Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association with Imlifidase in Subjects with Duchenne Muscular Dystrophy with Pre-existing Antibodies to rAAVrh74

This study is investigating an experimental gene therapy called "delandistrogene moxeparvovec-rokl" for Duchenne muscular dystrophy (DMD). It focuses specifically on males with DMD who have pre-existing antibodies, which can sometimes make gene therapies less effective. The study also uses a medication called "Idefirix" alongside the gene therapy. Researchers want to see if this combination therapy is safe, how well participants tolerate it, and if the gene therapy successfully helps their muscles produce a specific protein. This is an early-stage study, meaning it's primarily designed to understand the treatment's basic effects in humans.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

Sarepta Therapeutics Inc.

Enrolment target

Start

29 Aug 2024

Duchenne muscular dystrophy (DMD)

What is this study about?

This study is looking into a new treatment approach for Duchenne muscular dystrophy (DMD), a genetic condition that causes muscles to weaken over time. The main treatment being tested is a 'gene therapy' called delandistrogene moxeparvovec-rokl. Gene therapies aim to deliver new genetic material into cells to help them work properly. In DMD, the body doesn't make enough of a protein called dystrophin, which is vital for healthy muscles. This gene therapy is designed to help the body produce this missing dystrophin.

A key part of this study is its focus on participants who already have certain antibodies in their blood. Antibodies are like your body's defence system; they normally fight off infections, but sometimes they can also target and potentially block treatments, especially gene therapies. To address this, the study includes another medication called Idefirix. The researchers want to see if Idefirix can help reduce these antibodies, allowing the gene therapy to work more effectively.

Because this is an early-stage study, called a Phase 1 study, the main goals are to make sure the treatment combination is safe for people and to see how well their bodies tolerate it. They will also be measuring whether the gene therapy successfully helps muscle cells produce the dystrophin protein that is so crucial for muscle function. The results from this study will help decide if this treatment combination can move forward into larger, more extensive trials.

Key takeaways

This study is testing a new gene therapy for Duchenne muscular dystrophy.
It's for males who specifically have certain pre-existing antibodies.
Another medication (Idefirix) is used to try and manage these antibodies.
The main goals are to check the treatment's safety and if it helps muscles make dystrophin.
This is an early-stage (Phase 1) study, focusing on understanding basic effects and safety.
Participation involves infusions, muscle biopsies, blood tests, and long-term safety monitoring.

Who may be eligible?

This study is specifically designed for males. If you're a male with Duchenne muscular dystrophy, you might be able to take part. There are no age restrictions mentioned, so people of many ages could be considered.

However, it's very important to note that this study is for individuals who have already developed certain antibodies. These are specific antibodies that your body might have made in response to a type of virus (rAAVrh74) that is often used to deliver gene therapies. If you don't have these specific antibodies, this particular study might not be suitable for you.

Researchers will perform tests to confirm if you meet all the necessary health criteria for the study. Doctors and the study team will be able to tell you more about all the requirements and check if you are a good fit.

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.

Are you male?
Do you have a diagnosis of Duchenne muscular dystrophy?
Do you know if you have specific pre-existing antibodies to the rAAVrh74 virus?
Are you able to attend regular clinic visits and undergo medical procedures like infusions and muscle biopsies?

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive two main treatments: Idefirix, given as an infusion (into a vein), followed by the gene therapy, delandistrogene moxeparvovec-rokl, also given as an infusion. The study involves regular check-ups and tests. Initially, these checks will be quite frequent, especially in the first few days and weeks after treatment, to closely monitor your health and how your body responds.

Throughout the study, doctors will take small muscle samples (called biopsies) at certain times, including at the beginning and around 12 weeks later. These samples help them see if the gene therapy is working and producing the dystrophin protein in your muscles. You will also have blood tests to measure antibody levels and the concentration of the gene therapy in your system. The study team will assess your safety for up to 104 weeks (about two years) after the treatment, looking for any side effects.

Potential risks and benefits

Like all medical studies, there could be potential benefits and potential risks to taking part. A potential benefit is the chance to receive an investigational gene therapy that might improve your Duchenne muscular dystrophy by helping your muscles produce a vital protein. However, because this is an early-stage study, the full effects and potential side effects are still being investigated. Risks could include side effects from either Idefirix or the gene therapy itself, including reactions to the infusions or effects on other body systems. You will receive detailed information about all known risks before deciding to join. Remember, participation is completely voluntary, and you are free to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (1)

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

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Unverified
Spain

Common questions

What is Duchenne muscular dystrophy?

Duchenne muscular dystrophy (DMD) is a genetic condition that causes muscles to progressively weaken and waste away over time.

What is gene therapy?

Gene therapy is a type of treatment that aims to fix genetic problems at their source by introducing new genetic material into a person's cells.

Why is this study looking at people with antibodies?

Some people have existing antibodies that can interfere with gene therapies. This study is testing a way to potentially overcome this challenge using an additional medication.

Will I have to stay in the hospital?

While the detailed description doesn't specify hospital stays, infusions often require monitoring in a clinic or hospital setting, especially for novel treatments. The study team will provide full details.

What does 'Phase 1' mean?

Phase 1 means this is an early study to check if a new treatment is safe in humans and to learn how the body handles it. It's not primarily designed to see if it cures the condition.