Danon Disease Natural History Study
ConditionDanon Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age8+
SponsorRocket Pharmaceuticals Inc.
About this trial
The goal of this international observational study is to learn about the natural history of Danon disease in male patients (≥8 years of age) and female patients (8 to 50 years of age).
Eligibility criteria
Qualifiers
Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory
Patient or parent/legal guardian are capable and willing to provide signed informed consent
Age ≥ 8 years at enrollment
Evidence of left ventricular hypertrophy in the 12 months prior to or at enrollment.
Disqualifiers
Concurrent enrollment in any other clinical investigation involving use of an investigational agent for any condition at time of enrollment to this study that could confound interpretation of this study
Previous treatment with a gene therapy
Prior mechanical circulatory support at time of enrollment to this study
Prior cardiac transplantation at time of enrollment to this study
Trial design
Treatments tested in this trial
- No intervention
Treatment groups
60 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov