An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This study is an ongoing look at a medicine called BMN 351 for Duchenne Muscular Dystrophy (DMD). It's designed for boys and young men, aged 4 and older, who have already been taking BMN 351 in a previous study. The main goals are to check the medicine's long-term safety and how well people tolerate it, meaning if they have side effects over time. Researchers will also be monitoring if BMN 351 continues to help improve or maintain muscle function and physical abilities. Participants will receive weekly infusions of BMN 351 and continue to be monitored by the study team, building on the information gathered from the earlier study.
At a glance
What is this study about?
This research study is about a medicine codenamed BMN 351 for Duchenne Muscular Dystrophy, a condition that causes muscles to weaken over time. This particular study is an extension, meaning it's a continuation for people who have already been taking BMN 351 in an earlier study. The main aim is to see how safe the medicine is over a longer period and whether people can keep taking it without too many problems. It also aims to understand how well BMN 351 helps with physical abilities in the long run.
The study involves giving BMN 351 as a drip, or infusion, once a week. Doctors and nurses will regularly check on the participants to see if the medicine is causing any unwanted effects and how their physical strength and movement are changing over time. By continuing to follow participants, the study hopes to gather more important information about BMN 351, which could eventually help decide if it can become a widely available treatment for DMD.
Because Duchenne Muscular Dystrophy is a serious condition, finding treatments that are safe and effective over many years is very important. This type of long-term study helps researchers understand the full picture of how a new medicine works and whether it can make a real difference in the lives of those living with the condition.
Key takeaways
- This study is a long-term continuation for those already on BMN 351.
- It aims to check the safety and how well the medicine works over time.
- Participants will receive weekly infusions of BMN 351.
- Regular health check-ups and physical movement tests will be part of the study.
- It's for males aged 4 and up with Duchenne Muscular Dystrophy.
Who may be eligible?
To be part of this study, you must be a male between 4 years old and any age, who has already completed an earlier study (called 351-201) with BMN 351 and hasn't stopped the treatment early. You also need to be taking steroid medication for DMD at a steady dose and be able to stick to the study's visit schedule.
There are some reasons why you might not be able to join. These include having a known blood clotting problem or currently taking certain other medications. If you have been treated with other similar DMD medicines or gene therapy, you would not be able to join this study. Specifically, other 'exon skipping' therapies within the last 12 weeks, or any gene therapy for DMD at any time, would exclude you. Also, certain blood thinners or medicines that suppress your immune system would make you ineligible.
If you are a sexually mature male, you would need to agree to use contraception throughout the study and for a period after your last dose. For participants under 16, a parent or guardian would need to give permission, and the participant themselves would also need to agree if required by local rules.
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.
- Have I completed the previous BMN 351 study?
- Am I currently taking oral steroid medication for DMD?
- Am I willing and able to attend all study visits?
- Do I have any known blood clotting disorders?
- Am I taking any other specific DMD medicines, blood thinners, or immune-suppressing drugs?
What does participation involve?
Taking part in this study means you'll continue to receive BMN 351 as a drip (infusion) once a week. These infusions will happen at a study clinic. You will also have regular check-ups and assessments by the study team to monitor your health, how well you're tolerating the medicine, and your physical abilities. These assessments might include physical examinations, blood tests, and tests to measure your muscle strength and movement.
The exact duration of the study is not specified, but as an "open-label extension," it means it will continue for a longer period, building on the earlier study. Your first visit for this extension study will be the same time as your very last visit for the previous study. You'll need to attend all scheduled appointments and follow the instructions given by the study team. If you are a male able to have children, you must use contraception during the study and for 90 days after your last dose of BMN 351.
Potential risks and benefits
Locations (7)
- Fondazione Serena ETS - Centro Clinico NeMO MilanoVerified postcodeMilan, Italy
- UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro CuoreVerified postcodeRome, Italy
- Leids Universitair Medisch CentrumVerified postcodeLeiden, Netherlands
- Hospital Sant Joan de DeuVerified postcodeBarcelona, Spain
- Hospital Viamed Santa Angela De la CruzVerified postcodeSeville, Spain
- Yeditepe University Kosuyolu HospitalVerified postcodeIstanbul, Turkey (Türkiye)
- Great Ormond Street Hospital NHS Foundation TrustVerified postcodeLondon, 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 is BMN 351?
BMN 351 is a new medicine being studied to see if it can help treat Duchenne Muscular Dystrophy.
Who can join this study?
This study is for males aged 4 and older who have already taken BMN 351 in a previous study.
How often will I receive the medicine?
You will receive BMN 351 as an infusion (drip) once a week at the study clinic.
Can I stop participating in the study if I want to?
Yes, you can choose to leave the study at any time without it affecting your medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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