have ADPKD, you may want to learn more about the REPRISE clinical research study.

    What is the REPRISE study?

    The purpose of the REPRISE study is to evaluate the safety and effectiveness of an investigational medication called tolvaptan in patients with late stage 2 to early stage 4 chronic kidney disease caused by ADPKD.

    Who can participate?

    You may be able to join the study if you:

    • Are between the ages of 18 to 55 years old; patients aged 56 to 65 may be able to participate in some circumstances
    • Are diagnosed with ADPKD

    What is the investigational medication?

    Tolvaptan is being investigated in this study as a potential treatment to slow the progression of cyst growth in the kidneys of adults with ADPKD.

    What are the potential risks and benefits?

    Everyone who participates in a research study must first complete a screening visit. During the screening visit, the study doctor will explain to you the study details and the potential risks and benefits of participation in the study.

    Study participation is voluntary. If you choose to participate, you may leave the study at any time for any reason. The study doctor and staff will be available to help you throughout the study and to address any questions you may have. Your health is very important to them.

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    Polycystic Kidney Disease - PKD Registry

    This project consists of the development of a Polycystic Kidney Disease Research Registry database for the purpose of conducting retrospective research on Polycystic Kidney Disease using information from patient encounters in the Nephrology clinic at Tufts Medical Center, obtaining permission from patients in order to contact them in the future to ascertain any interest in participating in research studies that they may be eligible for, and permitting review of information contained within the registry to identify patients who may be eligible for future research studies.

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    Preventive Medicine


    CS SCARLETT: Covert Strokes – Semi-structured interviews of Clinicians Assessing Risk, Logical inference, and Equipoise of Treatments and Testing decisions

    This study is a qualitative exploration of the beliefs, attitudes, and behaviors of clinicians who provide care for patients with covert brain infarcts (silent strokes). We hope to better understand how clinicians are currently approaching incidentally discovered strokes without apparent symptoms.

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    CS SPECTRE: Covert Strokes – Semi-structured interviews of Patient Experiences, Concerns, information Transfer, and Risk Estimation

    This study is a qualitative exploration of the experiences and concerns of patients following the detection of incidentally discovered strokes without apparent symptoms (covert strokes, or silent strokes). We hope to better understand the needs of patients in order to orient future studies in screening, treatment, and prevention to meet those needs.

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    Prostate Cancer


    A031201: Phase III Trial of Enzalutamide (NSC # 766085) versus Enzalutamide, Abiraterone and Prednisone for Castration Resistant Metastatic Prostate Cance

    This study is being done to evaluate what effects, if any, the study drug Enzalutamide has on prostate cancer when given alone or in combination with hormonal agents Abiraterone and Prednisone. It will look at how the study drug alone compares to the study drug plus these hormonal agents in keeping the cancer from progressing.

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    Respiratory Distress Syndrome in Premature Infant


    Efficacy of Recombinant Human Clara Cell 10 Protein (rhCC10) Administered to Premature Neonates With Respiratory Distress Syndrome

    Recombinant human CC10 protein (rhCC10) is a novel therapeutic agent used to prevent the development of chronic respiratory morbidity (CRM; repeated respiratory infections, asthma, re-hospitalizations) in preterm infants. Native CC10 is a natural anti-inflammatory and immunomodulatory factor produced by Clara Cells in the lung and is the most abundant protein in respiratory mucosa. Animal data demonstrate that a single intratracheal dose of rhCC10 administered shortly after birth reduces lung inflammation (important biomarkers linked to lung injury in preterm infants), promotes normal lung development, preserves lung architecture, improves pulmonary function, suppresses the response to endotoxin and enhances resistance to pulmonary infections. In preterm infants who die or develop lung inflammation and subsequent bronchopulmonary dysplasia (BPD), both the concentration and activity of CC10 are significantly reduced indicating that CC10 is essential for preventing lung injury and promoting normal lung development. In a small phase I study, rhCC10 significantly decreased several indices of pulmonary inflammation in the lungs of premature infants who were at risk of developing BPD and associated CRM. The drug appeared to be safe, well-tolerated, and reduce risk of re-hospitalization due to respiratory illness for 9-10 months after a single intratracheal dose at the time of birth (0/11 rhCC10-treated infants vs. 3/6 placebo-treated). This supports the protective role of rhCC10 against damage from hyperoxia, mechanical ventilation, inflammation, and infection in the immature lung. A more normal airway epithelium will produce significantly more endogenous CC10, with both factors contributing to enhanced resistance to infections, less asthma, and improved long-term respiratory outcome. We propose to conduct a Phase 2 clinical trial to evaluate rhCC10 in extremely premature infants (<29 weeks gestation) for the prevention of BPD and CRM. This will be a randomized, double-blind, placebo-controlled dose escalation study in 88 premature infants. A single intratracheal dose of study drug (rhCC10 or placebo) will be administered to preterm infants receiving surfactant and mechanical ventilation for treatment of RDS. Infants will be followed to evaluate safety, pharmacokinetics, and short and long term efficacy of this approach. Safety will be evaluated through serious adverse event (SAE) and adverse event monitoring and by Bayley neurodevelopmental assessments at 18 months corrected gestational age (CGA). Efficacy measurements will include the primary combined endpoint of alive without evidence of CRM at 12 months CGA (defined by parental diaries and pulmonary questionnaires) comparing rhCC10 treated to placebo controls. The availability of a therapy which prevents lung injury, promotes lung development, and prevents serious respiratory infections and asthma in high risk preterm infants would be a highly significant advancement in care.

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    Stroke


    A Randomized, Double-Blind, Multinational Study to Prevent Major Vascular Events with Ticagrelor Compared to Aspirin (ASA) in Patients with Acute Ischemic Stroke or TIA

    This study is to compare the effect and safety of the investigational drug, ticagrelor, given twice daily compared to aspirin, given once daily for the prevention of death, heart attack, and stroke in patients with acute ischemic stroke or TIA. Ticagrelor is not FDA approved for these indications.

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    CS SCARLETT: Covert Strokes – Semi-structured interviews of Clinicians Assessing Risk, Logical inference, and Equipoise of Treatments and Testing decisions

    This study is a qualitative exploration of the beliefs, attitudes, and behaviors of clinicians who provide care for patients with covert brain infarcts (silent strokes). We hope to better understand how clinicians are currently approaching incidentally discovered strokes without apparent symptoms.

    More

    CS SPECTRE: Covert Strokes – Semi-structured interviews of Patient Experiences, Concerns, information Transfer, and Risk Estimation

    This study is a qualitative exploration of the experiences and concerns of patients following the detection of incidentally discovered strokes without apparent symptoms (covert strokes, or silent strokes). We hope to better understand the needs of patients in order to orient future studies in screening, treatment, and prevention to meet those needs.

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    Multicenter, randomized, double-blind, double-dummy, active-comparator, event-driven, superiority phase III study of secondary prevention of stroke and prevention of systemic embolism in patients with a recent Embolic Stroke of Undetermined Source (ESUS) comparing rivaroxaban 15 mg once daily with aspirin 100 mg

    The purpose of this study is to find out whether a new anti-clotting medication called rivaroxaban leads to fewer blood clots in your brain (stroke) or in other blood vessels in your body when compared with aspirin.

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    Secondary Stroke Prevention in Patients with Patent Foramen Ovale: The International PFO-Consortium

    The purpose of this study is to learn more about how often people with PFOs have a second stroke after having the first one. This registry will investigate the impact of age, blood thinning drugs (such as aspirin and Coumadin), and closure of the PFO on the risk of stroke.

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    Stroke Hyperglycemia Insulin Network Effort Trial

    The purpose of this study is to find out the effects of controlling blood sugar within a narrow range by giving IV insulin in patients with high blood sugar within 12 hours of developing symptoms of a stroke. The study is to determine if giving IV insulin to people with high blood sugar and who have had a stroke can improve recovery as compared to those who receive insulin by an injection under the skin. Studies have shown that high blood sugar levels during a stroke could be associated with more damage to the brain than normal blood sugar levels. Blood sugar levels can be lowered with insulin. The five different insulins that will be used in this study are approved by the FDA and are approved for this use as well. A subject will be assigned to one of 2 study groups: IV insulin and subcutaneous insulin. The subject will have a 50% chance to be assigned in one of the two groups by an internet-based randomization system.

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    Thrombocytopenia


    A Phase 3, Randomized, Global, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Once-daily Oral Avatrombopag for the Treatment of Adults with Thrombocytopenia Associated with Liver Disease Prior to an Elective Procedure

    The main purpose of this research study is to investigate the effectiveness of avatombopag (the study drug under investigation) in increasing the amount of platelets (a type of cell found in the blood) in patients with chronic liver disease who need to have an elective procedure but have thrombocytopenia (low platelet counts) related to chronic liver disease. This study will look if avatrombopag has the ability to increase your platelet count high enough for you to have an elective procedure and lower the need for procedure-related platelet transfusions.

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    Transient Ischemic Attack


    A Randomized, Double-Blind, Multinational Study to Prevent Major Vascular Events with Ticagrelor Compared to Aspirin (ASA) in Patients with Acute Ischemic Stroke or TIA

    This study is to compare the effect and safety of the investigational drug, ticagrelor, given twice daily compared to aspirin, given once daily for the prevention of death, heart attack, and stroke in patients with acute ischemic stroke or TIA. Ticagrelor is not FDA approved for these indications.

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