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.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Type d'étude
Inscription (Estimé)
Contacts et emplacements
Coordonnées de l'étude
- Nom: Xiaojie Xie, MD, PhD
- Numéro de téléphone: 86+571-87784700
- E-mail: xiexj@zju.edu.cn
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
Méthode d'échantillonnage
Population étudiée
Patients with symptomatic non-obstructive hypertrophic cardiomyopathy
La description
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.
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Modèles d'observation: COHORT
- Perspectives temporelles: PROSPECTIVE
Cohortes et interventions
Groupe / Cohorte |
Intervention / Traitement |
|---|---|
|
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. |
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Délai |
|---|---|
|
Percentage change from baseline in NT-proBNP and/or absolute change from baseline in KCCQ-CSS
Délai: From enrollment to the end of treatment at 24 months
|
From enrollment to the end of treatment at 24 months
|
Mesures de résultats secondaires
Mesure des résultats |
Délai |
|---|---|
|
Absolute change from baseline in NT-proBNP
Délai: 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
Délai: 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
Délai: 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)
Délai: 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'
Délai: 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)
Délai: 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
Délai: From enrollment to the end of treatment at 24 months
|
From enrollment to the end of treatment at 24 months
|
Collaborateurs et enquêteurs
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Estimé)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- 2026-1382
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
Étudie un produit d'appareil réglementé par la FDA américaine
produit fabriqué et exporté des États-Unis.
Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .