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Gene Therapy

LION-101 for Limb-Girdle Muscular Dystrophy

Phase 1 & 2
Recruiting
Research Sponsored by Asklepios Biopharmaceutical, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Ability to walk/run 10 meters in < 30 seconds
Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 0-52 weeks
Awards & highlights

Study Summary

This trial tests if a new drug is safe & tolerable for adults with a specific type of muscular dystrophy. It will be studied in sequential groups at different dose levels.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
While not explicitly listed here, potential side effects may include reactions at the infusion site such as pain or swelling; general symptoms like fever or chills; allergic reactions; and possibly effects on muscle tissue where the drug acts.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I can walk or run 10 meters in less than 30 seconds.
Select...
I am between 18 and 65 years old and have been diagnosed with LGMD2I/R9 with a confirmed FKRP gene mutation.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~0-52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 0-52 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Adverse Events

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: AB-1003 Cohort 2Experimental Treatment1 Intervention
Group II: AB-1003 Cohort 1Experimental Treatment1 Intervention
Group III: Placebo (Cohorts 1 and 2)Placebo Group1 Intervention

Find a Location

Who is running the clinical trial?

Asklepios Biopharmaceutical, Inc.Lead Sponsor
7 Previous Clinical Trials
423 Total Patients Enrolled

Media Library

LION-101 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05230459 — Phase 1 & 2
Limb-Girdle Muscular Dystrophy Research Study Groups: AB-1003 Cohort 1, AB-1003 Cohort 2, Placebo (Cohorts 1 and 2)
Limb-Girdle Muscular Dystrophy Clinical Trial 2023: LION-101 Highlights & Side Effects. Trial Name: NCT05230459 — Phase 1 & 2
LION-101 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05230459 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

May I qualify to participate in this medical research?

"The eligibility criteria for this medical trial mandates that participants have a diagnosis of duchenne muscular dystrophy and are aged between 18 to 65 years. At present, 10 individuals may join the study."

Answered by AI

Are there any vacancies available for volunteers in this experiment?

"Affirmative. The clinicaltrials.gov is displaying that the trial, which was first posted on March 12th 2023, is presently hunting for participants. There are 10 individuals needed to be enrolled from 1 medical centre."

Answered by AI

How many participants are actively involved in this clinical trial?

"Affirmative. The clinicaltrials.gov website shows that this trial is currently recruiting participants, having been posted on March 12th 2023 and updated as of the 15th of March. 10 individuals must be recruited from 1 medical facility to complete the study."

Answered by AI

Is the age limit for this trial participants in excess of 45 years?

"To be considered for enrollment in this clinical trial, patients must range between 18 and 65 years of age. Alternatively, there are 64 medical trials specifically designed for those below 18, as well as 54 studies available to individuals over the age of 65."

Answered by AI
~7 spots leftby Dec 2028