A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
This study is investigating a new medicine called ENTR-601-45 for Duchenne muscular dystrophy (DMD). Specifically, it's for boys aged 4 to 20 whose type of DMD might benefit from `exon 45 skipping`. Researchers want to understand how safe ENTR-601-45 is, what side effects it might cause, and if it could potentially help manage the condition, compared to a dummy medicine (placebo). Taking part involves receiving the study medicine through a drip into a vein and attending regular clinic appointments for checks. These checks include blood tests, physical exams, and sometimes a small muscle sample. The study has two parts to figure out the best dose and then test it further.
At a glance
What is this study about?
This research study is about a new medicine called ENTR-601-45, designed for boys who have Duchenne muscular dystrophy (DMD). DMD is a serious condition that causes muscles to weaken over time. This particular study is for boys whose DMD has a specific genetic change that might respond to a treatment strategy called `exon 45 skipping`.
The main goals of this study are to find out if ENTR-601-45 is safe to use and to understand any side effects it might cause. We also want to see if it could help improve the condition. To do this, some participants will receive ENTR-601-45, and others will receive a `placebo`. A placebo looks exactly like the study medicine but doesn't contain any active ingredients. This helps researchers compare the effects accurately. All participants will continue to receive their usual DMD care alongside the study treatment.
The study is split into two main parts. In Part A, we'll test different doses of ENTR-601-45 to find the safest and most effective one. Then, in Part B, we'll continue to test that chosen dose to learn even more about its effects and safety. After these parts, participants might have the option to continue receiving the medicine in an `open-label` period, meaning everyone would know they are getting the active drug.
Key takeaways
- This study is for boys (4-20 years old) with Duchenne muscular dystrophy amenable to exon 45 skipping.
- It tests a new medicine, ENTR-601-45, for safety and effectiveness.
- Participants will receive the medicine via a drip into a vein.
- Regular clinic visits, blood tests, and muscle biopsies are part of the study.
- Some participants will receive the active drug; others will receive a placebo.
- You can continue your standard DMD care during the study.
Who may be eligible?
To join this study, we are looking for boys, aged between 4 and 20 years old, who have Duchenne muscular dystrophy (DMD). Your doctor will need to check that your specific genetic type of DMD could potentially benefit from `exon 45 skipping` – this is a key part of the study.
You should generally be able to walk, or at least have a good level of upper limb function. You also need to be able to have a small muscle sample (biopsy) taken safely, as this helps us understand how the medicine works. Your overall health should be stable, without any major illnesses that could interfere with the study.
There are also reasons why someone might not be able to join. For example, if you've recently had other treatments for DMD that work in similar ways (like gene therapy or certain other `exon-skipping` drugs) within the last year, or certain other medications like blood thinners or specific immunosuppressants. We'll also check your blood test results and heart health to make sure it's safe for you to take part.
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 boy aged between 4 and 20 years old?
- Do you have a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD)?
- Has your doctor confirmed your DMD could benefit from 'exon 45 skipping'?
- Are you generally able to walk (even if with help) or have good upper body strength?
- Are you able to have a small muscle sample (biopsy) taken safely?
- Have you avoided certain other DMD treatments or specific medications recently?
What does participation involve?
If you decide to join this study, you will receive the study medicine (either ENTR-601-45 or the placebo) through a drip, which is a slow injection into a vein. These `infusions` will happen regularly over several weeks in both parts of the study.
You’ll need to visit the clinic often for check-ups. These visits will include various tests such as blood and urine tests, physical exams, and questionnaires about how you're feeling. To understand how the medicine affects your muscles, you'll have a small muscle sample (a `biopsy`) taken at the very beginning of your participation and again after your last dose of the study medicine. You might also do exercise tests.
Throughout the study, you'll continue to receive your usual medical care for DMD, provided your health remains stable. If you qualify and choose to, you may also have the option to continue receiving the study medicine after the main parts of the research are complete. The total length of your participation will depend on the study part and whether you choose to enter the extended treatment period.
Potential risks and benefits
Locations (15)
- 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
- Ospedale Pediatrico Bambino GesuVerified postcodeRome, Italy· Recruiting
- Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro CuoreVerified postcodeRome, Italy· Recruiting
- Leids Universitair Medisch CentrumVerified postcodeLeiden, Netherlands· Recruiting
- Stichting Radboud Universitair Medisch CentrumVerified postcodeNijmegen, Netherlands· 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
Common questions
What is 'exon 45 skipping'?
DMD is caused by a genetic mistake. 'Exon 45 skipping' is a way to try and fix parts of that mistake in a specific group of patients, allowing the body to make a more complete, though slightly shorter, muscle protein.
What is a placebo?
A placebo is a dummy medicine that looks just like the real study drug but doesn't contain any active ingredients. It helps researchers compare the effects of the new medicine accurately.
Will I know if I'm getting the real medicine or the placebo?
No, initially neither you nor your doctor will know whether you are receiving ENTR-601-45 or the placebo. This is to ensure the study results are fair. However, there might be an option to receive the active medicine in a later part of the study.
What is a muscle biopsy?
A muscle biopsy is a small procedure where a tiny piece of muscle tissue is taken, usually from your leg. It helps researchers see how the medicine might be affecting your muscles at a cellular level.
Can I continue my usual DMD medicines?
Yes, you can typically continue taking your regular treatment for DMD, like steroids, as long as your health generally remains stable throughout the study.
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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