Aficamten and Patient-Reported Outcomes in Real-World nHCM (CAMPHOR-HCM)

Comprehensive Aficamten Management and Patient-reported Health Outcomes in Real-world nHCM

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Hypertrophic Cardiomyopathy (HCM)
• Intervention / Treatment: Drug: receiving aficamten

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: Xiaojie Xie, MD, PhD; Phone Number: 86+571-87784700; Email: xiexj@zju.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Patients with symptomatic non-obstructive hypertrophic cardiomyopathy

Description

Inclusion Criteria:

• Age 18-85 years.

• Confirmed diagnosis of HCM:

  1. Left ventricular wall thickness ≥15 mm at any site on echocardiography or cardiac magnetic resonance.
  2. Left ventricular wall thickness ≥13 mm in individuals testing positive for a pathogenic variant or in genetically affected family members.
  3. 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:

  1. Left ventricular outflow tract gradient (LVOTG) <30 mmHg at rest and/or with provocation.
  2. Presence of any one of the following symptoms: fatigue (weakness), dyspnea, chest pain, palpitations, or syncope.
  3. 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.

Exclusion Criteria:

• 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.

Study Plan

How is the study designed?

Design Details

• Observational Models: COHORT
• Time Perspectives: PROSPECTIVE

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Aficamten group; Patients with non-obstructive hypertrophic cardiomyopathy (nHCM) receiving aficamten therapy	Drug: receiving aficamten; Aficamten will be administered at a specified dose according to each patient's tolerability, as assessed by echocardiography.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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 Outcome Measures

Outcome Measure	Time Frame
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
Absolute change from baseline in left ventricular end-diastolic volume index (LVEDdi)	From enrollment to the end of treatment at 24 months
Proportion of patients with improvement of ≥1 NYHA functional class	From enrollment to the end of treatment at 24 months

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2027
• Primary Completion (Estimated): June 1, 2029
• Study Completion (Estimated): December 1, 2029

Study Registration Dates

• First Submitted: September 23, 2026
• First Submitted That Met QC Criteria: September 23, 2026
• First Posted (Actual): September 29, 2026

Study Record Updates

• Last Update Posted (Actual): September 29, 2026
• Last Update Submitted That Met QC Criteria: September 23, 2026
• Last Verified: September 1, 2026

More Information

Terms related to this study

• Keywords: aficamten; non obstructive hypertrophic cardiomyopathy
• Additional Relevant MeSH Terms: Aortic Valve Disease; Cardiovascular Diseases; Heart Diseases; Heart Valve Diseases; Cardiomyopathies; Aortic Stenosis, Subvalvular; Aortic Valve Stenosis; Cardiomyopathy, Hypertrophic
• Other Study ID Numbers: 2026-1382

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No