Status:

COMPLETED

A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)

Lead Sponsor:

MyoKardia, Inc.

Conditions:

Non-obstructive Hypertrophic Cardiomyopathy

Eligibility:

All Genders

18+ years

Phase:

PHASE2

Brief Summary

This is a multicenter, exploratory, randomized, double-blind study of the administration of mavacamten in 60 participants with symptomatic nHCM randomized to receive a 16-week course of mavacamten dos...

Eligibility Criteria

Inclusion

  • Key
  • Diagnosed with nHCM (hypertrophied and non-dilated left ventricle in absence of systemic or other known cause), with LV wall thickness ≥ 15mm at Screening or ≥ 13mm with a positive family history of HCM.
  • Age 18 and greater, Body weight \> 45kg
  • Documented LVEF ≥ 55% at the Screening as determined by echo central lab
  • LVOT gradient \< 30 mmHg at rest AND during Valsalva AND post-exercise
  • NYHA functional class II or III
  • Elevated NT-proBNP at rest
  • Key

Exclusion

  • History of syncope, sustained ventricular tachyarrhythmia with exercise, obstructive coronary artery disease or myocardial infarction within the past 6 months
  • History of resuscitated sudden cardiac arrest at any time or known appropriate implantable cardioverter defibrillator (ICD) discharge within 6 months prior to Screening
  • Current treatment with disopyramide or ranolazine (within 14 days prior to Screening)
  • Current or planned treatment during the study with a combination of beta-blockers and calcium channel blockers
  • Has been treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation \[ASA\]) within 6 months prior to Screening
  • History of resting or post-exercise LVOT \>30 mmHg unless subsequently treated by septal reduction
  • Has QTc Fridericia (QTcF) \>480 ms or any other ECG abnormality considered by the investigator to pose a risk to participant safety (eg, second-degree atrioventricular block type II)
  • Has persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate-controlled within 1 year of Screening
  • History of clinically significant malignant disease within 10 years such as non-metastatic cutaneous squamous cell or basal cell carcinoma
  • History or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator or MyoKardia physician, would pose a risk to subject safety or interfere with the study evaluation, procedures, or completion.

Key Trial Info

Start Date :

March 30 2018

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

January 7 2020

Estimated Enrollment :

59 Patients enrolled

Trial Details

Trial ID

NCT03442764

Start Date

March 30 2018

End Date

January 7 2020

Last Update

August 9 2022

Active Locations (32)

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Page 1 of 8 (32 locations)

1

Mayo Clinic Arizona

Scottsdale, Arizona, United States, 85259

2

Cedars-Sinai Medical Center (Smidt Heart Institute)

Los Angeles, California, United States, 90048

3

Stanford Hospital and Clinics/Stanford University

Palo Alto, California, United States, 94305

4

University of California, San Francisco

San Francisco, California, United States, 94143