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.
Studie Overzicht
Toestand
Conditie
Interventie / Behandeling
Studietype
Inschrijving (Geschat)
Contacten en locaties
Studiecontact
- Naam: Xiaojie Xie, MD, PhD
- Telefoonnummer: 86+571-87784700
- E-mail: xiexj@zju.edu.cn
Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
Accepteert gezonde vrijwilligers
Geslachten die in aanmerking komen voor studie
Bemonsteringsmethode
Studie Bevolking
Patients with symptomatic non-obstructive hypertrophic cardiomyopathy
Beschrijving
Inclusion Criteria:
-
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.
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.
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Observatiemodellen: COHORT
- Tijdsperspectieven: PROSPECTIVE
Cohorten en interventies
Groep / Cohort |
Interventie / Behandeling |
|---|---|
|
Aficamten group
Patients with non-obstructive hypertrophic cardiomyopathy (nHCM) receiving aficamten therapy |
Aficamten will be administered at a specified dose according to each patient's tolerability, as assessed by echocardiography. |
Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Tijdsspanne |
|---|---|
|
Percentage change from baseline in NT-proBNP and/or absolute change from baseline in KCCQ-CSS
Tijdsspanne: From enrollment to the end of treatment at 24 months
|
From enrollment to the end of treatment at 24 months
|
Secundaire uitkomstmaten
Uitkomstmaat |
Tijdsspanne |
|---|---|
|
Absolute change from baseline in NT-proBNP
Tijdsspanne: From enrollment to the end of treatment at 24 months
|
From enrollment to the end of treatment at 24 months
|
|
Percentage change from baseline in TnT/TnI
Tijdsspanne: From enrollment to the end of treatment at 24 months
|
From enrollment to the end of treatment at 24 months
|
|
Absolute change from baseline in TnT/TnI
Tijdsspanne: From enrollment to the end of treatment at 24 months
|
From enrollment to the end of treatment at 24 months
|
|
Absolute change from baseline in maximal wall thickness (MWT)
Tijdsspanne: From enrollment to the end of treatment at 24 months
|
From enrollment to the end of treatment at 24 months
|
|
Absolute change from baseline in E/e'
Tijdsspanne: From enrollment to the end of treatment at 24 months
|
From enrollment to the end of treatment at 24 months
|
|
Absolute change from baseline in left ventricular end-diastolic volume index (LVEDdi)
Tijdsspanne: From enrollment to the end of treatment at 24 months
|
From enrollment to the end of treatment at 24 months
|
|
Proportion of patients with improvement of ≥1 NYHA functional class
Tijdsspanne: From enrollment to the end of treatment at 24 months
|
From enrollment to the end of treatment at 24 months
|
Medewerkers en onderzoekers
Studie record data
Bestudeer belangrijke data
Studie start (Geschat)
Primaire voltooiing (Geschat)
Studie voltooiing (Geschat)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- 2026-1382
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
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product vervaardigd in en geëxporteerd uit de V.S.
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