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)
Who is this study for? Adults with genetic confirmation of limb girdle muscular dystrophy type 2I/R9
What treatments are being studied? LION-101
Status: Recruiting
Location: See all (6) locations...
Intervention Type: Genetic, Other
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
View:
• Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation.
• Ability to walk/run 10 meters in \<30 seconds.
• Able to understand and comply with all study procedures.
• 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.
Locations
United States
California
University of California - Irvine
RECRUITING
Irvine
Iowa
University of Iowa
RECRUITING
Iowa City
Kansas
University of Kansas Medical Center
RECRUITING
Kansas City
Maryland
Kennedy Krieger Institute
RECRUITING
Baltimore
Virginia
VCU
RECRUITING
Richmond
Washington
University of Washington Medical Center
RECRUITING
Seattle
Contact Information
Primary
AskFirst Patient Engagement
AskFirst@askbio.com
919-561-6210
Backup
myTomorrows (see link below in reference section)
Time Frame
Start Date: 2023-05-15
Estimated Completion Date: 2032-12
Participants
Target number of participants: 10
Treatments
Experimental: AB-1003 Cohort 1
Experimental: AB-1003 Cohort 2
Placebo_comparator: Placebo (Cohorts 1 and 2)
Related Therapeutic Areas
Sponsors
Leads: AskBio Inc