دراسة لتقييم RO7204239 لدى المشاركين المصابين بضمور العضلات الوجهي الكتفي العضدي
This research study is investigating a new medication called RO7204239 for adults who have Facioscapulohumeral Muscular Dystrophy (FSHD). The main goal is to understand how this medication works in the body, if it's safe to use, and whether it can help improve the condition. Participants will either receive the new medication or a placebo (a dummy treatment) to compare the effects. Researchers will be checking various aspects, including how the medicine moves through the body, its overall safety, and if it makes any difference to the symptoms of FSHD. This is a Phase 2 study, which means it's still in the earlier stages of testing.
نظرة سريعة
ما هو موضوع هذه الدراسة؟
This study is an important step in understanding and potentially treating Facioscapulohumeral Muscular Dystrophy, often called FSHD. FSHD is a genetic condition that causes muscles to weaken over time. Researchers are exploring a new medication, known as RO7204239, which is a type of antibody designed to target a specific protein in the body. This protein, called myostatin, can sometimes stop muscles from growing.
The main aim of this study is to learn more about how RO7204239 affects people with FSHD. This includes looking at how the body handles the medication, if it's safe and well-tolerated, and whether it has any positive effects on the condition itself. Participants will be carefully monitored throughout the study to gather this information.
This is a 'Phase 2' study, which means it's an important stage for checking the medication's safety and effectiveness in a smaller group of people. If the results are promising, it could lead to larger studies in the future. The information gained from this research will help scientists understand if RO7204239 could be a helpful treatment option for people living with FSHD.
النقاط الرئيسية
- This study is testing a new medication (RO7204239) for FSHD.
- It aims to see if the medication is safe, well-tolerated, and effective.
- You might receive the new medication or a placebo.
- Participation involves regular clinic visits for checks, tests, and medication.
- You must be an adult with confirmed FSHD, able to walk unassisted.
- يمكنك الانسحاب من الدراسة في أي وقت.
من قد يكون مؤهلاً؟
To join this study, you must be an adult between 18 and 65 years old and have a confirmed diagnosis of FSHD (either type 1 or type 2), with symptoms that match the condition. You need to be able to walk without help and your FSHD should be within a certain range of severity. It's also important that you agree to keep your physiotherapy, occupational therapy, and other exercise routines the same throughout the study.
There are some reasons why you might not be able to join. For example, if you are pregnant, breastfeeding, or planning to become pregnant within 17 months after the study ends. You also can't have been treated with similar myostatin-targeting medications before, or with any other experimental medications in the last 90 days (or longer, depending on the medication). If you have certain medical conditions like serious heart problems, allergies to the study drug, or some types of cancer, you won't be able to take part.
Additionally, if you have any conditions that would make MRI scans unsafe, or issues with your skin where injections might be given, you wouldn't be eligible. Recent injuries or surgeries that could affect how your movement is assessed would also prevent you from joining, as would any planned surgery that might impact your motor function during the study.
هل يمكن أن تناسبك هذه الدراسة؟
أجب عن هذه الأسئلة السريعة لترى ما إذا كنت مؤهلاً. هذا دليل فقط - الفريق البحثي هو الذي يتخذ القرار النهائي.
- هل عمرك بين 18 و 65 سنة؟
- Do you have a confirmed diagnosis of FSHD?
- Are you able to walk without help?
- Are you currently pregnant, breastfeeding, or planning to become pregnant?
- Do you have any serious heart conditions or severe allergies?
- Have you been treated with similar experimental drugs recently?
ماذا يتضمن المشاركة؟
If you decide to take part in this study, you will either receive the new medication RO7204239 or a placebo (a treatment that looks like the medication but has no active ingredients). This is decided randomly, meaning you won't know which one you're getting, and neither will the study team. You will have regular visits to the study clinic for check-ups, assessments, and to receive your medication.
During these visits, the study team will perform various tests. These include physical examinations, blood tests, assessments of your muscle strength and movement, and potentially MRI scans. They will also ask you about any side effects you might be experiencing. The medication will likely be given as an injection under the skin. You'll need to continue your usual physiotherapy and exercise routines, but it's important not to change the frequency or intensity while you're in the study.
The total duration of your participation in the study, including follow-up appointments after your last dose of medication, will be explained by the study team. It's a commitment to regular visits and assessments over an extended period.
المخاطر والفوائد المحتملة
المواقع (9)
- University of Irvine Medical Center (UCIMC)الرمز البريدي المؤكدأورانج، الولايات المتحدة
- Regents of the University of Coloradoالرمز البريدي المؤكدأورورا، الولايات المتحدة
- مركز جامعة كانساس الطبيالرمز البريدي المؤكدفيرواي، الولايات المتحدة
- معهد كينيدي كريجرالرمز البريدي المؤكدبالتيمور، الولايات المتحدة
- Rigshospitaletالرمز البريدي المؤكدKøbenhavn Ø, Denmark
- Policlinico Universitario Agostino Gemelliالرمز البريدي المؤكدروما، إيطاليا
- Asst Grande Ospedale Metropolitano Niguardaالرمز البريدي المؤكدميلانو، إيطاليا
- National Hospital for Neurology and Neurosurgery,الرمز البريدي المؤكدلندن، المملكة المتحدة
- مستشفى رويال فيكتورياالرمز البريدي المؤكدنيوكاسل أبون تاين، المملكة المتحدة
أسئلة شائعة
What is FSHD?
FSHD stands for Facioscapulohumeral Muscular Dystrophy, a genetic condition that causes muscles to gradually weaken, often starting in the face, shoulders, and upper arms.
ما هو الدواء الوهمي؟
A placebo is a 'dummy' treatment that looks exactly like the study medication but contains no active ingredients. It helps researchers understand if the new medication is truly having an effect, or if improvements are due to other factors.
What is a 'humanized monoclonal antibody'?
It's a type of medication that is specially designed to target specific things in your body. In this study, it's designed to interact with a protein called myostatin, which is involved in muscle growth.
Will I know if I'm getting the actual medication or the placebo?
No, this is a 'blinded' study, meaning neither you nor the study team will know whether you are receiving RO7204239 or the placebo. This helps ensure the study results are as accurate as possible.
كم من الوقت سأكون في الدراسة؟
The full length of your participation, including any follow-up visits after your last dose, will be explained during the consent process. It's an extended commitment that will involve various visits over time.
كيفية معرفة المزيد
تحدث دائمًا إلى طبيبك العام أو الأخصائي قبل اتخاذ قرار بالمشاركة في دراسة.
نقاش
مناقشة المجتمع
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