RP-A501 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
32自己貪食空胞性ミオパチー1

32. 自己貪食空胞性ミオパチー


臨床試験数 : 1 薬物数 : 1 - (DrugBank : 0) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0
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 (DD) Using RP-A501; AAV9.LAMP2BA Clinical Study Evaluating 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 DDDanon DiseaseBiological: RP-A501Rocket Pharmaceuticals Inc.NULLActive, not recruiting8 YearsN/AMale7Phase 1United States