Actively Recruiting

Phase 2
Age: 18Years +
All Genders
ID07023614

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

Led by Imbria Pharmaceuticals, Inc. · Updated on 2026-05-18

165

Participants Needed

67

Research Sites

N/A

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating the effects of ninerafaxstat in adults with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM), a heart condition characterized by thickened heart muscle without obstruction. This global Phase 2b study aims to assess the safety and effectiveness of ninerafaxstat compared to a placebo, both given alongside standard care, to improve symptoms and heart function. The trial is double-blinded and placebo-controlled to ensure reliable comparisons between groups. Participants will be randomly assigned to receive either ninerafaxstat 200mg modified release tablets or matching placebo tablets, both taken twice daily. The study treatment will be given over a 12-week period. This design helps researchers observe how ninerafaxstat performs against placebo while maintaining standard therapies for nHCM. During the study, participants will undergo assessments including cardiopulmonary exercise testing and completion of the Kansas City Cardiomyopathy Questionnaire to track symptoms, physical limitations, and overall health status. The main focus is on changes in the questionnaire's clinical summary score from baseline to week 12. Safety and tolerability will also be monitored throughout the trial, which is expected to run until December 2026.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Adults aged 18 years or older
  • Clinical diagnosis of hypertrophic cardiomyopathy according to current guidelines
  • Confirmation of non-obstructive hypertrophic cardiomyopathy by echocardiography core laboratory
  • New York Heart Association (NYHA) functional Class II or III at screening
  • Functional limitation confirmed by cardiopulmonary exercise testing
Not Eligible

You will not qualify if you...

  • Known or suspected infiltrative, genetic, or storage disorder causing cardiac hypertrophy mimicking non-obstructive hypertrophic cardiomyopathy
  • Other conditions judged as the primary cause of shortness of breath, exercise intolerance, or chest pain
  • Inability to exercise on treadmill or bicycle due to orthopedic or other limitations
  • Any medical condition that prevents upright exercise stress testing

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - 12 weeks

Participants receive Ninerafaxstat or placebo tablets twice daily as part of the trial treatment.

Baseline visit and periodic visits during treatment

Trial Site Locations

Total: 67 locations

1

Imbria Investigational Site

La Jolla, California, United States, 92037

Actively Recruiting

2

Imbria Investigational Site

Los Angeles, California, United States, 90048

Actively Recruiting

3

Imbria Investigational Site

San Francisco, California, United States, 94143

Actively Recruiting

4

Imbria Investigational Site

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

Actively Recruiting

5

Imbria Investigational Site

Miami, Florida, United States, 33136

Actively Recruiting

6

Imbria Investigational Site

Chicago, Illinois, United States, 60611

Actively Recruiting

7

Imbria Investigational Site

Oak Lawn, Illinois, United States, 60453

Not Yet Recruiting

8

Imbria Investigational Site

Baltimore, Maryland, United States, 21218

Actively Recruiting

9

Imbria Investigational Site

Boston, Massachusetts, United States, 02114

Actively Recruiting

10

Imbria Investigational Site

Burlington, Massachusetts, United States, 01805

Actively Recruiting

11

Imbria Investigational Site

Grand Rapids, Michigan, United States, 49503

Actively Recruiting

12

Imbria Investigational Site

Rochester, Minnesota, United States, 55905

Actively Recruiting

13

Imbria Investigational Site

St Louis, Missouri, United States, 63110

Actively Recruiting

14

Imbria Investigational Site

New York, New York, United States, 10016

Actively Recruiting

15

Imbria Investigational Site

Charlotte, North Carolina, United States, 28204

Actively Recruiting

16

Imbria Investigational Site

Portland, Oregon, United States, 97239

Actively Recruiting

17

Imbria Investigational Site

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

18

Imbria Investigational Site

Philadelphia, Pennsylvania, United States, 19107

Actively Recruiting

19

Imbria Investigational Site

Houston, Texas, United States, 77030

Actively Recruiting

20

Imbria Investigational Site

Plano, Texas, United States, 75093

Actively Recruiting

21

Imbria Investigational Site

Charlottesville, Virginia, United States, 22903

Actively Recruiting

22

Imbria Investigational Site

Richmond, Virginia, United States, 23298

Actively Recruiting

23

Imbria Investigational Site

Graz, Austria, 8036

Actively Recruiting

24

Imbria Investigational Site

Edegem, Belgium, 2650

Actively Recruiting

25

Imbria Investigational Site

Montpellier, France, 34295

Actively Recruiting

26

Imbria Investigational Site

Paris, France, 75015

Actively Recruiting

27

Imbria Investigational Site

Saint-Herblain, France, 44800

Actively Recruiting

28

Imbria Investigational Site

Bad Oeynhausen, Germany, 32545

Actively Recruiting

29

Imbria Investigational Site

Göttingen, Germany, 37075

Actively Recruiting

30

Imbria Investigational Site

Heidelberg, Germany, 69120

Actively Recruiting

31

Imbria Investigational Site

Jena, Germany, 07747

Not Yet Recruiting

32

Imbria Investigational Site

Florence, Italy, 50139

Actively Recruiting

33

Imbria Investigational Site

Massa, Italy, 54100

Actively Recruiting

34

Imbria Investigational Site

Milan, Italy, 20131

Actively Recruiting

35

Imbria Investigational Site

Milan, Italy, 20146

Actively Recruiting

36

Imbria Investigational Site

Milan, Italy, 20162

Actively Recruiting

37

Imbria Investigational Site

Naples, Italy, 80131

Actively Recruiting

38

Imbria Investigational Site

Amsterdam-Zuidoost, Netherlands, 1105 AZ

Actively Recruiting

39

Imbria Investigational Site

Leiden, Netherlands, 2333ZC

Not Yet Recruiting

40

Imbria Investigational Site

Nijmegen, Netherlands, 6525 GA

Not Yet Recruiting

41

Imbria Investigational Site

Rotterdam, Netherlands, 3015 AA

Actively Recruiting

42

Imbria Investigational Site

Katowice, Poland, 40-555

Actively Recruiting

43

Imbria Investigational Site

Warsaw, Poland, 02-507

Actively Recruiting

44

Imbria Investigational Site

Wroclaw, Poland, 53-114

Actively Recruiting

45

Imbria Investigational Site

Guimarães, Portugal, 4835-044

Actively Recruiting

46

Imbria Investigational Site

Lisbon, Portugal, 1169-024

Actively Recruiting

47

Imbria Investigational Site

Lisbon, Portugal, 1500-650

Actively Recruiting

48

Imbria Investigational Site

Barcelona, Barcelona, Spain, 08035

Actively Recruiting

49

Imbria Investigational Site

A Coruña, Galicia, Spain, 15006

Actively Recruiting

50

Imbria Investigational Site

Madrid, Madrid, Spain, 28034

Actively Recruiting

51

Imbria Investigational Site

Vigo, Pontevedra, Spain, 36312

Actively Recruiting

52

Imbria Investigational Site

Seville, Sevilla, Spain, 41009

Actively Recruiting

53

Imbria Investigational Site

Seville, Sevilla, Spain, 41013

Actively Recruiting

54

Imbria Investigational Site

Barcelona, Spain, 8036

Actively Recruiting

55

Imbria Investigational Site

Barcelona, Spain, 8041

Actively Recruiting

56

Imbria Investigational Site

Barcelona, Spain, 8907

Actively Recruiting

57

Imbria Investigational Site

El Palmar, Spain, 30120

Actively Recruiting

58

Imbria Investigational Site

Madrid, Spain, 28222

Actively Recruiting

59

Imbria Investigational Site

Palma de Mallorca, Spain, 07198

Actively Recruiting

60

Imbria Investigational Site

Salamanca, Spain, 37007

Actively Recruiting

61

Imbria Investigational Site

Valencia, Spain, 03010

Actively Recruiting

62

Imbria Investigational Site

Valencia, Spain, 46026

Actively Recruiting

63

Imbria Investigational Site

London, England, United Kingdom, W12 0HS

Actively Recruiting

64

Imbria Investigational Site

Glasgow, Scotland, United Kingdom, G51 4TF

Actively Recruiting

65

Imbria Investigational Site

London, United Kingdom, EC1A 7BE

Actively Recruiting

66

Imbria Investigational Site

London, United Kingdom, SW3 6HP

Actively Recruiting

67

Imbria Investigational Site

Oxford, United Kingdom, OX3 9DU

Actively Recruiting

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Research Team

M

Medical Monitor

How is the study designed?

Study Type

INTERVENTIONAL

Masking

TRIPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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