LION-101 dose level 2 ( 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
1NCT05230459
(ClinicalTrials.gov)
March 12, 202327/1/2022A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)A Phase 1/2, Double-blind, Randomized, Placebo-controlled, Dose-escalation Study to Evaluate the Safety of LION-101 Gene Therapy in Adult Subjects (18-65 Years) With Genetic Confirmation of Limb Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) - Part 1Limb Girdle Muscular Dystrophy;Limb-Girdle Muscular Dystrophy Type 2;LGMD2I;Muscular Dystrophy;LGMD2;LGMD;FKRP;FKRP Mutation;Fukutin Related ProteinGenetic: LION-101 dose level 1;Genetic: LION-101 dose level 2;Other: PlaceboAsklepios Biopharmaceutical, Inc.NULLRecruiting18 Years65 YearsAll10Phase 1/Phase 2United States