RekryteringTerapeutisk utforskande (Fas II)Interventionell

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.

Överblick

Status

Rekrytering

Fas

Terapeutisk utforskande (Fas II)

Sponsor

Edgewise Therapeutics Inc.

Inskrivningsmål

Starta

12 okt 2022

McArdle Disease, Limb-Girdle Muscular Dystrophy, Becker Muscular Dystrophy

Vad handlar denna studie om?

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.

Viktiga punkter

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.

Vem kan vara berättigad?

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.

Kan denna studie passa dig?

Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.

Är du 18 år eller äldre?
Do you have a diagnosis of Becker muscular dystrophy, McArdle disease, or limb-girdle muscular dystrophy?
Kan du delta i regelbundna klinikbesök?
Are you willing to take the study medicine and have various tests?

Svara på varje fråga för att se ditt resultat.

Vad innebär deltagande?

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.

Potentiella risker och fördelar

Taking part in any research study has potential benefits and risks. A potential benefit of this study is that the new medicine, EDG-5506, might help improve your muscle condition or reduce muscle damage, though this is not guaranteed as it is still being tested. There's also the benefit of contributing to medical knowledge that could help others in the future. Potential risks include experiencing side effects from the medicine, which could be mild or, in rare cases, more serious. There's also the chance the medicine might not help you. You are free to ask questions at any time and can leave the study at any point without giving a reason, and this will not affect your usual medical care.

Platser (1)

Vissa platsangivelser är ungefärliga. Vi arbetar på att förbättra detta — vänligen kontrollera med testteamet innan du reser.

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Obekräftad
Danmark

Vanliga frågor

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.

Hur länge behöver jag vara med i studien?

The study could last for about a year (52 weeks). There will be regular visits during this time.

Vad händer om jag mår dåligt under studien?

The study team will monitor you closely. If you feel unwell, you should tell them immediately, and they can advise you.

Hur du får reda på mer

Officiell rättegångsprotokoll

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