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2 recruiting studies matching your filters
Phase 1
A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I/R9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)
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 …
Limb Girdle Muscular DystrophyLimb-Girdle Muscular Dystrophy Type 2LGMD2I+6 more
AskBio IncNCT05230459
GRASP-01-003: Trial Readiness and Endpoint Assessment in LGMD R1
This is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.
Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2ALimb Girdle Muscular DystrophyLimb Girdle Muscular Dystrophy Type R1+1 more
Virginia Commonwealth UniversityNCT05618080