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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and tolerability of a new treatment called LION-101 for individuals with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). The study includes two groups receiving different doses of the treatment and a placebo group for comparison. It suits adults diagnosed with LGMD2I/R9 who can climb four stairs within a set time and walk or run 10 meters in under 30 seconds. Participants must also follow study procedures and meet specific health criteria. As a Phase 1 trial, this research aims to understand how LION-101 works in people, offering participants the chance to be among the first to receive this new treatment.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that AB-1003, the treatment tested in the LION-101 trial, has promising safety results from earlier studies. Data from the first group of five participants suggested the treatment was generally well-tolerated. No serious side effects occurred up to 52 weeks after treatment. Additionally, no dose-limiting toxicities emerged, indicating participants could handle the dosage without major issues. This information offers a positive outlook on the safety of AB-1003 for those considering joining the trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for muscular dystrophy?

Researchers are excited about LION-101 for Limb-Girdle Muscular Dystrophy because it represents a novel approach in gene therapy. Unlike traditional treatments for this condition, which may focus on managing symptoms with physical therapy or medications like corticosteroids, LION-101 aims to address the root cause by delivering a healthy version of the gene directly to muscle cells. This targeted delivery could potentially lead to more effective and lasting improvements in muscle strength and function. Additionally, LION-101's gene therapy approach offers hope for a one-time treatment, reducing the need for ongoing medication.

What evidence suggests that LION-101 might be an effective treatment for limb-girdle muscular dystrophy?

Research shows that LION-101, particularly the treatment AB-1003, holds promise for treating limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). In this trial, participants will receive either AB-1003 or a placebo. Early results suggest that AB-1003 is safe, as it has not caused serious side effects in the first group of patients. No issues have emerged that would halt further treatment, and no serious health problems have been reported so far. While the main focus has been on safety, these early findings encourage future studies on the treatment's effectiveness. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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)

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. askbio.com askbio.com/interim-safety-results-from-lion-cs101-clinical-trial-of-ab-1003-limb-girdle-muscular-dystrophy-2i-r9/

AskBio Presents Interim Safety Results from Phase 1 ... Data from first cohort of five participants suggest an acceptable safety profile for AB-1003 in participants with limb-girdle muscular ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05230459

NCT05230459 | A Study to Evaluate the Safety of AB-1003 ... There are three types of violations: Failure to submit required clinical trial information; Submission of false or misleading clinical trial information ...

3. cgtlive.com cgtlive.com/view/patients-treated-askbio-limb-girdle-muscular-dystrophy-gene-therapy-ab-1003-free-serious-adverse-events-52-weeks

Patients Treated With AskBio's Limb-Girdle Muscular ... 1. AskBio presents interim safety results from phase 1/phase 2 LION-CS101 clinical trial of AB-1003 in participants with limb-girdle muscular ...

4. clinicaltrialsarena.com clinicaltrialsarena.com/news/askbio-ab1003-safety-muscular-dystrophy/

AskBio reports AB-1003 safety data for limb-girdle ... AskBio reports AB-1003 safety data for limb-girdle muscular dystrophy. No dose-limiting toxicities or serious adverse events were reported up to ...

5. scge.mcw.edu scge.mcw.edu/platform/data/clinicalTrials/report/NCT05230459

Gene Therapy Trial Report 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 ...

6. finance.yahoo.com finance.yahoo.com/news/askbio-presents-interim-safety-results-120000076.html

AskBio Presents Interim Safety Results from Phase 1 ... The LION-CS101 clinical trial is a double-blind, randomized, placebo-controlled, dose-escalation trial to evaluate the safety of AB-1003 gene ...