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Rp-a501    (DrugBank: -)

1 disease
IDDisease name (Link within this page)Number of trials
32Autophagic vacuolar myopathy1

32. Autophagic vacuolar myopathy    [ 1 clinical trial,   1 drug,   (DrugBank: 0),   0 drug target gene,   0 drug target pathway]
Searched query = "Autophagic vacuolar myopathy", "Danon disease", "X-linked myopathy with excessive autophagy", "XMEA"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 1 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT03882437
(ClinicalTrials.gov)		April 17, 201912/3/2019Gene Therapy for Male Patients With Danon Disease Using RP-A501; AAV9.LAMP2BGene Therapy for Danon Disease (DD): A Clinical Study Evaluating the Infusion of a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B) Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon DiseaseDanon DiseaseBiological: RP-A501Rocket Pharmaceuticals Inc.NULLRecruiting8 YearsN/AMale24Phase 1United States