Trial Search Results

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

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 doses titrated to achieve 1 of 2 target drug concentrations.

Stanford is currently accepting patients for this trial.

Lead Sponsor:

MyoKardia, Inc.

Stanford Investigator(s):

Intervention(s):

  • Drug: mavacamten
  • Drug: Placebo

Phase:

Phase 2

Eligibility


Key Inclusion Criteria:

   - 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 Criteria:

   - 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.

Ages Eligible for Study

18 Years - N/A

Genders Eligible for Study

All

Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Recruiting