Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)
This research study is investigating a new medication, BBP-418 (ribitol), for a specific type of muscle weakness called LGMD2I. Many people with this condition experience progressive muscle weakening, especially in their shoulders, hips, and thighs. The study's main goal is to check how safe this new medication is over a long period and if it can effectively help manage the condition. Participants between 12 and 60 years old who have a confirmed diagnosis of LGMD2I are invited. We want to understand if BBP-418 can make a positive difference in the lives of those affected. This is a crucial step in developing new treatments for LGMD2I.
De un vistazo
¿De qué trata este estudio?
This study is focusing on a specific type of muscular dystrophy called Limb-Girdle Muscular Dystrophy type 2I, often shortened to LGMD2I. This condition causes muscles, particularly around the hips and shoulders, to gradually weaken. There isn't a cure for LGMD2I yet, so researchers are constantly looking for new ways to help people manage their symptoms and improve their quality of life.
Here, scientists are testing a new medication called BBP-418, also known as ribitol. They want to find out two main things: firstly, if BBP-418 is safe for people with LGMD2I to take over a longer period, and secondly, if it can effectively help with their muscle weakness. To do this, some participants will receive BBP-418, while others will receive a 'placebo', which looks like the medicine but contains no active ingredients. This is a standard way to ensure any improvements seen are genuinely due to the study medication.
This is a 'Phase 3' study, which means it's one of the larger, final stages of testing a new medication before it can potentially be made widely available. The results from this study will be very important in understanding if BBP-418 could become a future treatment option for LGMD2I. The study includes a wide age range of participants, from 12 to 60 years, because the researchers want to gather information from many people who live with this condition.
Puntos clave
- This study is for people with a specific muscle condition called LGMD2I.
- It's testing a new medication, BBP-418, to see if it's safe and helps improve symptoms.
- Participants will either receive the new medication or a 'dummy' pill (placebo).
- It's a long-term study with regular clinic visits and health checks.
- You can be aged 12 to 60 to take part (18 to 60 in Europe).
- Your decision to join or leave the study won't affect your regular medical care.
¿Quién puede ser elegible?
To join this study, people need to have a confirmed diagnosis of LGMD2I through genetic testing and show some signs of muscle weakness, either in their shoulders and hips or in their arms and legs. Participants must be between 12 and 60 years old (though in Europe, it's 18-60 years old). They also need to weigh more than 30 kg and be able to understand the study and agree to take part, with a parent or guardian's help if they are under 18.
There are also some reasons why someone might not be able to join. For example, if you have other serious health problems like heart, kidney, lung, liver, or hormone issues that aren't related to LGMD2I, or if your kidney function is below a certain level. If you've had major surgery like for scoliosis that might make it hard to do the study tests, or if the doctors think joining the study might not be safe for you, you also might not be eligible. Women who could have children and men who could father children must agree to use effective contraception during the study and for some time after.
¿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.
- Do I have a confirmed genetic diagnosis of LGMD2I with some current muscle weakness?
- Am I between 12 and 60 years old (18-60 if I live in Europe)?
- Do I weigh more than 30 kilograms?
- Am I generally healthy, without other serious health conditions not related to LGMD2I, or severe kidney problems?
- Am I willing and able to attend all study appointments and complete all required tests?
¿En qué consiste la participación?
If you decide to take part in this study, you will be given either the study medication (BBP-418) or a placebo (a dummy medicine) on a long-term basis. You'll have regular visits to the clinic where doctors and nurses will check your health, measure your muscle strength, and perform other tests to see how you're responding to the treatment. These tests might include blood and urine samples, and possibly some small muscle biopsies (where a tiny piece of muscle is taken for examination). You'll also have your vital signs (like blood pressure and heart rate) checked, and potentially heart recordings (ECG). These visits will be scheduled over a significant period. The study team will explain the full schedule of visits and procedures in detail, so you know exactly what to expect and how much time it might involve. You will also have follow-up checks after you finish taking the medication. The total duration of your participation will be explained by the study team.
Riesgos y beneficios potenciales
Locations (21)
- Arkansas Children's HospitalCódigo postal verificadoLittle Rock, Estados Unidos
- Universidad de California en IrvineCódigo postal verificadoIrvine, United States
- University of Colorado Anschutz Medical CampusCódigo postal verificadoAurora, Estados Unidos
- Universidad de FloridaCódigo postal verificadoGainesville, Estados Unidos
- University of IowaCódigo postal verificadoIowa City, Estados Unidos
- Centro Médico de la Universidad de KansasCódigo postal verificadoKansas City, Estados Unidos
- Kennedy Krieger InstituteCódigo postal verificadoBaltimore, Estados Unidos
- University of Minnesota, Twin CitiesCódigo postal verificadoMinneapolis, United States
- Washington University School of MedicineCódigo postal verificadoSan Luis, Estados Unidos
- Oregon Health & Science University (OHSU) - Neurology Clinic - South WaterfrontCódigo postal verificadoPortland, Estados Unidos
- The Children's Hospital of PhiladelphiaCódigo postal verificadoFiladelfia, Estados Unidos
- University of PennsylvaniaCódigo postal verificadoFiladelfia, Estados Unidos
Preguntas comunes
What is LGMD2I?
LGMD2I is a type of muscular dystrophy that causes weakness in muscles, especially around the hips and shoulders, and can affect other muscles too.
What is BBP-418?
BBP-418, also known as ribitol, is a new medication being tested to see if it can help people with LGMD2I.
Will I definitely get the active medication?
No, this is a 'blinded' study, meaning some participants will receive BBP-418 and others will receive a placebo, and neither you nor the doctors will know which one you are getting until the study ends.
¿Quién puede unirse al estudio?
Generally, people aged 12 to 60 with a confirmed diagnosis of LGMD2I and certain health requirements may be able to join, though there are specific criteria.
What does a 'Phase 3' study mean?
Phase 3 means this is a later stage of research, where the medication is tested on more people to confirm its safety and effectiveness before it can potentially be approved for general use.
Cómo obtener más información
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
Discusión
Discusión comunitaria
Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.