A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Recruiting
Phase 2
Interventional Study
Danon Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 8 and older
- Sex
- Male
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 14 (estimated)
- Sponsor
- Rocket Pharmaceuticals Inc. · Industry
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About This Trial
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.
Trial Locations
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Eligibility Criteria
Key Inclusion Criteria:
1. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
2. Male.
3. Age ≥8 years.
4. Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following:
1. Abnormal thickening of Left ventricul…
Contacts