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

NCTID	NCT06092034 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Active
Indication	Danon Disease
Disease Ontology Term	DOID:0050437
Compound Name	RP-A501
Sponsor	Rocket Pharmaceuticals Inc.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	12
Results Posted	Not Available

Target Gene/Variant	LAMP2B
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type	none
Dose 1	6.7E13 GC/kg
Dose 2	1.1E14 GC/kg (dose discontinued)
Dose 3
Dose 4
Dose 5

No.of Trial Sites	6
Locations	United States,Italy,United Kingdom,Germany

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	Phase II enrollment completed 09/2024