Actively Recruiting
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
Led by AskBio Inc · Updated on 2026-02-20
10
Participants Needed
6
Research Sites
498 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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)
CONDITIONS
Official Title
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female subjects aged 18 to 65 years with clinical diagnosis of LGMD2I/R9 and confirmed FKRP gene mutation
- Ability to walk or run 10 meters in less than 30 seconds
- Ability to understand and comply with all study procedures
- Sexually active females of childbearing potential and male partners must use barrier contraception for 6 months after dosing
You will not qualify if you...
- Significant cardiomyopathy or heart abnormalities including left ventricular ejection fraction below 40%, conduction defects, NYHA Class 3 or 4 heart failure, or clinically important myocardial fibrosis
- Contraindication to MRI or hypersensitivity to contrast dyes, shellfish, or iodine
- Presence of implanted spinal rods, cardiac pacemaker, or other devices that affect cardiac MRI images
- History of active chronic liver disease or abnormal liver function tests
- Abnormal kidney function (GFR below 60 ml/min)
- Life-threatening diseases including recent malignant neoplasms within 5 years (except certain skin cancers)
- Pre-existing medical conditions increasing risk beyond potential benefits
- Requirement for daytime ventilatory support
- Change in glucocorticosteroid treatment within 3 months before screening
- Exposure to investigational drugs or gene therapy within 3 months prior to treatment
- Participation in other therapeutic clinical trials
- Neutralizing antibody titer to AAV9 greater than 1:5
- Females who are pregnant, planning pregnancy within 12 months, or breastfeeding
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 6 locations
1
University of California - Irvine
Irvine, California, United States, 92697
Actively Recruiting
2
University of Iowa
Iowa City, Iowa, United States, 52242
Actively Recruiting
3
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Actively Recruiting
4
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Actively Recruiting
5
VCU
Richmond, Virginia, United States, 23298
Actively Recruiting
6
University of Washington Medical Center
Seattle, Washington, United States, 98195
Actively Recruiting
Research Team
A
AskFirst Patient Engagement
CONTACT
M
myTomorrows (see link below in reference section)
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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