A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

Recruiting now · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein

In brief

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)

Key facts

Study ID

NCT05230459

Run by

AskBio Inc

People needed

10

Starts

2023-05-15

Expected to finish

2032-12-01

Last updated by the study team

2026-02-20

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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).
• In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits.
• Requirement for daytime ventilatory support.
• Change in glucocorticosteroid treatment within 3 months prior to screening visit.
• Exposure to another investigational drug within 3 months prior to study treatment or any previous treatment with gene therapy.
• Ongoing participation in any other therapeutic clinical trial.
• Neutralizing antibody titer to AAV9 >1:5.
• Female subjects who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.

Where it is running

• University of California - Irvine — Irvine, California, United States (enrolling)
• University of Iowa — Iowa City, Iowa, United States (enrolling)
• University of Kansas Medical Center — Kansas City, Kansas, United States (enrolling)
• Kennedy Krieger Institute — Baltimore, Maryland, United States (enrolling)
• VCU — Richmond, Virginia, United States (enrolling)
• University of Washington Medical Center — Seattle, Washington, United States (enrolling)

Full record on ClinicalTrials.gov

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