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Ninerafaxstat for symptomatic non-obstructive hypertrophic cardiomyopathy

A Global Phase 2b, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy - FORTITUDE-HCM

PHASE2 · Imbria Pharmaceuticals, Inc. · NCT07023614

This trial will test whether taking ninerafaxstat with usual care helps adults with symptomatic non-obstructive hypertrophic cardiomyopathy feel better and improve exercise capacity compared with a placebo.

Quick facts

PhasePHASE2
Study typeInterventional
Enrollment165 (estimated)
Ages18 Years and up
SexAll
SponsorImbria Pharmaceuticals, Inc. (industry)
Locations56 sites (La Jolla, California and 55 other locations)
Trial IDNCT07023614 on ClinicalTrials.gov

What this trial studies

FORTITUDE-HCM is a randomized, double-blind, placebo-controlled Phase 2b trial comparing ninerafaxstat 200 mg MR to placebo on top of standard of care in patients with symptomatic non-obstructive HCM. Participants will be confirmed by core lab echocardiography and must have NYHA class II or III symptoms and a functional limitation on cardiopulmonary exercise testing. The trial measures clinical benefit and safety outcomes to determine whether ninerafaxstat improves symptoms and exercise performance versus placebo. Patients with infiltrative or other non-HCM causes of hypertrophy or who cannot perform upright exercise testing are excluded.

Who should consider this trial

Good fit: Ideal candidates are adults with confirmed non-obstructive HCM who have NYHA class II–III symptoms, exercise limitation on CPET, and can perform treadmill or bicycle stress testing.

Not a fit: People with obstructive HCM, infiltrative or storage disorders that mimic HCM, or those unable to perform upright exercise testing are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, ninerafaxstat could improve symptoms and exercise tolerance for people with symptomatic non-obstructive HCM.

How similar studies have performed: Ninerafaxstat is a relatively novel agent with limited published clinical data, and while some early-phase HCM programs have shown promising signals, definitive benefits for similar approaches have not yet been established.

Eligibility criteria

Show full inclusion / exclusion criteria 
Select Inclusion Criteria:

* Has a clinical diagnosis of HCM consistent with current American College of Cardiology/American Heart Association and European Society of Cardiology Guideline definitions
* Has had confirmation of nHCM by the echocardiography core laboratory based on screening rest and exercise stress echocardiography
* New York Heart Association (NYHA) functional Class II or III at screening
* Functional limitation as defined by a screening CPET

Select Exclusion Criteria:

* Has a known or suspected infiltrative, genetic, or storage disorder causing cardiac hypertrophy that mimics nHCM
* Has any other condition judged by the investigator to be the primary cause of dyspnea, exercise intolerance, and/or angina
* Has an inability to exercise on a treadmill or bicycle (eg, orthopedic limitations)
* Has any medical condition that precludes upright exercise stress testing

Other protocol-defined inclusion and exclusion criteria apply.

Where this trial is running

La Jolla, California and 55 other locations

+6 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Non-obstructive Hypertrophic Cardiomyopathy, nHCM, non-obstructive HCM, non-obstructive hypertrophic cardiomyopathy, HCM, hypertrophic cardiomyopathy

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.