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Efficacy and Safety of Tesevatinib in Subjects with Autosomal Dominant Polycystic Kidney Disease


Title A Double-blind Randomized Parallel Group Study of the Efficacy and Safety of Tesevatinib in Subjects with Autosomal Dominant Polycystic Kidney Disease
Therapeutic Area Polycystic Kidney Disease
Principal Investigator Ronald D. Perrone, MD
Min Age 18 Years
Max Age 60 Years
Gender All
Contact Margaret Healy 617-636-8117
Carly Tucker 617-636-7914
More Information https://clinicaltrials.gov/ct2/show/NCT03203642

Overview

The purpose of this study is to find out if the experimental study drug, tesevatinib, works as a treatment for ADPKD and if it is safe. Tesevatinib is made by Kadmon, who provides funding to this facility for the conduct of this study.

About 100 subjects will participate in this study across the U.S. Half the subjects (50 subjects) will receive tesevatinib, and the other half (50 subjects) will receive placebo. The study will last approximately 2 years, and will require frequent visits to Tufts Medical Center

Study Details

Inclusion Criteria

  • Confirmed diagnosis of ADPKD based on Ravine’s criteria. Subjects < 30 years of age must have at least 2 cysts (unilateral or bilateral) while subjects ≥ 30 years of age must have at least 2 cysts in each kidney (ie, total ≥ 4 cysts).
  • eGFR ≥ 30 mL/min/1.73 m2 and ≤ 80 mL/min/1.73 m2, using the Modification of Diet in Renal Disease-4 variable formula.
  • Not pregnant or planning to become pregnant.

Exclusion Criteria

  • Previous partial or total nephrectomy or a kidney transplant
  • Uncontrolled hypertension
  • Moderate hematuria

Study Requirements

This is a multicenter, double-blind, randomized, parallel group study of tesevatinib tabletformulation administered to male and female subjects with ADPKD, 18 to 60 years of age and with an estimated glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73 m2 and ≤ 80 mL/min/1.73 m2.

Subjects will be screened up to 28 days before initiation of treatment with tesevatinib or placebo (certain screening assessments may be conducted up to 42 days prior to dosing). Screening assessments will include medical and PKD history, physical examination, vital signs, electrocardiograms (ECGs), clinical laboratory evaluation (including hematology, serum chemistry, coagulation, thyroid stimulating hormone, urinalysis, and pregnancy), ocular examination, echocardiogram, and MRI. Calculation of htTKV at screening for eligibilitydetermination may be performed by a local radiologist.

Starting on Day 1, subjects will receive 50 mg QD of tesevatinib or placebo for up to 24 months. During the Early Treatment Period (Days 1 to 28), subjects will report to the study site on Days 1, 14, and 28; subjects will be followed every 28 days from Months 2 to 12 andevery other month from Months 12 to 24. Safety assessments will be performed at regular intervals throughout the study and will include physical examinations, vital signs, ECGs, clinical laboratory evaluations, ocular examination and echocardiograms. Magnetic resonance imaging to determine the change from baseline in htTKV will be performed at Months 12, 18, and 24. Entry into the study will be based on local MRI readings, but central MRI readings and htTKV calculation will be performed at baseline and during the study.

All subjects will undergo an end-of-study evaluation approximately 30 days after the last dose of study drug.