Open Label Study of RP-A501 in Male Patients with Danon Disease
Research type
Research Study
Full title
Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the Human LAMP2 Isoform B Transgene (RP-A501; AAV9.LAMP2B) in Male Patients with Danon Disease
IRAS ID
1008965
Contact name
Miriam Zeini Moreno
Contact email
Sponsor organisation
Rocket Pharmaceuticals, Inc.
Research summary
SCIENTIFIC RATIONALE: Danon disease is a rare genetic disorder. Symptoms of Danon disease include the development of heart disease called cardiomyopathy. Danon disease frequently causes severe and even life-threatening heart failure at a young age.
To date, there are no effective therapies for Danon disease-associated cardiomyopathy. When heart disease becomes life-threatening, patients may need a heart transplantation.
Defects in a gene called LAMP-2B can cause Danon disease. In this clinical study, the damaged LAMP2B gene in participants with Danon disease is replaced with a correct copy of the gene, so that the heart is able to work properly. This special treatment is called ‘gene therapy’.
The Sponsor has conducted an initial study with the investigational product, RP-A501, in male participants with Danon disease. Overall, RP-A501 was generally well-tolerated in adult and paediatric participants.
The goal of this study is to provide more information on how safe and effective RP-A501 is for participants with Danon disease.
NAME OF INVESTIGATIONAL PRODUCT: RP-A501 (AAV9.LAMP2B) and is a gene therapy containing the LAMP2B transgene.
STUDY POPULATION: Males with Danon disease and confirmed LAMP2 pathogenic or likely pathogenic variants.
NUMBER OF PARTICIPANTS AND SITES PLANNED: Approximately 12 participants globally across United States and Europe.
STUDY DESIGN: This study will enroll participants ≥8 years of age with a defect in the LAMP2 gene. Participants will be followed for 60 months.
DURATION OF TREATMENT: Single intravenous infusion of RP-A501.
DATA AND SAFETY MONITORING AND OTHER COMMITTEES: The study will have 2 committees responsible for safety oversight: the Clinical Monitoring Team (CMT) and an Independent Data and Safety Monitoring Committee (IDSMC).REC name
London - West London & GTAC Research Ethics Committee
REC reference
23/LO/0932
Date of REC Opinion
31 Jan 2024
REC opinion
Further Information Favourable Opinion