تمديد طويل الأمد لـ GTX-102 في متلازمة أنجلمان
This research is a continuation study for a treatment called GTX-102, designed for individuals with Angelman Syndrome. If you've already taken part in a previous study involving GTX-102, you might be able to join this one. The main aim is to carefully monitor the safety of GTX-102 over a longer period. Researchers will also look at how well the treatment works in the long run. This is a vital step in understanding how GTX-102 could help people with Angelman Syndrome in a lasting way. It's important that participants meet certain criteria, including having finished an earlier study and agreeing to specific health and safety guidelines throughout the trial.
نظرة سريعة
ما هو موضوع هذه الدراسة؟
This study, called a 'long-term extension', is a follow-on from an earlier research project looking at a potential treatment called GTX-102 for Angelman Syndrome. Angelman Syndrome is a rare genetic condition that affects a person's development, movement, and speech. If you or someone you care for has already been involved in a study using GTX-102, this new study offers the chance to continue with the treatment and help us understand more about it over time.
The main goal of this study is to see how safe GTX-102 is when used for a longer period. All medications can have side effects, and researchers need to carefully track these to make sure the treatment is as safe as possible. They will also be looking at whether the treatment continues to have positive effects on people with Angelman Syndrome over time. This information is really important because it helps doctors and scientists understand the full picture of the treatment and how it might help people in the future.
By taking part, you'd be contributing valuable information that could help others living with Angelman Syndrome. The study will involve regular check-ups and assessments to monitor your health and how the treatment is working. It's a careful and ongoing process designed to gather the best possible information about GTX-102.
النقاط الرئيسية
- This study is for people with Angelman Syndrome who have already received GTX-102 in a previous trial.
- The main goal is to check the long-term safety and how well GTX-102 works over time.
- Participation involves ongoing treatment and regular check-ups with the study team.
- Strict contraception rules apply for both male and female participants.
- You cannot join if you had a serious side effect from GTX-102 in the previous study or have other health issues that make participation unsafe.
- Your contribution could help improve understanding of Angelman Syndrome treatment.
من قد يكون مؤهلاً؟
To be able to join this study, you must have already taken part in a previous study involving GTX-102 and completed your final visit for that study. If you had any health problems during the previous study, a doctor on the research team would need to confirm that it's safe for you to continue.
There are also some important rules around pregnancy and contraception. If you are female and could become pregnant and are sexually active, you must use effective contraception or choose not to have sex for the entire study and for six months after your last dose of the study medicine. If you are male, you must agree to either not have sex or use reliable contraception for the study period and for three months after your last dose.
You wouldn't be able to join if you had to stop the previous GTX-102 study because of a bad side effect that the doctors thought was related to the treatment, and they believe the risks are too high. Also, if you have any other serious health conditions that could make taking part unsafe or interfere with the study, you might not be able to join. Finally, if you are allergic to GTX-102 or anything in it, or if you are pregnant, breastfeeding, or planning to become pregnant, you cannot take part.
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.
- Have you already completed an earlier study with GTX-102?
- هل تستطيع حضور المواعيد الدراسية المنتظمة؟
- If you are a female who could become pregnant, are you able to use reliable contraception for the full study and for six months after?
- If you are a male, are you able to use reliable contraception or abstain from sex for the full study and for three months after?
- Do you have any known allergies to the study medicine, GTX-102, or its ingredients?
- Are you currently pregnant or breastfeeding, or planning to become so?
ماذا يتضمن المشاركة؟
If you decide to join this study, you'll be taking GTX-102 for a longer period. You'll need to attend regular appointments at the study clinic, where the research team will carefully check your health and how you're responding to the treatment. These visits will involve various tests and assessments, similar to what you might have experienced in the earlier study. The doctors and nurses will monitor you closely for any side effects and see how the treatment is affecting your Angelman Syndrome.
The study involves ongoing monitoring and might include blood tests, physical examinations, and other checks to make sure the treatment is safe and working as expected. The total duration of your participation will depend on the study's plan and will be discussed with you in detail by the study team. You'll receive all necessary information and support throughout your involvement.
المخاطر والفوائد المحتملة
المواقع (23)
- جامعة كاليفورنيا، لوس أنجلوس (UCLA)الرمز البريدي المؤكدلوس أنجلوس، الولايات المتحدة
- University of California, San Diego - Rady Children's Hospitalالرمز البريدي المؤكدسان دييغو، الولايات المتحدة
- البحث في الأمراض النادرة، ذ.م.مالرمز البريدي المؤكدأتلانتا، الولايات المتحدة
- مركز جامعة روش الطبيالرمز البريدي المؤكدشيكاغو، الولايات المتحدة
- مستشفى بوسطن للأطفالالرمز البريدي المؤكدبوسطن، الولايات المتحدة
- Weill Cornell Medicineالرمز البريدي المؤكدنيويورك، الولايات المتحدة
- مستشفى كوينزلاند للأطفالالرمز البريدي المؤكدجنوب بريزبين، أستراليا
- مستشفى الأطفال الملكيالرمز البريدي المؤكدملبورن، أستراليا
- MAGIC Clinic Ltdالرمز البريدي المؤكدكالجاري، كندا
- مستشفى الأطفال في كولومبيا البريطانيةالرمز البريدي المؤكدفانكوفر، كندا
- Childrens Hospital London Health Sciences Centreالرمز البريدي المؤكدلندن، كندا
- Childrens Hospital of Eastern Ontarioالرمز البريدي المؤكدأوتاوا، كندا
أسئلة شائعة
What is Angelman Syndrome?
Angelman Syndrome is a rare genetic condition that mainly affects the nervous system, leading to developmental delays, movement issues, and problems with speech.
What is GTX-102?
GTX-102 is a potential treatment being investigated for Angelman Syndrome. This study is looking at its long-term safety and effectiveness.
Why is this study called an 'extension'?
It's called an extension because it's a continuation for people who have already taken part in an earlier study with GTX-102, allowing researchers to gather more information over time.
Will I get the actual treatment or a placebo?
Because this is an extension study for people who have already been on GTX-102, you would continue to receive the active study medicine, GTX-102.
كم من الوقت سأحتاج للبقاء في الدراسة؟
The total length of time you'd be involved in the study will be explained by the study team, as it varies depending on the research plan.
كيفية معرفة المزيد
تحدث دائمًا إلى طبيبك العام أو الأخصائي قبل اتخاذ قرار بالمشاركة في دراسة.
نقاش
مناقشة المجتمع
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