NCT06092034View on ClinicalTrials.gov

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

PHASE2RecruitingINTERVENTIONAL
Enrollment

14

Participants

Timeline

Start Date

September 5, 2023

Primary Completion Date

April 30, 2028

Study Completion Date

April 30, 2032

Conditions
Danon Disease
Interventions
GENETIC

RP-A501

RP-A501 is a gene therapy product consisting of a rAAV9 capsid containing the human LAMP2B transgene which will be administered as a single IV infusion.

Trial Locations (6)

19104

RECRUITING

Children's Hospital of Philadelphia, Philadelphia

92037

RECRUITING

University of California, San Diego, La Jolla

02115

RECRUITING

Boston Children's Hospital, Boston

Unknown

RECRUITING

German Heart Center Munich, Munich

RECRUITING

Meyer Childrens Hospital, Florence

RECRUITING

Great Ormund Street Hospital & UCL Institute of Cardiovascular Science, London

All Listed Sponsors
lead

Rocket Pharmaceuticals Inc.

INDUSTRY