Clinical Trial
Limb Girdle Muscular
Dystrophy Type 2I/R9
A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
What is Limb Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)?
The term limb-girdle muscular dystrophy refers to a group of conditions that cause weakness and wasting of the muscles in the arms and legs. The proximal muscles (those closest to the body such as the upper arms and thighs) are generally most affected by the condition. In LGMD2I/R9, a form of LGMD, signs and symptoms often develop in late childhood (average age 11.5 years) and may include difficulty running and walking. The symptoms gradually worsen over time, and affected people generally rely on a wheelchair for mobility within decades after onset. LGMD2I/R9 is also associated with worsening heart failure and respiratory failure.
LGMD 2I/R9 is a rare genetic disease, which means that it is caused by one or more genes not working correctly. Though it is one of the more common forms of muscular dystrophy in Northern Europe, it is inherited in an autosomal recessive manner. This genetic condition causes a mutation in fukutin related protein (FKRP). FKRP is an enzyme used to glycosylate α-Dystroglycan (α-DG) within muscle cells, a step required in the formation of muscle fibers used to maintain normal muscle integrity and function. Without sufficient FKRP, muscle fibers are prone to chronic and irreversible injury.
The medical management of LGMD 2I/R9 is only supportive. There are no disease-modifying therapies approved for the treatment of this rare disease.
About the LION-101 Clinical Study
LION-101 is a therapy that is developed for the treatment of LGMD2I/R9. LION-101 is a one-time gene therapy that targets and expresses FKRP protein, predominantly in muscle tissues.
A first-in-human clinical study in the US has been started to evaluate LION-101 for the treatment of LGMD2I/R9. This is a double-blind, randomized, placebo-controlled study, which means you may initially receive either gene therapy or placebo. However, at the end of the study all eligible patients who were initially given placebo will be offered an opportunity to receive gene therapy.
Learn more about the LION-101 clinical study (NCT05230459) at A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) – Full Text View – ClinicalTrials.gov.
LION-101 is an investigational therapy currently being studied for the treatment of LGMD2I/R9. LION-101 is not currently approved by any regulatory agency for use, as efficacy and safety have not been established.
Clinical trial activities are outlined below:
Pre-screening and Screening period
Used to determine eligibility for enrollment into the LION-101 clinical study.
Gene Therapy Administration
Administration of one infusion of LION -101 (or placebo) by single intravenous infusion at the bedside.
At the end of the study, those who initially received placebo and remain eligible will be offered gene therapy.
Health Assessments
Regular study visits during the 1st year, to measure the immediate and long-term effects of the treatment, are performed after receiving therapy.
These study visits may take place over several days at the clinical study site, local laboratory, physical therapy facility, or at a home visit.
Long Term Follow Up Period
Follow up visits will occur for total 5 years after receiving gene therapy.
Are You Eligible?
Clinical studies have specific eligibility criteria that Participants must meet, to be enrolled into the study. These are called inclusion and exclusion criteria and are related to a person’s health when they enter the screening process. Please refer to the full inclusion and exclusion criteria for the LION-101 clinical study (NCT05230459) at A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) – Full Text View – ClinicalTrials.gov.
You may be eligible if:
- Are between 18 -65 years of age and diagnosed with LGMD2I/R9.
- Have the ability to ascend 4 stairs between 2.5 and 10 seconds.
- Have the ability to walk/run 10 meters in < 30 seconds.
You are not eligible if:
- Have been diagnosed with a significant heart defect, heart failure, or heart conduction defect.
- Have chronic liver disease or abnormal liver function.
- Have abnormal kidney function.
- Require daytime ventilatory support.
- Have other life-threatening disease or medical history of life-threatening disease.
- Have the presence of neutralizing antibodies to the therapy in your blood.
The LION-101 Clinical Study
To find out if you are eligible for the LION-101 clinical trial (NCT05230459), please talk to your doctor. Visit A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) – Full Text View – ClinicalTrials.gov to learn more.
