Share on facebook Share on Twitter Share on Google Plus Share This
Back to Results

A Phase 2 Open-label Pilot Study Evaluating MYK-461 in Subjects With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction


Title A Phase 2 Open-label Pilot Study to Evaluate Efficacy, Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of MYK-461 in Subjects With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction
Therapeutic Area Cardiovascular Disease, Cardiomyopathy, Myocardial Heart Disease
Principal Investigator Martin Maron, MD
Min Age 18 Years
Max Age 70 Years
Gender Both
Contact Martin Maron
617 636-8066
More Information https://clinicaltrials.gov/ct2/show/NCT02842242

Overview

The purpose of this phase 2 open-label pilot study is to evaluate the efficacy, pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of MYK-461 in subjects with symptomatic HCM and LVOT obstruction aged 18-70 years.

Study Details

Inclusion Criteria

  • Patients age 18 - 70 years who carry a diagnosis of hypertrophic cardiomyopathy (HCM) with left ventricular outflow tract obstruction (LVOT)
  • Symptomatic NYHA functional Class II - IV
  • BMI 18-37 kg/m2

Exclusion Criteria

  • History of syncope (passing out) with exercise within previous 6 months
  • Current or history of irregular heart beats (atrial fibrillation, ventricular tachyarrhythmia)
  • History of obstructive coronary artery disease

Study Requirements

This study will last for 16 weeks. The first eight (8) weeks require a weekly visit to Tufts Medical Center. There is an additional visit to Tufts Medical Center at week 12 and a final visit to Tufts Medical Center at week 16. The following are procedures that will occur at some or all of the visits:

  • Review your medical history and perform a physical examination
  • Check your body height, weight, blood pressure, heart rate, and breathing rate
  • Collect blood samples for routine laboratory tests and pharmacokinetic sample (a test to quantify how much of the study drug is in your body). Depending on the specific visit, the amount of blood taken will range from one (1) teaspoon (5 mL) to up to four (4) teaspoons (20 mL).
  • Perform an Electrocardiogram (ECG) - a test that produces a tracing of your heartbeat
  • Perform a resting echocardiogram (ECHO) – a test that uses sound waves to produce images of your heart
  • Cardiopulmonary Exercise Test – a test that uses a treadmill to determine your exercise ability (Weeks 1 and 12 only)
  • Place a wearable ECG device on your chest (removed and replaced at every other visit, i.e. every 2 weeks)
  • Review your study medication and any side effects you have felt
  • Review any medical events (such as hospital admissions) that have happened to you since your last visit
  • Provide you with more study drug and instructions on how to take it
  • For females only – a urine pregnancy test

Optional Procedures:

  • HCM genotype blood test
    • provide one additional blood sample for each test on Day 1 before receiving study drug
  • Pharmacogenomic blood test
    • provide one additional blood sample for each test on Day 1 before receiving study drug
  • Pulse wave biosensor testing.
    • A bracelet device that measures heart rate, blood pressure, and other health information.
These three tests are optional. You do not have to decide to do these tests in order to participate in the main study.