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

February 18, 2026 updated by: AskBio Inc

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)

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)

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

10

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: myTomorrows (see link below in reference section)

Study Contact Backup

Study Locations

  • United States
    • California
      • Irvine, California, United States, 92697
        • Recruiting
        • University of California - Irvine
        • Principal Investigator:
          • Tahseen Mozaffar, MD
        • Contact:
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • University of Iowa
        • Contact:
        • Principal Investigator:
          • Katherine Mathews, MD
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • University of Kansas Medical Center
        • Principal Investigator:
          • Jeffrey Statland, MD
        • Contact:
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Recruiting
        • Kennedy Krieger Institute
        • Principal Investigator:
          • Doris Leung, MD, PhD
        • Contact:
    • Virginia
      • Richmond, Virginia, United States, 23298
    • Washington
      • Seattle, Washington, United States, 98195
        • Recruiting
        • University of Washington Medical Center
        • Principal Investigator:
          • B. Jane Distad, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation.
  2. Ability to walk/run 10 meters in <30 seconds.
  3. Able to understand and comply with all study procedures.
  4. 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:

  1. 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.
  2. Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine.
  3. Implanted spinal rods, cardiac pacemaker or other implantation that would distort cardiac MRI images.
  4. 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).
  5. Abnormal renal function (GFR <60 ml/min, using the Modification of Diet in Renal Disease equation).
  6. 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).
  7. In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits.
  8. Requirement for daytime ventilatory support.
  9. Change in glucocorticosteroid treatment within 3 months prior to screening visit.
  10. Exposure to another investigational drug within 3 months prior to study treatment or any previous treatment with gene therapy.
  11. Ongoing participation in any other therapeutic clinical trial.
  12. Neutralizing antibody titer to AAV9 >1:5.
  13. Female subjects who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo (Cohorts 1 and 2)
Other: Placebo
Single intravenous infusion of Placebo
Experimental: AB-1003 Cohort 1
Genetic: AB-1003 dose level 1
Single intravenous infusion of AB-1003 gene therapy at dose level 1
Experimental: AB-1003 Cohort 2
Genetic: AB-1003 dose level 2
Single intravenous infusion of AB-1003 gene therapy at dose level 2

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse Events
Time Frame: 0-52 weeks
Treatment Emergent Adverse Events, Serious Adverse Events, Dose Limiting Toxicity
0-52 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

AskBio Inc

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 15, 2023

Primary Completion (Estimated)

December 1, 2028

Study Completion (Estimated)

December 1, 2032

Study Registration Dates

First Submitted

January 27, 2022

First Submitted That Met QC Criteria

January 27, 2022

First Posted (Actual)

February 9, 2022

Study Record Updates

Last Update Posted (Actual)

February 20, 2026

Last Update Submitted That Met QC Criteria

February 18, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • LION-CS101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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