A randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of ninerafaxstat in patients with symptomatic non-obstructive hypertrophic cardiomyopathy

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Imbria Pharmaceuticals Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

8 Dec 2025 → ongoing

Decision date (initial)

2025-11-23

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Imbria Pharmaceuticals, Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the effect of ninerafaxstat on health status in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).

Secondary objectives 5

1. 1. To evaluate the effect of ninerafaxstat on ventilatory efficiency during exercise in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM)
2. 2. To evaluate the effect of ninerafaxstat on patient reported symptom frequency and severity.
3. 3. To evaluate the effect of ninerafaxstat on patient reported physical limitation.
4. 4. To evaluate the effect of ninerafaxstat on patient reported health-related quality of life and overall health status.
5. 5. To evaluate the effect of ninerafaxstat on exercise capacity.

Conditions and MedDRA coding

Non-obstructive hypertrophic cardiomyopathy

Study design 2 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. All inclusion criteria can be found in the protocol (Section 5.1). 1. Informed Consent: Able to comprehend and willing to sign an informed consent form (ICF), and willing to comply with all trial procedures and restrictions for the duration specified in the Schedule of Events. Understands the risks involved.
2. 2. Age: Individual ≥18 years of age at the time of signing informed consent.
3. 3. Echocardiography: Has adequate acoustic windows for echocardiography.
4. 4. Clinical Diagnosis: Has a clinical diagnosis of hypertrophic cardiomyopathy (HCM) consistent with current American College of Cardiology/American Heart Association and European Society of Cardiology Guideline definition, with unexplained left ventricular hypertrophy (LVH) in the absence of other cardiac or systemic diseases that can produce the magnitude of hypertrophy observed.

Exclusion criteria 7

1. All exclusion criteria can be found in the protocol (Section 5.2). 1. Informed Consent: Inability or unwillingness to provide informed consent or comply with trial procedures.
2. 2. Cardiovascular Conditions: • History of obstructive hypertrophic cardiomyopathy (HCM) with left ventricular outflow tract (LVOT) gradient ≥30 mmHg at rest or with provocation. • History of major adverse cardiovascular events (MACE) within 6 months prior to screening, including myocardial infarction, stroke, or cardiac surgery.
3. 3. Medical Conditions: • Significant comorbidities that may impact the ability to complete the trial or the interpretation of results, such as uncontrolled diabetes, severe renal impairment, or active infection. • Known history of hepatitis B or C infection, or HIV infection.
4. 4. Medications: • Use of disallowed medications that may interfere with the trial, such as other investigational drugs within 30 days prior to screening. • Requirement for treatment with drugs that may affect the study outcomes, such as certain antiarrhythmic or antihypertensive medications.
5. 5. Substance Abuse: History of alcohol or substance abuse within the past year.
6. 6. Pregnancy and Lactation: • Pregnant or breastfeeding women. • Women of childbearing potential not willing to use effective contraception during the study and for a specified period after the last dose.
7. 7. Other Exclusions: • Participation in another clinical trial within 30 days prior to screening. • Any condition that, in the opinion of the investigator, would make participation in the trial unsafe or otherwise not in the best interest of the participant.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline to Week 12 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).

Secondary endpoints 5

1. 1. Change in ventilatory efficiency (VE/VCO2 slope) during CPET from baseline to Week 12
2. 2. Change from baseline to Week 12 in the KCCQ-TSS
3. 3. Change from baseline to Week 12 in the KCCQ-PLS
4. 4. Change from baseline to Week 12 in the KCCQ-OSS
5. 5. Change in exercise duration during CPET from baseline to Week 12

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Imbria Pharmaceuticals Inc.

Sponsor organisation

Imbria Pharmaceuticals Inc.

Address

265 Franklin Street Suite 1702

City

Boston

Postcode

02110-3144

Country

United States

Scientific contact point

Organisation

Imbria Pharmaceuticals Inc.

Contact name

Arash Yavari

Public contact point

Organisation

Imbria Pharmaceuticals Inc.

Contact name

Komal Joshi

Third parties 8

Locations

9 EU/EEA countries · 40 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 155 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications