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NCT Number: NCT07023614

A Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients With Symptomatic Non-obstructive Hypertrophic Cardiomyopathy

FORTITUDE-HCM is a global, multicenter, double-blind, parallel-group, placebo-controlled Phase 2b study that will assess the efficacy and safety of ninerafaxstat compared to placebo on top of Standard of Care in patients with symptomatic nHCM

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Key information

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Imbria Investigational Site, Graz, Austria

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Ninerafaxstat 200mg MR

Drug

Ninerafaxstat 200mg Modified Release tablet administered BID

Placebo

Drug

Matching placebo tablet administered BID

Primary outcomes

  1. Change in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS)

    Time frame: Baseline to Week 12

    The Clinical Summary Score is a merging of symptom frequency and symptom burden domains into a total symptom score combined with the physical limitation domain. Scoring ranges from 0 to 100, with higher scores indicating better health status.

Secondary outcomes

  1. Change in ventilatory efficiency (VE/VCO2 slope) during cardiopulmonary exercise testing (CPET)

    Time frame: Baseline to Week 12

  2. Change in the Kansas City Cardiomyopathy Questionnaire Total Symptom Score (KCCQ-TSS)

    Time frame: Baseline to Week 12

    The Total Symptom Score is a merging of symptom frequency and symptom burden domains. Scoring ranges from 0 to 100, with higher scores indicating better health status.

  3. Change in the Kansas City Cardiomyopathy Questionnaire Physical Limitations Score (KCCQ-PLS)

    Time frame: Baseline to Week 12

    The Physical Limitations Score is a single-domain score. Scoring ranges from 0 to 100, with higher scores indicating better health status.

  4. Change in the Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS)

    Time frame: Baseline to Week 12

    The Overall Summary Score is a merging of symptom, physical limitations, social limitations, and quality of life domains. Scoring ranges from 0 to 100, with higher scores indicating better health status.

Study contacts

Contact information is provided by the study sponsor or research team.

Medical Monitor

CONTACT

[email protected]

(617) 675-4060

Sponsors and collaborators

Lead sponsor

Imbria Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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

Acronym: FORTITUDE-HCM

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Jun 17, 2025
Registry last updated
May 18, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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