A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
About the 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)
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
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 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 AB-1003 must use a barrier method of contraception for the first 6 months after dosing.
EXCLUSION CRITERIA
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 chronic liver disease (e.g. hepatitis, HIV, steatosis) or abnormal liver function (abnormal GGT and/or abnormal total/direct bilirubin and/or AST and ALT >2 ULN).
- Abnormal renal function (GFR < 60 ml/min, using the MDRD 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 baseline 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.
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study Details
Contition
Limb Girdle Muscular Dystrophy,Limb-Girdle Muscular Dystrophy Type 2,LGMD2I,Muscular Dystrophy,LGMD2,LGMD,FKRP,FKRP Mutation,Fukutin Related Protein
Age
18 - 65
Phase
PHASE1/PHASE2
Participants Needed
10
Est. Completion Date
Dec 31, 2028
Treatment Type
INTERVENTIONAL
Sponsor
Asklepios Biopharmaceutical, Inc.
ClinicalTrials.gov NCT Identifier
NCT05230459
Study Number
LION-CS101
Understanding Clinical Trials
Get answers to your questions about clinical trials.What is clinical research?What does taking part in clinical trials involve?What should I ask the trial doctor?