RAAV1.tMCK.human-alpha-sarcoglycan- Second cohort ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
113Muscular dystrophy1

113. Muscular dystrophy


Clinical trials : 646 Drugs : 471 - (DrugBank : 105) / Drug target genes : 59 - Drug target pathways : 170
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1NCT00494195
(ClinicalTrials.gov)
March 200827/6/2007Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)Phase I Gene Transfer of rAAV1.tMCK.Human-alpha-sarcoglycan for Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)Muscular DystrophiesGenetic: rAAV1.tMCK.human-alpha-sarcoglycan- First cohort;Genetic: Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohortNationwide Children's HospitalNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS);Muscular Dystrophy AssociationCompleted5 YearsN/ABoth6Phase 1United States