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

Phase 2RecruitingNCT07023614

Imbria Pharmaceuticals, Inc.165 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

165

Conditions

Non-obstructive Hypertrophic Cardiomyopathy

Interventions

Ninerafaxstat 200mg MRDRUG

Ninerafaxstat 200mg Modified Release tablet administered BID

PlaceboDRUG

Matching placebo tablet administered BID

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (23)

Imbria Investigational Site

La Jolla, California, United States

Outcomes

Primary Outcomes

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

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.