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)
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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
- Age range
- 18–65 years
- Sex
- All
- Healthy volunteers
- No
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 \<30 seconds. 3. Able to understand and comply with all study procedures. 4. Sexually active females of childbearing potential and female and male partners of male subjects receiving study intervention must use a barrier method of contraception for the first 6 months after dosing. Exclusion Criteria: 1. Significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction \<40%), evidence o…
Interventions
- GeneticAB-1003 dose level 1
Single intravenous infusion of AB-1003 gene therapy at dose level 1
- GeneticAB-1003 dose level 2
Single intravenous infusion of AB-1003 gene therapy at dose level 2
- OtherPlacebo
Single intravenous infusion of Placebo
Locations (6)
- University of California - IrvineIrvine, California
- University of IowaIowa City, Iowa
- University of Kansas Medical CenterKansas City, Kansas
- Kennedy Krieger InstituteBaltimore, Maryland
- VCURichmond, Virginia
- University of Washington Medical CenterSeattle, Washington