A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirma… (NCT05230459) | Clinical Trial Compass
RecruitingPhase 1/2
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
United States10 participantsStarted 2023-05-15
Plain-language 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)
Who can participate
Age range18 Years – 65 Years
SexALL
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Inclusion criteria
✓. 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.
Exclusion criteria
✕. 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.
✕. Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine.
✕. Implanted spinal rods, cardiac pacemaker or other implantation that would distort cardiac MRI images.
✕. 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).
✕. Abnormal renal function (GFR \<60 ml/min, using the Modification of Diet in Renal Disease equation).
✕. 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).