Clinical trials: Pediatric Cardiomyopathy
Pediatric, Symptomatic Obstructive Hypertrophic Cardiomyopathy
A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
- Trial name
- CEDAR-HCM (NCT06412666)
- Testing
- Aficamten
- Where
- 36 sites in United States, Canada and 4 more
- Volunteers
- 65 people
- Sponsor
- Cytokinetics
Danon Disease
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B)…
- Trial name
- NCT06092034
- Testing
- RP-A501
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Myocardial Ischemia, Heart Defects, Congenital
Spectroscopic Assessment of Myocardial Oxygenation Changes in Pediatric Heart Surgery
The goal of this observational study is to investigate the ability of the SpectroCor Tissue Oxygenation Monitor and its accessory SpectroCor Sensor to observe and monitor changes in cardiac oxygen availability in pediatric…
- Conditions
- Myocardial Ischemia, Heart Defects, Congenital, Reperfusion Injury, Myocardial
- Trial name
- NCT07785141
- Where
- Finland
- Volunteers
- 30 people
- Sponsor
- Spectrocor
Danon Disease
Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2…
- Trial name
- NCT03882437
- Testing
- RP-A501
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Heart Failure, Cardiomyopathies
Assess Safety and Probable Benefit of the EXCOR® Pediatric Ventricular Assist Device Under a Continued Access Protocol
The main purpose of this protocol is to provide a mechanism for continued access for patients that would have been enrolled into the primary cohorts of the IDE study.
- Trial name
- CAP (NCT01242891)
- Testing
- EXCOR® Pediatric Ventricular Assist Device
- Sponsor
- Berlin Heart, Inc
Heart Failure, Cardiomyopathies
The Use of the EXCOR Active Driving Unit for Mobilization of Pediatric Patients With Ventricular Assist Device Support
The purpose of this observational study is to evaluate the performance of the Berlin Heart EXCOR Active driving unit while being used with the approved EXCOR ventricular assist device system.
- Conditions
- Heart Failure, Cardiomyopathies, Ventricular Dysfunction, Heart Diseases
- Trial name
- E-Motion (NCT04634708)
- Testing
- No intervention
- Where
- Germany
- Volunteers
- 24 people
- Sponsor
- Berlin Heart GmbH
Cardiomyopathy, Hypertrophic
A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
- Trial name
- NCT06253221
- Testing
- Mavacamten
- Where
- 47 sites in United States, Australia and 7 more
- Volunteers
- 44 people
- Sponsor
- Bristol-Myers Squibb
Pulmonary Arterial Hypertension, Dilated Cardiomyopathy
IntelliStent for Pulmonary Flow Adjustment in Congenital Heart Disease and Dilated Cardiomyopathy
IntelliStent is intended to achieve reduction of pulmonary hypertension, improvements in symptoms and quality of life in pediatric, adolescent and adult patients with congenital heart disease associated pulmonary arterial…
- Conditions
- Pulmonary Arterial Hypertension, Dilated Cardiomyopathy, Congenital Heart Disease
- Trial name
- HEARTFUL-CHD (NCT06072937)
- Testing
- IntelliStent
- Volunteers
- 10 people
- Sponsor
- HeartPoint Global
Heart Failure, Cardiomyopathies
Assess Safety and Probable Benefit of the EXCOR® Pediatric Ventricular Assist Device
The purpose of the study is to determine whether use of the Berlin Heart EXCOR® Pediatric Ventricular Assist Device for bridge-to-transplant is associated with a reasonable assurance of safety and probable benefit such that the…
- Trial name
- NCT00583661
- Testing
- EXCOR Pediatric
- Where
- 17 sites in United States, Canada
- Volunteers
- 48 people
- Sponsor
- Berlin Heart, Inc
Cardiomyopathy
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality…
- Trial name
- MyCLIMB (NCT05112237)
- Where
- 29 sites in United States, Canada and 2 more
- Volunteers
- 200 people
- Sponsor
- Tenaya Therapeutics