Aficamten and Patient-Reported Outcomes in Real-World nHCM

NCT07846176 | Not Yet Recruiting DMC FDA Drug

• 1 other identifier: 2026-1382
• Study Type: observational
• Target: 100
• Locations: 0 countries
• Sites: N/A

Brief Summary

This multicenter, prospective, observational, real-world clinical study aims to evaluate the efficacy and safety of Aficamten in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM) by prospectively observing changes in symptoms, biomarkers, imaging findings, and adverse reactions, etc. It will also record the dosing regimens at which individual patients achieve steady state, and explore the treatment patterns of Aficamten in Chinese patients with symptomatic nHCM.

Conditions

Hypertrophic Cardiomyopathy (HCM)

Keywords

non obstructive hypertrophic cardiomyopathy; aficamten

Outcome Measures

Primary Outcomes (1)

• Percentage change from baseline in NT-proBNP and/or absolute change from baseline in KCCQ-CSS

  From enrollment to the end of treatment at 24 months

Secondary Outcomes (7)

• Absolute change from baseline in NT-proBNP

  From enrollment to the end of treatment at 24 months

• Percentage change from baseline in TnT/TnI

  From enrollment to the end of treatment at 24 months

• Absolute change from baseline in TnT/TnI

  From enrollment to the end of treatment at 24 months

• Absolute change from baseline in maximal wall thickness (MWT)

  From enrollment to the end of treatment at 24 months

• Absolute change from baseline in E/e'

  From enrollment to the end of treatment at 24 months

Study Arms (1)

Aficamten group

Patients with non-obstructive hypertrophic cardiomyopathy (nHCM) receiving aficamten therapy

Drug: receiving aficamten

Interventions

receiving aficamten DRUG

Aficamten will be administered at a specified dose according to each patient's tolerability, as assessed by echocardiography.

Aficamten group

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Patients with symptomatic non-obstructive hypertrophic cardiomyopathy

You may qualify if:

• Age 18-85 years.
• Confirmed diagnosis of HCM:
• Left ventricular wall thickness ≥15 mm at any site on echocardiography or cardiac magnetic resonance.
• Left ventricular wall thickness ≥13 mm in individuals testing positive for a pathogenic variant or in genetically affected family members.
• Ventricular wall thickening caused by other cardiovascular diseases or systemic or metabolic disorders has been excluded.
• Symptomatic nHCM patients meeting all of the following conditions:
• Left ventricular outflow tract gradient (LVOTG) \<30 mmHg at rest and/or with provocation.
• Presence of any one of the following symptoms: fatigue (weakness), dyspnea, chest pain, palpitations, or syncope.
• New York Heart Association (NYHA) functional class II or III.
• Left ventricular ejection fraction (LVEF) ≥60% as assessed by transthoracic echocardiography.
• Signed and dated written informed consent, and willingness to return for clinical follow-up.

You may not qualify if:

• LVOTG ≥30 mmHg at rest and/or with provocation.
• LVEF \<50% on transthoracic echocardiography.
• Symptomatic hypotension or systolic blood pressure (SBP) \<100 mmHg.
• Severe renal impairment, estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73 m², or currently receiving blood purification therapy.
• Planned implantable cardioverter-defibrillator (ICD) implantation or cardiac resynchronization therapy (CRT) within 3 months.
• Women who are pregnant or planning to become pregnant during the trial.
• Currently participating in another investigational device or drug trial.
• Any other clinical condition with an expected survival of less than 2 years.

Sponsors & Collaborators

• Second Affiliated Hospital, Zhejiang University, School of Medicine: lead

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

Xiaojie Xie, MD, PhD

CONTACT

86+571-87784700; xiexj@zju.edu.cn

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2026
• First Posted: September 29, 2026
• Study Start (Estimated): June 1, 2027
• Primary Completion (Estimated): June 1, 2029
• Study Completion (Estimated): December 1, 2029
• Last Updated: September 29, 2026

Record last verified: 2026-09