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

Phase 2

Not yet recruiting

• Conditions: Non-obstructive Hypertrophic Cardiomyopathy
• Interventions: Drug: Ninerafaxstat 200mg MR; Drug: Placebo

• Registration Number: NCT07023614
• Lead Sponsor: Imbria Pharmaceuticals, Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-06
• Study First Submitted: 2025-06-09
• Study First Submitted QC: 2025-06-09
• Last Update Submitted: 2025-06-16
• Study First Posted: 2025-06-17
• Last Update Posted: 2025-06-19
• Study Start: 2025-07
• Primary Completion: 2026-12
• Study Completion: 2026-12

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 165

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Ninerafaxstat	Ninerafaxstat 200mg MR	-
Placebo	Placebo	-

Primary Outcome Measures

Name	Time	Method
Change in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS)	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 Outcome Measures

Name	Time	Method
Change in ventilatory efficiency (VE/VCO2 slope) during cardiopulmonary exercise testing (CPET)	Baseline to Week 12
Change in the Kansas City Cardiomyopathy Questionnaire Total Symptom Score (KCCQ-TSS)	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.
Change in the Kansas City Cardiomyopathy Questionnaire Physical Limitations Score (KCCQ-PLS)	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.
Change in the Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS)	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.