Non-obstructive Hypertrophic CardiomyopathyPhase 2FORTITUDE-HCMNCT07023614
A Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients With Symptomatic Non-obstructive Hypertrophic Cardiomyopathy
A Phase 2 study testing Ninerafaxstat 200mg MR in people with Non-obstructive Hypertrophic Cardiomyopathy, run by Imbria Pharmaceuticals, Inc. at 76 sites in 11 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Non-obstructive Hypertrophic Cardiomyopathy
- What is being tested?
- Ninerafaxstat 200mg MR
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 165 participants
- Where
- 76 sitesin 11 countries
- Study dates
- 6 Oct 2025to Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Trial locations and contacts
76 sites in 11 countries. Contact the study team or a site near you to ask about taking part.
76 sites in 11 countries
United States27 sites, 27 recruiting
- Not yet recruiting
Imbria Investigational Site
Atlanta, Georgia, 30322
Study Coordinator
- Recruiting
Imbria Investigational Site
Baltimore, Maryland, 21218
Study Coordinator
44327891702
- Recruiting
Imbria Investigational Site
Boston, Massachusetts, 02114
Study Coordinator, 203-450-8273
- Recruiting
Imbria Investigational Site
Burlington, Massachusetts, 01805
Study Coordinator, 781-825-8658
- Recruiting
Imbria Investigational Site
Charlotte, North Carolina, 28204
Study Coordinator, 7043554796
- Recruiting
Imbria Investigational Site
Charlottesville, Virginia, 22903
Study Coordinator, 434-982-1058
- Recruiting
Imbria Investigational Site
Chicago, Illinois, 60611
Study Coordinator, 312-695-4067
Study Coordinator, 312-926-8629
- Recruiting
Imbria Investigational Site
Grand Rapids, Michigan, 49503
Study Coordinator, 6163910284
- Recruiting
Imbria Investigational Site
Houston, Texas, 77030
Study Coordinator, 7134414960
- Not yet recruiting
Imbria Investigational Site
Houston, Texas, 77030
Study Coordinator
- Recruiting
Imbria Investigational Site
La Jolla, California, 92037
Study Coordinator, 858-246-5640
- Recruiting
Imbria Investigational Site
Los Angeles, California, 90048
Study Coordinator, 6264094913
- Recruiting
Imbria Investigational Site
Miami, Florida, 33136
Study Coordinator, 7865785926
- Not yet recruiting
Imbria Investigational Site
Milwaukee, Wisconsin, 53215
Study Coordinator
- Recruiting
Imbria Investigational Site
New York, New York, 10016
Study Coordinator, 6465010568
- Not yet recruiting
Imbria Investigational Site
New York, New York, 10032
Study Coordinator, (212) 305-1368
- Not yet recruiting
Imbria Investigational Site
Oak Lawn, Illinois, 60453
Study Coordinator, 708.684.5137
- Recruiting
Imbria Investigational Site
Philadelphia, Pennsylvania, 19104
Study Coordinator, 2156623768
- Recruiting
Imbria Investigational Site
Philadelphia, Pennsylvania, 19107
Study Coordinator, (215) 503-4913
- Recruiting
Imbria Investigational Site
Plano, Texas, 75093
Study Coordinator, 4698144853
- Recruiting
Imbria Investigational Site
Portland, Oregon, 97239
Study Coordinator, 5034948799
- Recruiting
Imbria Investigational Site
Richmond, Virginia, 23298
Study Coordinator, 8048281601
- Recruiting
Imbria Investigational Site
Rochester, Minnesota, 55905
Study Coordinator, 5072844253
- Not yet recruiting
Imbria Investigational Site
Salt Lake City, Utah, 84132-0001
Study Coordinator
- Recruiting
Imbria Investigational Site
San Francisco, California, 94143
Study Coordinator, 415-476-2060, Jonathan.Thomas2@ucsf.edu
- Recruiting
Imbria Investigational Site
St Louis, Missouri, 63110
Study Coordinator, 314-362-1962
- Recruiting
Imbria Investigational Site
Washington D.C., District of Columbia, 20010
Study Coordinator, 202-877-6334
Spain17 sites, 17 recruiting
- Recruiting
Imbria Investigational Site
A Coruña, Galicia, 15006
Study Coordinator, +34 981 176 540
- Recruiting
Imbria Investigational Site
Barcelona, Barcelona, 08035
Study Coordinator, +34 677 381 128
- Recruiting
Imbria Investigational Site
Barcelona, 8036
Study Coordinator, 3463329611
- Recruiting
Imbria Investigational Site
Barcelona, 8041
Study Coordinator, 3493291900
- Recruiting
Imbria Investigational Site
Barcelona, 8907
Study Coordinator, 9326073202108
- Recruiting
Imbria Investigational Site
El Palmar, 30120
Study Coordinator, 34689931292
- Not yet recruiting
Imbria Investigational Site
Granada, 18014
Study Coordinator
- Recruiting
Imbria Investigational Site
Madrid, Madrid, 28034
Study Coordinator, 34 913 368 954
- Not yet recruiting
Imbria Investigational Site
Madrid, 28046
Study Coordinator
- Recruiting
Imbria Investigational Site
Madrid, 28222
Study Coordinator, 34911917297
- Recruiting
Imbria Investigational Site
Palma de Mallorca, 07198
Study Coordinator, 34871202102
- Recruiting
Imbria Investigational Site
Salamanca, 37007
Study Coordinator, 34658403315
- Recruiting
Imbria Investigational Site
Seville, Sevilla, 41009
Study Coordinator, 34 600 162 398
- Recruiting
Imbria Investigational Site
Seville, Sevilla, 41013
Study Coordinator, +34 955 013 105
- Recruiting
Imbria Investigational Site
Valencia, 03010
Study Coordinator, 34965913678
- Recruiting
Imbria Investigational Site
Valencia, 46026
Study Coordinator, 34965913921
- Recruiting
Imbria Investigational Site
Vigo, Pontevedra, 36312
Study Coordinator
Italy7 sites, 7 recruiting
- Not yet recruiting
Imbria Investigational Site
Bologna, 40138
Study Coordinator
- Recruiting
Imbria Investigational Site
Florence, 50139
Study Coordinator, 390557945138
- Recruiting
Imbria Investigational Site
Massa, 54100
Study Coordinator, 393277861790
- Recruiting
Imbria Investigational Site
Milan, 20131
Study Coordinator, 390264447940
- Recruiting
Imbria Investigational Site
Milan, 20146
Study Coordinator, +39 02619112895
- Recruiting
Imbria Investigational Site
Milan, 20162
Study Coordinator, +39 0264446715
- Recruiting
Imbria Investigational Site
Naples, 80131
Study Coordinator, +39 3357621204
United Kingdom5 sites, 5 recruiting
- Recruiting
Imbria Investigational Site
Glasgow, Scotland, G51 4TF
Study Coordinator, 44-141 232 7600
- Recruiting
Imbria Investigational Site
London, England, W12 0HS
Study Coordinator, 44 020 3313 7156
- Recruiting
Imbria Investigational Site
London, EC1A 7BE
Study Coordinator, 442037658635
- Recruiting
Imbria Investigational Site
London, SW3 6HP
Study Coordinator, 4403301288121
- Recruiting
Imbria Investigational Site
Oxford, OX3 9DU
Study Coordinator, 441865234554
Germany4 sites, 4 recruiting
- Recruiting
Imbria Investigational Site
Bad Oeynhausen, 32545
Study Coordinator, 495731973399
- Recruiting
Imbria Investigational Site
Göttingen, 37075
Study Coordinator, 4905513962720
- Recruiting
Imbria Investigational Site
Heidelberg, 69120
Study Coordinator, 4962215638336
- Not yet recruiting
Imbria Investigational Site
Jena, 07747
Study Coordinator, 4915232184941
Netherlands4 sites, 4 recruiting
- Recruiting
Imbria Investigational Site
Amsterdam-Zuidoost, 1105 AZ
Study Coordinator, 310204444912
- Not yet recruiting
Imbria Investigational Site
Leiden, 2333ZC
Study Coordinator, 31715263009
- Not yet recruiting
Imbria Investigational Site
Nijmegen, 6525 GA
Study Coordinator, 31243616785
- Recruiting
Imbria Investigational Site
Rotterdam, 3015 AA
Study Coordinator, +31 10 704 0 704
Portugal4 sites, 4 recruiting
- Recruiting
Imbria Investigational Site
Guimarães, 4835-044
Study Coordinator
- Recruiting
Imbria Investigational Site
Lisbon, 1169-024
Study Coordinator
- Recruiting
Imbria Investigational Site
Lisbon, 1500-650
Study Coordinator, 351969870409
- Not yet recruiting
Imbria Investigational Site
Lisbon, 1998-018
Study Coordinator
France3 sites, 3 recruiting
- Recruiting
Imbria Investigational Site
Montpellier, 34295
Study Coordinator, +33 467 336 431
- Recruiting
Imbria Investigational Site
Paris, 75015
Study Coordinator, 33 1 56 09 27 27
- Recruiting
Imbria Investigational Site
Saint-Herblain, 44800
Study Coordinator, +33 240 165 710
Poland3 sites, 3 recruiting
- Recruiting
Imbria Investigational Site
Katowice, 40-555
Study Coordinator, +48 604 122 669
- Recruiting
Imbria Investigational Site
Warsaw, 02-507
Study Coordinator, 48227221113
- Recruiting
Imbria Investigational Site
Wroclaw, 53-114
Study Coordinator, 48 517 311 583
Austria1 site, 1 recruiting
- Recruiting
Imbria Investigational Site
Graz, 8036
Study Coordinator, 4331638580264
Belgium1 site, 1 recruiting
- Recruiting
Imbria Investigational Site
Edegem, 2650
Study Coordinator, 3238213000
About this study
Official 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
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
- Being tested
- Ninerafaxstat 200mg MR, Placebo
- Keywords
- nHCM, non-obstructive HCM, non-obstructive hypertrophic cardiomyopathy, HCM, hypertrophic cardiomyopathy
- Medical subject headings
- Cardiomyopathy, Hypertrophic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ninerafaxstat 200mg MRDrug
Ninerafaxstat 200mg Modified Release tablet administered BID
PlaceboDrug
Matching placebo tablet administered BID
| Study group | Role | What they receive |
|---|---|---|
| Ninerafaxstat | Experimental | Not described Receives: Drug: Ninerafaxstat 200mg MR |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline to Week 12
Secondary outcomes (4)
Change in ventilatory efficiency (VE/VCO2 slope) during cardiopulmonary exercise testing (CPET)
Measured: Baseline to Week 12
Change in the Kansas City Cardiomyopathy Questionnaire Total Symptom Score (KCCQ-TSS)
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.
Measured: Baseline to Week 12
Change in the Kansas City Cardiomyopathy Questionnaire Physical Limitations Score (KCCQ-PLS)
The Physical Limitations Score is a single-domain score. Scoring ranges from 0 to 100, with higher scores indicating better health status.
Measured: Baseline to Week 12
Change in the Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS)
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.
Measured: Baseline to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2025
- Study start6 Oct 2025
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Non-obstructive Hypertrophic CardiomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 165 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Imbria Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07023614
- Sponsor's protocol ID
- IMB101-010
- Acronym
- FORTITUDE-HCM
- Status last verified
- Oct 2026
- First submitted
- 9 Jun 2025
- Last updated
- 26 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT07023614
- Acronym / sponsor ID
- FORTITUDE-HCM / IMB101-010
- Keywords
- nHCM, non-obstructive HCM, non-obstructive hypertrophic cardiomyopathy, HCM, hypertrophic cardiomyopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 165 participants (estimated)
- Lead sponsor
- Imbria Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 6 Oct 2025 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 17 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ninerafaxstat | Experimental | Drug: Ninerafaxstat 200mg MR |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ninerafaxstat 200mg MRNinerafaxstat 200mg Modified Release tablet administered BID | Drug | Ninerafaxstat |
| PlaceboMatching placebo tablet administered BID | Drug | Placebo |
Primary outcome
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.
4 secondary outcomes
Change in ventilatory efficiency (VE/VCO2 slope) during cardiopulmonary exercise testing (CPET)
Time frame: Baseline to Week 12
Change in the Kansas City Cardiomyopathy Questionnaire Total Symptom Score (KCCQ-TSS)
Time frame: Baseline to Week 12
Change in the Kansas City Cardiomyopathy Questionnaire Physical Limitations Score (KCCQ-PLS)
Time frame: Baseline to Week 12
Change in the Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS)
Time frame: Baseline to Week 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2026.
- Individual participant dataSharing planned: Undecided
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Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of HTX-001
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Not yet recruitingPhase 4
Dapagliflozin in Patients With Non-Obstructive Hypertrophic Cardiomyopathy: A Prospective, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial
Nonobstructive Hypertrophic Cardiomyopathy
Enrolling by invitationPhase 2/3
Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Aficamten in Adults With HCM
Symptomatic Hypertrophic Cardiomyopathy (HCM)
Not yet recruiting
Coronary Sinus Reducer Objective Impact on SymptoMs, Ischaemia and miCrovascular Resistance in Non-obstructive Hypertrophic CardioMyopathy
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Not yet recruitingObservational study
Aficamten and Patient-Reported Outcomes in Real-World nHCM
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
76 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ninerafaxstat 200mg MR in people with Non-obstructive Hypertrophic Cardiomyopathy, run by Imbria Pharmaceuticals, Inc. at 76 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 76 sites in United States, Spain, Italy, United Kingdom, Germany and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Medical Monitor ((617) 675-4060, info@imbria.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) (time frame: Baseline to Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2026.