Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)
This study, called ELEVATE-LTE, is for boys and young men aged 4 to 20 who have Duchenne Muscular Dystrophy (DMD) and have already taken part in a previous trial for the same medicines. The main aim is to see how safe the trial medicines, ENTR-601-44 and ENTR-601-45, are over a longer period, and if they continue to help improve muscle health. Participants will receive these medicines through a drip into a vein and will have regular check-ups, including blood tests, physical exams, and muscle tests. You can continue your usual DMD treatments during the study. This study helps researchers understand the long-term effects of these new potential treatments.
At a glance
What is this study about?
This research is called the ELEVATE-LTE study and it's looking into two new trial medicines, ENTR-601-44 and ENTR-601-45, for Duchenne Muscular Dystrophy (DMD). DMD is a condition that causes muscles to get weaker over time. These medicines are designed to help with the underlying problem in some people with DMD.
Researchers want to see if these medicines are safe to use over a longer period and if they continue to be helpful for muscle strength and health. This study is an 'extension' of earlier trials, meaning participants have already started taking these medicines in a previous study. By continuing to follow these participants, the researchers can gather important information about the long-term effects that is not possible to get from shorter studies.
The information gathered from this study, both about safety and any changes in muscle health, will help doctors and researchers understand if these new medicines could be a good treatment for DMD in the future. Finding effective, long-term treatments is a big step towards improving the lives of individuals with DMD.
Key takeaways
- This study is for boys and young men with DMD, aged 4-20.
- It's an extension of previous trials for two new medicines for DMD.
- The goal is to check the long-term safety and effectiveness of the medicines.
- Participation involves regular clinic visits, IV drips, and medical tests like blood tests and muscle biopsies.
- You can continue your standard DMD care while in the study.
- You can leave the study at any time.
Who may be eligible?
This study is specifically for boys and young men aged between 4 and 20 years old who have Duchenne Muscular Dystrophy (DMD). To join this particular study, you must have already taken part in and successfully completed one of the previous studies for ENTR-601-44 or ENTR-601-45. It's important that you, or your parent or guardian if you're under 18, are happy and able to agree to take part.
There are also some things that might mean you can't join. For example, if there were any safety concerns during your previous study that mean receiving the medicine again might not be safe, or if doctors think you might struggle to stick with the study plan. If you are a male and are sexually active with a female partner who could get pregnant, you would need to agree to use contraception like condoms during the study.
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.
- Am I a male aged between 4 and 20 years old?
- Do I have Duchenne Muscular Dystrophy (DMD)?
- Have I already taken part in a previous study for ENTR-601-44-201 or ENTR-601-45-201?
- Am I (or my parent/guardian) able to understand and agree to take part?
- If I am sexually active with a female partner who could get pregnant, am I willing to use condoms?
What does participation involve?
If you join this study, you will continue to receive the study medicine, ENTR-601-44 or ENTR-601-45, through a drip into a vein. These infusions (slow injections) will happen regularly over several weeks. The dose you receive will start at the same level you finished your previous study on, but it might be changed later based on how well everyone is doing in the study.
You will need to visit the clinic regularly for check-ups. These visits will include tests like blood and urine samples, physical examinations, and answering questions about how you feel. You will also do exercise tests to see how your muscles are working. At the beginning of the study and again after your last dose, you will have a muscle biopsy. This is a small sample taken from a muscle to help researchers see if the medicine has caused any changes. You can continue your usual treatments for DMD as long as your health is stable throughout the study. The total duration of your participation will depend on the study plan, but it's designed to be a long-term follow-up.
Potential risks and benefits
Locations (17)
- University Hospital GentVerified postcodeGhent, Belgium
- UZ LeuvenVerified postcodeLeuven, Belgium
- Centre Hospitalier Régional de la CitadelleVerified postcodeLiège, Belgium
- IRCCS Ospedale San RaffaeleVerified postcodeMilan, Italy
- Fondazione Serena Onlus - Centro Clinico NeMO MilanoVerified postcodeMilan, Italy
- Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro CuoreVerified postcodeRoma, Italy
- Ospedale Pediatrico Bambino GesuVerified postcodeRome, Italy
- Leids Universitair Medisch CentrumVerified postcodeLeiden, Netherlands
- Stichting Radboud Universitair Medisch CentrumVerified postcodeNijmegen, Netherlands
- Hospital Universitario Vall d'HebronVerified postcodeBarcelona, Spain
- Hospital Sant Joan de DeuVerified postcodeBarcelona, Spain
- Leeds General InfirmaryVerified postcodeLeeds, United Kingdom
Common questions
What is Duchenne Muscular Dystrophy (DMD)?
DMD is a genetic condition that causes muscles to become weak and waste away over time.
What are ENTR-601-44 and ENTR-601-45?
These are trial medicines being developed to treat Duchenne Muscular Dystrophy. They are not yet approved for general use.
Why is this study called an 'extension' study?
It's called an extension study because participants have already taken these medicines in a previous trial, and this study continues to follow them for a longer time.
Can I continue my regular DMD treatment if I join?
Yes, you are allowed to continue receiving your standard DMD treatments during the study, as long as your health allows.
What is a muscle biopsy?
A muscle biopsy is when a very small piece of muscle is carefully removed for examination under a microscope. It helps researchers see changes in the muscle.
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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