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


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
113筋ジストロフィー1

113. 筋ジストロフィー


臨床試験数 : 646 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
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