A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I/R9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)
1 other identifier
interventional
10
1 country
6
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). Participants will be treated in sequential, dose-level cohorts. (Part 1)
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started May 2023
Longer than P75 for phase_1
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 27, 2022
CompletedFirst Posted
Study publicly available on registry
February 9, 2022
CompletedStudy Start
First participant enrolled
May 15, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2032
February 20, 2026
February 1, 2026
5.6 years
January 27, 2022
February 18, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Adverse Events
Treatment Emergent Adverse Events, Serious Adverse Events, Dose Limiting Toxicity
0-52 weeks
Study Arms (3)
AB-1003 Cohort 1
EXPERIMENTALAB-1003 Cohort 2
EXPERIMENTALPlacebo (Cohorts 1 and 2)
PLACEBO COMPARATORInterventions
Single intravenous infusion of AB-1003 gene therapy at dose level 1
Single intravenous infusion of AB-1003 gene therapy at dose level 2
Eligibility Criteria
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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AskBio Inclead
Study Sites (6)
University of California - Irvine
Irvine, California, 92697, United States
University of Iowa
Iowa City, Iowa, 52242, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
VCU
Richmond, Virginia, 23298, United States
University of Washington Medical Center
Seattle, Washington, 98195, United States
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
myTomorrows (see link below in reference section)
CONTACT
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 27, 2022
First Posted
February 9, 2022
Study Start
May 15, 2023
Primary Completion (Estimated)
December 1, 2028
Study Completion (Estimated)
December 1, 2032
Last Updated
February 20, 2026
Record last verified: 2026-02
Data Sharing
- IPD Sharing
- Will not share