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Active Not Recruiting
NCT05230459
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
Conditions: Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein
Sex: All
Ages: 18 Years – 65 Years
Healthy volunteers: No
Phase: PHASE1, PHASE2
Enrollment: 9
Sponsor: AskBio Inc
Location: University of California - Irvine Irvine California
Summary
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)
Eligibility Criteria
Inclusion Criteria:
1. Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation.
2. Ability to walk/run 10 meters in \upper limit of normal \[ULN\] and/or elevated AST and ALT \>2 ULN).
5. Abnormal renal function (GFR \1:5.
13. Female subjects who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.
Source: ClinicalTrials.gov (NCT05230459). StuddyBuddy aggregates publicly available trial information.
