NCT05230459 - A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

May 15, 2023

Primary Completion Date

December 31, 2028

Study Completion Date

December 31, 2028

Conditions

Limb Girdle Muscular DystrophyLimb-Girdle Muscular Dystrophy Type 2LGMD2IMuscular DystrophyLGMD2LGMDFKRPFKRP MutationFukutin Related Protein

Interventions

GENETIC

AB-1003 dose level 1

Single intravenous infusion of AB-1003 gene therapy at dose level 1

GENETIC

AB-1003 dose level 2

Single intravenous infusion of AB-1003 gene therapy at dose level 2

OTHER

Placebo

Single intravenous infusion of Placebo

Trial Locations (6)

RECRUITING

Kennedy Krieger Institute, Baltimore

RECRUITING

VCU, Richmond

RECRUITING

University of Iowa, Iowa City

RECRUITING

University of Kansas Medical Center, Kansas City

RECRUITING

University of California - Irvine, Irvine

RECRUITING

University of Washington Medical Center, Seattle