A Phase 2 Randomized, Double-blind, Placebo-controlled Study of the Effect of EDG-5506 on Biomarker Response to Exercise in Adults with Becker Muscular Dystrophy, McArdle Disease, or Limb-Girdle Muscular Dystrophy
This study is looking at a new medicine called EDG-5506. It's for adults who have Becker muscular dystrophy, McArdle disease, or limb-girdle muscular dystrophy. These conditions can cause muscle weakness and pain. The main aim is to see how safe the new medicine is and if it causes any side effects compared to a 'dummy' medicine (placebo). We'll also be checking if it helps to lower a chemical in the blood called creatine kinase (CK), which can be a sign of muscle damage. Taking part would involve regular check-ups and tests over several months to understand how the medicine affects your body and your muscles. We hope this research will lead to better treatments for these muscle conditions.
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¿De qué trata este estudio?
This research study is focused on a new medicine called EDG-5506. It's being investigated for adults who live with specific muscle conditions: Becker muscular dystrophy, McArdle disease, or limb-girdle muscular dystrophy. These conditions can lead to muscle weakness and other problems over time. The study wants to find out if EDG-5506 can help people with these conditions feel better and if it can reduce signs of muscle damage.
To do this, some people will receive the new medicine, while others will receive a 'dummy' medicine, called a placebo. This is a common way to test new medicines fairly, so we can see if any changes are truly due to the new medicine or something else. Neither you nor your study doctor will know whether you are getting the active medicine or the placebo, which helps make the results as accurate as possible.
The main things we'll be looking at are how safe the medicine is and if it causes any unwanted effects. We'll also be measuring a substance in your blood called creatine kinase (CK). High levels of CK can indicate that muscles are being damaged. We want to see if EDG-5506 can help lower these levels, which would be a good sign for muscle health.
Puntos clave
- This study is testing a new medicine for specific muscle diseases.
- It aims to see if the medicine is safe and reduces muscle damage.
- Some participants will get the medicine, others a dummy pill.
- Regular health checks and tests will be part of the study.
- The study lasts about a year and you can leave at any time.
- Your participation helps advance understanding of these conditions.
¿Quién puede ser elegible?
To be able to take part in this study, you need to be an adult, aged 18 or older. Both men and women can join.
The study is specifically for people who have been diagnosed with one of three muscle conditions: Becker muscular dystrophy, McArdle disease, or limb-girdle muscular dystrophy.
Before you can join, the study team will carry out a full check-up to make sure the study is suitable and safe for you. This will involve looking at your medical history and doing various tests.
¿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.
- ¿Tienes 18 años o más?
- Do you have a diagnosis of Becker muscular dystrophy, McArdle disease, or limb-girdle muscular dystrophy?
- Are you able to attend regular clinic appointments?
- Are you willing to take the study medicine and have various tests?
¿En qué consiste la participación?
If you decide to take part in this study, you would receive either the new medicine (EDG-5506) or a 'dummy' medicine (placebo) in tablet form. You would take this medicine regularly as instructed by the study team.
Throughout the study, you'd have several visits to the clinic. These visits would involve various tests to check your health and how the medicine is affecting you. These tests could include blood tests, urine tests, having your heart checked with an ECG (electrocardiogram) and an echocardiogram (a scan of your heart), and breathing tests (spirometry). We would also check your vital signs like blood pressure and pulse, and perform physical examinations.
These check-ups would happen at different times over the study period, which could last up to about a year (52 weeks). The study team will carefully explain the schedule of visits and what each test involves. You’ll be closely monitored for any changes in your health or any side effects.
Riesgos y beneficios potenciales
Ubicaciones (1)
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Preguntas comunes
What is a 'dummy' medicine (placebo)?
A dummy medicine looks just like the real medicine but contains no active ingredients. It helps us see if the new medicine truly works better than taking nothing.
What is creatine kinase (CK)?
CK is a substance found in your blood. High levels can sometimes mean your muscles are experiencing damage or stress.
Will I know if I'm getting the active medicine or the dummy one?
No, neither you nor your study doctor will know. This is called 'double-blind' and makes the results of the study more reliable.
How long will I need to be in the study?
The study could last for about a year (52 weeks). There will be regular visits during this time.
What happens if I feel unwell during the study?
The study team will monitor you closely. If you feel unwell, you should tell them immediately, and they can advise you.
Cómo obtener más información
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
¿Interesado en participar?
Discusión
Discusión comunitaria
Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.