LION-101 for Limb-Girdle Muscular Dystrophy

Recruiting at 5 trial locations

Overseen By Doris Leung, MD, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: AskBio Inc

Disqualifiers: Cardiomyopathy, Liver disease, Renal dysfunction, others

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

10 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing the safety and tolerability of an IV dose of AB-1003 in adults with a specific type of muscular dystrophy (LGMD2I/R9). The goal is to see if the treatment can help improve muscle function or slow down muscle damage.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, if you are on glucocorticosteroids, your treatment should not have changed in the last 3 months before the screening visit.

Eligibility Criteria

This trial is for adults aged 18-65 with a confirmed diagnosis of LGMD2I/R9 due to FKRP gene mutation. Participants should be able to climb stairs and walk 10 meters within specific times, understand study procedures, and use barrier contraception if sexually active. Exclusions include significant heart issues, MRI contraindications, other clinical trial participation, certain antibody levels, pregnancy or breastfeeding women, liver/renal diseases, cancer history in the last 5 years (except some skin cancers), and those needing ventilatory support.

Inclusion Criteria

Able to understand and comply with all study procedures

I am using or willing to use barrier contraception for 6 months after treatment.

I can walk or run 10 meters in less than 30 seconds.

Exclusion Criteria

I need help with breathing during the day.

Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine

My steroid treatment hasn't changed in the last 3 months.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous infusion of AB-1003 in sequential, dose-level cohorts

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

52 weeks

Multiple visits (in-person and virtual)

Treatment Details

Interventions

• LION-101
• Placebo

Trial Overview The study tests the safety of LION-101 in treating limb girdle muscular dystrophy type 2I/R9 through a single intravenous infusion. It's conducted in sequential groups receiving different doses of LION-101 versus placebo to determine tolerability.

Participant Groups

Group I: AB-1003 Cohort 2 Experimental Treatment 1 Intervention

Group II: AB-1003 Cohort 1 Experimental Treatment 1 Intervention

Group III: Placebo (Cohorts 1 and 2) Placebo Group 1 Intervention

LION-101 is already approved in United States, European Union for the following indications:

Approved in United States as AB-1003 for:

• Limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)

Approved in European Union as AB-1003 for:

• Limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)

Who Is Running the Clinical Trial?

AskBio Inc

Lead Sponsor

Trials

8

Recruited

440+

Asklepios Biopharmaceutical, Inc.

Lead Sponsor

Trials

8

Recruited

440+