Active not recruitingPHASE3INTERVENTIONAL

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

This research is looking into a new experimental gene therapy called SRP-9003 for a specific type of muscular dystrophy known as Limb-girdle Muscular Dystrophy type 2E/R4 (LGMD2E/R4). The main goal is to understand how a single dose of this therapy affects the gene called beta-sarcoglycan (β-SG). This is a global study, bringing together different centers to gather information. The study will include two groups of people: those who can still walk on their own (ambulatory participants) and those who can no longer walk (non-ambulatory participants). By studying both groups, researchers hope to get a full picture of how this potential treatment works.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Sarepta Therapeutics, Inc.

Enrolment target

Start

15 Jan 2024

Estimated completion

30 Nov 2029

Limb-girdle Muscular Dystrophy

What is this study about?

This study is a clinical trial, which means it's a carefully planned research project to test a new medicine or treatment. In this case, the treatment is an experimental gene therapy called SRP-9003, and it's being tested for a condition called Limb-girdle Muscular Dystrophy type 2E/R4 (LGMD2E/R4). This type of muscular dystrophy causes muscles to weaken over time.

The main idea behind this gene therapy is to try and correct a problem with a specific gene called beta-sarcoglycan (β-SG). In people with LGMD2E/R4, there's an issue with this gene that leads to muscle weakness. The therapy aims to introduce a healthy version of this gene into the body to help the muscles work better. The researchers want to see if giving a single dose of SRP-9003 can improve how this gene functions in participants.

This is a 'Phase 3' study, which means it's one of the final stages of testing a new treatment before it might be considered for regular use. The study will involve people from different clinics around the world, and it's designed to include both individuals who are still able to walk and those who are not. By studying a wide range of people, the researchers hope to understand the full effects of this experimental treatment.

Key takeaways

This is a study of a new gene therapy (SRP-9003) for LGMD2E/R4.
It aims to see how a single dose helps with a specific gene problem.
The study includes both people who can and cannot walk.
It's a Phase 3 trial, meaning it's in the later stages of testing.
Participation involves receiving the therapy and regular check-ups.

Who may be eligible?

To join this study, participants must have Limb-girdle Muscular Dystrophy type 2E/R4 (LGMD2E/R4) and specific genetic changes in the beta-sarcoglycan gene. There are two main groups being looked for: those who can walk independently and those who cannot.

If you can walk, you would need to be able to walk a certain distance within a specific time and have good scores on some activity tests. If you cannot walk, there are different criteria related to how quickly you can walk that distance, or if you can't walk it at all, and your scores on other activity measures.

There are also some other important checks. For example, your body shouldn't have too many antibodies to a certain virus (AAVrh74). Also, you wouldn't be able to join if you have serious heart or lung problems, an autoimmune disease that needs active treatment, or any other major health issues besides your LGMD2E/R4.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do I have Limb-girdle Muscular Dystrophy type 2E/R4?
Do I have specific genetic changes in the beta-sarcoglycan gene?
Can I walk easily, or do I need help/cannot walk?
Do I have any serious heart or lung problems?
Am I currently being treated for an autoimmune disease?
Have I been told I have a high level of certain antibodies (AAVrh74)?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive a single dose of the experimental gene therapy, SRP-9003. You would also receive a type of medicine called a glucocorticoid, which helps manage the body's reaction to the gene therapy. Throughout the study, you would attend regular appointments at a clinic where doctors and nurses would perform various tests. These tests might include muscle strength assessments, walking tests, blood tests, and potentially others to see how the treatment is affecting you and your condition. The study duration isn't specified, but clinical trials typically involve several follow-up visits over months or even years to monitor effects and safety.

Potential risks and benefits

Taking part in any clinical trial has potential benefits and risks. You might benefit from receiving a new, experimental treatment that could potentially improve your condition, or help researchers learn more about LGMD2E/R4. However, as this is an experimental therapy, there's no guarantee it will work, and it might have side effects that are not yet fully known. Potential risks might include reactions to the gene therapy or the glucocorticoid medicine. You would be carefully monitored for any side effects. Remember, joining this study is completely voluntary, and you have the right to withdraw at any time, for any reason, without it affecting your medical care.

Locations (10)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of California, San Diego-Altman Clinical and Translational Research Institute
Verified postcode
La Jolla, United States
Nationwide Childrens Hospital
Verified postcode
Columbus, United States
The Children's Hospital of Philadelphia
Verified postcode
Philadelphia, United States
Children's Hospital of The King's Daughter
Verified postcode
Norfolk, United States
University Hospital Leuven (UZ Leuven)
Verified postcode
Leuven, Belgium
NMRC Gent (UZ Gent)
Verified postcode
Ghent, Belgium
Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum
Verified postcode
Essen, Germany
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Verified postcode
Milan, Italy
Hospital Sant Joan de Deu
Verified postcode
Barcelona, Spain
Newcastle University
Verified postcode
Newcastle upon Tyne, United Kingdom

Common questions

What is gene therapy?

Gene therapy is a technique that changes a person's genes to treat or prevent disease. In this study, it aims to introduce a healthy gene to help muscles work better.

What is Limb-girdle Muscular Dystrophy 2E/R4?

It's a rare genetic muscle condition that causes weakness and wasting, mainly in the muscles around the hips and shoulders. It is caused by a problem with a specific gene.

What does 'experimental' mean?

It means the treatment is new and still being tested. It hasn't been approved for general use by health authorities yet because more research is needed to prove it's safe and effective.

Why are there two groups (ambulatory and non-ambulatory)?

Researchers want to see how the treatment affects people at different stages of LGMD2E/R4, both those who can walk and those who can't, to get a complete picture of its potential benefits.

Will I know if I'm getting the active treatment?

The information provided suggests everyone in this study receives the active treatment (SRP-9003). Sometimes trials compare a new drug to a dummy treatment, but this is not mentioned here.