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

This trial does not accept healthy volunteers

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 population

Male patients 8 years of age and over, Female patients 8 to 50 years of age (at the point of enrollment) with confirmed Danon disease recruited from a range of applicable care settings.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

No intervention
Other intervention
No intervention

Treatment groups

60 Participants

are divided into 2 treatment groups

Group A: Prospective Cohort1 intervention

Group B: Retrospective Cohort1 intervention