A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
This research is testing a new medicine called ENTR-601-44 for Duchenne muscular dystrophy (DMD), a rare genetic condition that causes muscles to weaken over time. The main goals are to find out if the medicine is safe, understand any side effects, and see if it can help improve the condition, compared to a dummy medicine (placebo). Participants will receive the study medicine through slow injections into a vein and will have regular appointments for check-ups and tests, including blood tests, physical exams, and muscle samples. The study is split into two parts, first to find the best dose and then to check its effects over a longer period. It's for males aged 4 to 20 who have a specific type of DMD.
At a glance
What is this study about?
This study is exploring a new medicine, ENTR-601-44, for a condition called Duchenne Muscular Dystrophy, or DMD. DMD is a rare genetic condition that causes muscles to become weaker over time. This particular study focuses on people with a specific type of DMD where a genetic 'mistake' can potentially be corrected using a method called 'exon 44 skipping'. Think of your genes as a set of instructions for building and running your body. Sometimes, there's a missing instruction or a piece that doesn't quite fit, and in DMD, this affects how muscles are made and repaired.
The main purpose of this research is to see if ENTR-601-44 is safe for people to use and to understand any side effects it might cause. We also want to find out if this new medicine can help improve the condition or slow its progress, compared to a placebo. A placebo looks exactly like the real medicine but doesn't contain any active ingredients. This helps researchers understand whether any changes are truly due to the medicine or other factors.
The study has two main parts. The first part aims to check the safety of the medicine and figure out the best dose to use. The second part then continues to look at how well that chosen dose works and its safety over a longer time. People taking part will get regular check-ups and tests, and importantly, they can continue with their usual DMD treatments as long as their health stays stable.
Key takeaways
- This study tests a new medicine (ENTR-601-44) for a specific type of Duchenne muscular dystrophy.
- The main goals are to check its safety, side effects, and potential benefits.
- Participants will receive the medicine through regular IV infusions.
- Regular clinic visits, blood tests, and muscle samples are part of taking part.
- It's for males aged 4-20 with a particular genetic profile.
- Your current DMD treatments can likely continue during the study.
Who may be eligible?
This study is looking for males born with Duchenne muscular dystrophy (DMD) between 4 and 20 years old. An important requirement is that your specific type of DMD must be suitable for a treatment approach called 'exon 44 skipping'. This means your genetic report needs to show a particular kind of change in your dystrophin gene. You should also be able to walk, and the doctors will need to be able to take a small muscle sample safely.
There are also some reasons why you might not be able to join. For example, if you have other serious health conditions that could interfere with the study or your safety. You also can't have taken similar gene therapies or certain other medications, like some blood thinners or specific immunosuppressants (unless they are corticosteroids for DMD). Your heart health will be checked, and you cannot have certain abnormal heart readings. If you've been on a ventilator during the day, you also won't be able to participate. The study team will review all your medical information carefully to see if you're 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 a male, assigned male at birth?
- Are you between 4 and 20 years old?
- Do you have a confirmed diagnosis of Duchenne Muscular Dystrophy that is suitable for 'exon 44 skipping'?
- Can you walk or get around on your own?
- Do you have a healthy heart, as confirmed by an ECG?
- Are you able to have a small muscle sample taken?
- Are you not currently on similar investigational therapies or certain other specific medications?
What does participation involve?
If you decide to join this study, you will receive the study medicine (either ENTR-601-44 or the placebo) through slow injections directly into a vein. These will be given multiple times over several weeks in both parts of the study. You'll need to visit the clinic regularly for various check-ups and tests. These will include blood and urine tests, physical examinations, answering questions about how you feel, and doing exercise tests to measure your strength and ability. To understand how the medicine affects your muscles, a small muscle sample (called a biopsy) will be taken at the very beginning of the study and again after your last dose of the study medicine. The total duration of your involvement will depend on the part of the study you are in, and the researchers will explain this fully. You are also allowed to continue your current standard treatments for DMD, provided your health remains stable.
Potential risks and benefits
Locations (14)
- University Hospital GentVerified postcodeGhent, Belgium· Recruiting
- UZ LeuvenVerified postcodeLeuven, Belgium· Recruiting
- Centre Hospitalier Régional de la CitadelleVerified postcodeLiège, Belgium· Recruiting
- IRCCS Ospedale San RaffaeleVerified postcodeMilan, Italy· Recruiting
- Fondazione Serena Onlus - Centro Clinico NeMO MilanoVerified postcodeMilan, Italy· Recruiting
- Ospedale Pediatrico Bambino GesuVerified postcodeRome, Italy· Recruiting
- Hospital Universitario Vall d'HebronVerified postcodeBarcelona, Spain· Recruiting
- Hospital Sant Joan de DeuVerified postcodeBarcelona, Spain· Recruiting
- Leeds General InfirmaryVerified postcodeLeeds, United Kingdom· Recruiting
- Alder Hey Children's NHS Foundation TrustVerified postcodeLiverpool, United Kingdom· Not yet recruiting
- Great Ormond Street Hospital for ChildrenVerified postcodeLondon, United Kingdom· Recruiting
- Royal Manchester Children's HospitalVerified postcodeManchester, United Kingdom· Not yet recruiting
Common questions
What is Duchenne Muscular Dystrophy (DMD)?
DMD is a rare genetic condition that causes muscles to progressively weaken and waste away over time.
What does 'exon 44 skipping' mean?
It's a way of trying to 'skip over' a genetic mistake in your DNA to help your body make a more complete, even if shorter, version of a protein called dystrophin, which is vital for muscle health.
What is a 'placebo'?
A placebo is a dummy medicine that looks just like the real medicine but doesn't contain any active ingredients. It helps researchers compare how well the new medicine works.
Will I know if I'm getting the real medicine or the placebo?
For the first part of the study, neither you, your family, nor the immediate study team will know who is getting the real medicine or the placebo. This is called 'double-blind' and helps keep the results fair and unbiased.
Can I continue my usual DMD treatments during the study?
Yes, you can typically continue your standard DMD treatments as long as your health remains stable and your study doctor agrees, and it doesn't interfere with the trial.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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