An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of BBP-418 (Ribitol) in Participants with Limb-Girdle Muscular Dystrophy 2I/R9 (LGMD2I/R9)
Limb-girdle muscular dystrophy type 2I (LGMD2I) is a rare genetic condition that causes muscles in the hips, thighs, and shoulders to weaken over time. This trial tests a medicine called Ribitol (also known as BBP-418) to find out if it is safe and works well when taken over a longer period. Because this is an 'open-label extension' study, everyone who takes part receives the real study medicine—there is no dummy drug (placebo). Researchers will track participants' everyday movement, walking speed, arm strength, and lung health. They will also do regular blood tests to check muscle health. The main goal is to find out whether Ribitol can safely slow down muscle weakness and help people live more independently.
De un vistazo
¿De qué trata este estudio?
Limb-girdle muscular dystrophy type 2I (or LGMD2I) is a rare condition that gradually weakens the muscles around the hips and shoulders. Over time, everyday tasks like climbing stairs, walking, lifting objects, or even breathing easily can become much harder. Right now, there are very few treatments that directly target the root cause of this muscle damage.
This study is testing an investigational medicine called Ribitol (BBP-418). In people with LGMD2I, the body has trouble making a specific sugar-protein shield that protects muscle cells from breaking down. Ribitol is designed to help the body rebuild this protective shield, which doctors hope will protect muscle fibres and prevent further weakness.
This trial is an 'extension' study, which means it follows people over a longer period to see how the medicine works in real life over time. By looking at long-term safety and changes in movement and breathing, doctors hope to gather the evidence needed to see if Ribitol could become a standard treatment for LGMD2I in the future.
Puntos clave
- Tests whether the experimental drug Ribitol is safe and effective over the long term for LGMD2I.
- This is an open-label study, meaning every participant receives the real medicine.
- Open to adults aged 18 and older, including people who can walk and those who use a wheelchair.
- Tracks muscle strength, walking ability, arm function, and breathing health over time.
- Participation is entirely voluntary and you can stop at any time.
¿Quién puede ser elegible?
This trial is for adults aged 18 and older who have been diagnosed with limb-girdle muscular dystrophy type 2I (also called LGMD2I or LGMD R9). Both men and women can take part.
Because this is an extension study, it is generally designed for people who have already completed an earlier phase of the Ribitol study programme. Participants may be able to walk, or they may use a wheelchair; both groups are included so researchers can understand how the treatment affects different stages of the condition.
Your study doctor will check whether your current health and any other medicines you take make it safe for you to take part. You will not be able to join if you have other serious medical issues that could make the trial unsafe for you.
¿Podría este estudio ser adecuado para ti?
Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.
- I am 18 years of age or older.
- I have a confirmed diagnosis of limb-girdle muscular dystrophy type 2I (LGMD2I/R9).
- I have taken part in a previous trial stage for Ribitol, or have been referred by my specialist.
- I am able and willing to attend regular hospital appointments for check-ups and tests.
¿En qué consiste la participación?
If you take part, you will be given Ribitol to take as directed by the clinical team. Because it is an open-label study, you will definitely receive the active medicine rather than a dummy pill.
You will need to attend regular clinic appointments at a hospital or specialist research centre. During these visits, doctors will measure how well your muscles and lungs are working. Assessments include timed walking tests, simple checks of how well you can move your arms and do daily activities, and a breathing test where you blow into a tube while sitting down. The team will also take routine blood tests to look for signs of muscle stress and check on your overall well-being. The study team will explain the exact visit schedule and how long your participation will last before you agree to join.
Riesgos y beneficios potenciales
Ubicaciones (5)
- —No verificadoAlemania
- —No verificadoDinamarca
- —No verificadoPaíses Bajos
- —No verificadoItalia
- —No verificadoNoruega
Preguntas comunes
¿Qué significa 'etiqueta abierta'?
'Open-label' means both you and the research doctors know you are receiving the real study medicine. There is no placebo (dummy treatment) in this part of the study.
Can I join if I use a wheelchair?
Yes. The trial includes specific tests (like arm mobility checks) designed for people who cannot walk, as well as walking tests for those who can.
How is Ribitol taken?
Ribitol is an oral medicine that you swallow. The study team will give you clear instructions on how and when to take it at home.
Will taking part cost me anything?
No, the study medicine and all trial tests are provided free of charge. Most clinical trials also help cover travel expenses to and from the hospital.
¿Qué pasa si cambio de opinión?
You can leave the trial at any point, for any reason, without giving an explanation. Your normal medical care will continue as usual.
Cómo obtener más información
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
Discusión
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Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.