Status:
RECRUITING
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
Lead Sponsor:
AskBio Inc
Conditions:
Limb Girdle Muscular Dystrophy
Limb-Girdle Muscular Dystrophy Type 2
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
PHASE2
Brief 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). Partici...
Eligibility Criteria
Inclusion
- Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation.
- Ability to ascend 4 stairs between 2.5 and 10 seconds.
- 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
- 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.
Key Trial Info
Start Date :
May 15 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2032
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT05230459
Start Date
May 15 2023
End Date
December 1 2032
Last Update
December 18 2025
Active Locations (6)
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1
University of California - Irvine
Irvine, California, United States, 92697
2
University of Iowa
Iowa City, Iowa, United States, 52242
3
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
4
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205