Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9
This study is an ongoing project designed to check the long-term safety and how well a medicine called BBP-418 (ribitol) works for people living with a muscle condition known as Limb-Girdle Muscular Dystrophy Type 2I, sometimes called LGMD2I/R9. Participants in this study will have already taken part in a previous study of the same medicine. The BBP-418 medicine is taken by mouth, and researchers will carefully watch for any side effects and how people's muscle function changes over time. The study will last for up to three years, with a final check-up about a month after the last dose to make sure everyone is doing well. This helps doctors understand more about this potential treatment.
At a glance
What is this study about?
Imagine you've been taking part in a study for a new medicine for your muscle condition, LGMD2I. This new study is designed for people like you who have already been in that first study. It's called an "open-label extension" study, which simply means everyone taking part will know they are receiving the study medicine, BBP-418 (which is also known as ribitol). The main goal is to see how safe the medicine is over a longer period and whether it continues to help with your condition.
Doctors want to understand if taking BBP-418 for a longer time leads to any unexpected side effects. They will also be looking closely at whether the medicine keeps helping with muscle strength and movement, which are important for people with LGMD2I. By continuing to gather information from people who have already started the treatment, researchers can build a more complete picture of its long-term effects.
This kind of study is really important because it helps scientists learn how a new medicine performs over many months or even years, not just weeks or months. This long-term information is crucial for deciding if a medicine is safe and effective enough to be considered for wider use in the future. Your continued participation helps researchers get closer to finding better ways to manage LGMD2I.
Key takeaways
- This study is a continuation for people already taking BBP-418 (ribitol) for LGMD2I.
- It aims to find out the long-term safety and effectiveness of the medicine.
- Participants will take the medicine by mouth and have regular health checks.
- The study could last up to three years, with a final follow-up after the last dose.
- You must have successfully completed a previous study (MLB-01-005) to be eligible.
Who may be eligible?
To be able to join this study, you must have already finished a previous study called MLB-01-005, where you were taking the study medicine right up until your last visit, usually at 36 months or a similar final check-up. You (or your parent/guardian if you're under 18) need to fully understand what the study involves and agree to take part by signing a consent form.
You also need to be willing and able to attend all the scheduled appointments and follow the study's instructions carefully. If you are a woman who could become pregnant, or a man who is not sterile, you must agree to use an approved method of birth control from the time you agree to join the study until about 30 days after your last dose of the medicine.
However, you won't be able to join if you have any other significant health problems that your doctor thinks might make it unsafe for you to take part, or if they might affect the study results. This includes serious heart, kidney, lung, liver, or hormone conditions not connected to your LGMD2I. Also, if you are pregnant, breastfeeding, or planning to get pregnant during the study period (plus 30 days after your last dose), you cannot participate. Finally, if you are currently having very strong thoughts about harming yourself, you would also not be able to join.
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.
- Did I finish the previous study (MLB-01-005) and take the study medicine right to the end?
- Do I understand what this study involves and am I happy to give my consent to take part?
- Am I willing and able to attend all the study appointments?
- If I am a woman who could get pregnant, or a man who is not sterile, am I willing to use approved birth control during the study and for 30 days afterwards?
- Do I have any other major health conditions that might make taking part unsafe?
- Am I pregnant or breastfeeding, or planning to be during the study period?
What does participation involve?
If you decide to take part in this study, you will continue to receive the BBP-418 (ribitol) medicine, taken by mouth, following a specific plan set out by the study doctors. You'll have regular appointments where the study team will carefully check your health. They'll monitor for any side effects, take blood samples for lab tests, and conduct other safety checks.
To see how well the medicine is working, you'll also have tests that measure your muscle function and strength. These might include various physical tasks or measurements relevant to your LGMD2I. These assessments help doctors understand if your condition is improving, staying the same, or changing in any way.
You will be involved in the study for up to 36 months (three years). After your last dose of the study medicine, there will be one final safety check-up about 30 days later to make sure you're doing well. Throughout this time, you will have regular contact with the study team.
Potential risks and benefits
Locations (20)
- Arkansas Children's HospitalVerified postcodeLittle Rock, United States
- University of California Irvine Medical CenterVerified postcodeOrange, United States
- University of Colorado Hospital (UCH) - Anschutz Medical CampusVerified postcodeAurora, United States
- University of Florida Health Center for Pediatric Neuromuscular and Rare DiseasesVerified postcodeGainesville, United States
- University of Iowa Stead Family Children's HospitalVerified postcodeIowa City, United States
- University of Kansas Medical Center (KUMC)Verified postcodeKansas City, United States
- Kennedy Krieger InstituteVerified postcodeBaltimore, United States
- University of Minnesota Medical SchoolVerified postcodeMinneapolis, United States
- Washington University School of MedicineVerified postcodeSt Louis, United States
- The Children's Hospital of PhiladelphiaVerified postcodePhiladelphia, United States
- University of PennsylvaniaVerified postcodePhiladelphia, United States
- Children's Hospital of the Kings DaughtersVerified postcodeNorfolk, United States
Common questions
What is BBP-418 (ribitol)?
BBP-418, also known as ribitol, is the medicine being tested in this study for people with LGMD2I.
Why is this study only for people who were in another study?
This study is a continuation for people who already started taking BBP-418 in an earlier study, so researchers can see its long-term effects.
How long will I be in this study?
You could be in this study for up to three years (36 months), with one last check-up about a month after your final dose.
Will I know if I'm getting the active medicine?
Yes, this is an 'open-label' study, meaning everyone taking part will know they are receiving the BBP-418 medicine.
What if I have other health problems?
Certain other health conditions might prevent you from joining, as they could affect your safety or the study results. Your doctor can tell you more.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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