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

NCT07023614 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: IMB101-010
• Study Type: interventional
• Target: 165
• Locations: 11 countries
• Sites: 56

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

Conditions

Non-obstructive Hypertrophic Cardiomyopathy

Keywords

nHCM; non-obstructive HCM; non-obstructive hypertrophic cardiomyopathy; HCM; hypertrophic cardiomyopathy

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline to Week 12

Secondary Outcomes (4)

• 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

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

  Baseline to Week 12

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

  Baseline to Week 12

Study Arms (2)

Ninerafaxstat

EXPERIMENTAL

Drug: Ninerafaxstat 200mg MR

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Ninerafaxstat 200mg MR DRUG

Ninerafaxstat 200mg Modified Release tablet administered BID

Ninerafaxstat

Placebo DRUG

Matching placebo tablet administered BID

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Imbria Pharmaceuticals, Inc.: lead

Study Sites (56)

Imbria Investigational Site

La Jolla, California, 92037, United States

RECRUITING

Imbria Investigational Site

Los Angeles, California, 90048, United States

RECRUITING

Imbria Investigational Site

San Francisco, California, 94143, United States

RECRUITING

Imbria Investigational Site

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Imbria Investigational Site

Miami, Florida, 33136, United States

RECRUITING

Imbria Investigational Site

Chicago, Illinois, 60611, United States

RECRUITING

Imbria Investigational Site

Boston, Massachusetts, 02114, United States

RECRUITING

Imbria Investigational Site

Burlington, Massachusetts, 01805, United States

RECRUITING

Imbria Investigational Site

Grand Rapids, Michigan, 49503, United States

RECRUITING

Imbria Investigational Site

Rochester, Minnesota, 55905, United States

RECRUITING

Imbria Investigational Site

St Louis, Missouri, 63110, United States

RECRUITING

Imbria Investigational Site

New York, New York, 10016, United States

RECRUITING

Imbria Investigational Site

Charlotte, North Carolina, 28204, United States

RECRUITING

Imbria Investigational Site

Portland, Oregon, 97239, United States

RECRUITING

Imbria Investigational Site

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Imbria Investigational Site

Houston, Texas, 77030, United States

RECRUITING

Imbria Investigational Site

Plano, Texas, 75093, United States

RECRUITING

Imbria Investigational Site

Charlottesville, Virginia, 22903, United States

RECRUITING

Imbria Investigational Site

Graz, 8036, Austria

RECRUITING

Imbria Investigational Site

Edegem, 2650, Belgium

RECRUITING

Imbria Investigational Site

Montpellier, 34090, France

RECRUITING

Imbria Investigational Site

Paris, 75015, France

RECRUITING

Imbria Investigational Site

Saint-Herblain, 44800, France

RECRUITING

Imbria Investigational Site

Bad Oeynhausen, 32545, Germany

RECRUITING

Imbria Investigational Site

Göttingen, 37075, Germany

RECRUITING

Imbria Investigational Site

Heidelberg, 69120, Germany

RECRUITING

Imbria Investigational Site

Florence, 50134, Italy

RECRUITING

Imbria Investigational Site

Milan, 20131, Italy

RECRUITING

Imbria Investigational Site

Naples, 80131, Italy

RECRUITING

Imbria Investigational Site

Amsterdam-Zuidoost, 1105 AZ, Netherlands

RECRUITING

Imbria Investigational Site

Rotterdam, 3015 GD, Netherlands

RECRUITING

Imbria Investigational Site

Katowice, 40-555, Poland

RECRUITING

Imbria Investigational Site

Warsaw, 02-507, Poland

RECRUITING

Imbria Investigational Site

Wroclaw, 50-981, Poland

RECRUITING

Imbria Investigational Site

Guimarães, 4835-044, Portugal

RECRUITING

Imbria Investigational Site

Lisbon, 1169-024, Portugal

RECRUITING

Imbria Investigational Site

Lisbon, 1500-650, Portugal

RECRUITING

Imbria Investigational Site

Barcelona, Barcelona, 08035, Spain

RECRUITING

Imbria Investigational Site

A Coruña, Galicia, 15006, Spain

RECRUITING

Imbria Investigational Site

Madrid, Madrid, 28034, Spain

RECRUITING

Imbria Investigational Site

Vigo, Pontevedra, 36312, Spain

RECRUITING

Imbria Investigational Site

Seville, Sevilla, 41009, Spain

RECRUITING

Imbria Investigational Site

Seville, Sevilla, 41013, Spain

RECRUITING

Imbria Investigational Site

Barcelona, 8036, Spain

RECRUITING

Imbria Investigational Site

Barcelona, 8041, Spain

RECRUITING

Imbria Investigational Site

Barcelona, 8907, Spain

RECRUITING

Imbria Investigational Site

El Palmar, 30120, Spain

RECRUITING

Imbria Investigational Site

Madrid, 28034, Spain

RECRUITING

Imbria Investigational Site

Palma de Mallorca, 7198, Spain

RECRUITING

Imbria Investigational Site

Salamanca, 37007, Spain

RECRUITING

Imbria Investigational Site

Valencia, 46009, Spain

RECRUITING

Imbria Investigational Site

Valencia, 46009, Spain

RECRUITING

Imbria Investigational Site

London, England, W12 0HS, United Kingdom

RECRUITING

Imbria Investigational Site

Glasgow, Scotland, G51 4TF, United Kingdom

RECRUITING

Imbria Investigational Site

London, 401109, United Kingdom

RECRUITING

Imbria Investigational Site

London, EC1A 7BE, United Kingdom

RECRUITING

MeSH Terms

Conditions

Cardiomyopathy, Hypertrophic

Condition Hierarchy (Ancestors)

Cardiomyopathies; Heart Diseases; Cardiovascular Diseases; Aortic Stenosis, Subvalvular; Aortic Valve Stenosis; Aortic Valve Disease; Heart Valve Diseases

Central Study Contacts

Medical Monitor

CONTACT

(617) 675-4060; info@imbria.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 9, 2025
• First Posted: June 17, 2025
• Study Start: October 6, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: April 27, 2026

Record last verified: 2026-04

Locations

DE (3); NL (2); IT (3); AU (1); BE (1); PT (3); FR (3); PL (3); ES (15); GB (4); US (18)