RecruitingLimb Girdle Muscular DystrophyLimb-Girdle Muscular Dystrophy Type 2LGMD2I
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
Eligible age
18–65 yrs
Accepts
All genders
Locations
6 states
Healthy volunteers
No
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About this study
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)
Sponsor: AskBio Inc
You may qualify if…
- ✓ 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.
You may not qualify if…
- ✕ 1. Significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction \<40%), evidence of conduction defect (increased PR and RR intervals, left bundle branch block and QTcF \>480m/sec), NYHA Class 3 or 4 heart failure, or MRI gadolinium enhancement evidence of clinically important myocardial fibrosis.
- ✕ 2. Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine.
- ✕ 3. Implanted spinal rods, cardiac pacemaker or other implantation that would distort cardiac MRI images.
- ✕ 4. History of active, ongoing chronic liver disease (e.g. hepatitis, HIV-related liver disease, hemochromatosis, steatosis, etc.) or abnormal liver function tests (abnormal GGT and/or abnormal total/direct bilirubin \>upper limit of normal \[ULN\] and/or elevated AST and ALT \>2 ULN).
- ✕ 5. Abnormal renal function (GFR \<60 ml/min, using the Modification of Diet in Renal Disease equation).
- ✕ 6. Any life-threatening disease, including malignant neoplasms and medical history or malignant neoplasms within the past 5 years prior to screening (except basal and squamous cell skin cancer).
- ✕ 7. In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits.
- ✕ 8. Requirement for daytime ventilatory support.
Where it's recruiting
Source: ClinicalTrials.gov · NCT05230459 · last updated 2026-02-20