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.
Studieöversikt
Status
Betingelser
Intervention / Behandling
Studietyp
Inskrivning (Beräknad)
Kontakter och platser
Studiekontakt
- Namn: Xiaojie Xie, MD, PhD
- Telefonnummer: 86+571-87784700
- E-post: xiexj@zju.edu.cn
Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
Tar emot friska volontärer
Kön som är behöriga för studier
Testmetod
Studera befolkning
Patients with symptomatic non-obstructive hypertrophic cardiomyopathy
Beskrivning
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.
Studieplan
Hur är studien utformad?
Designdetaljer
- Observationsmodeller: COHORT
- Tidsperspektiv: PROSPECTIVE
Kohorter och interventioner
Grupp / Kohort |
Intervention / Behandling |
|---|---|
|
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. |
Vad mäter studien?
Primära resultatmått
Resultatmått |
Tidsram |
|---|---|
|
Percentage change from baseline in NT-proBNP and/or absolute change from baseline in KCCQ-CSS
Tidsram: From enrollment to the end of treatment at 24 months
|
From enrollment to the end of treatment at 24 months
|
Sekundära resultatmått
Resultatmått |
Tidsram |
|---|---|
|
Absolute change from baseline in NT-proBNP
Tidsram: 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
Tidsram: 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
Tidsram: 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)
Tidsram: 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'
Tidsram: 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)
Tidsram: 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
Tidsram: From enrollment to the end of treatment at 24 months
|
From enrollment to the end of treatment at 24 months
|
Samarbetspartners och utredare
Studieavstämningsdatum
Studera stora datum
Studiestart (Beräknad)
Primärt slutförande (Beräknad)
Avslutad studie (Beräknad)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Faktisk)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- 2026-1382
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