Long-term Extension of GTX-102 in Angelman Syndrome
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.
Em resumo
Sobre o que é este estudo?
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.
Pontos principais
- 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.
Quem pode ser elegível?
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.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- Have you already completed an earlier study with GTX-102?
- Are you able to attend regular study appointments?
- 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?
O que envolve a participação?
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.
Riscos e benefícios potenciais
Localizações (23)
- University of California, Los Angeles (UCLA)Código postal verificadoLos Angeles, Estados Unidos
- University of California, San Diego - Rady Children's HospitalCódigo postal verificadoSan Diego, Estados Unidos
- Rare Disease Research, LLCCódigo postal verificadoAtlanta, Estados Unidos
- Centro Médico da Universidade RushCódigo postal verificadoChicago, Estados Unidos
- Hospital Infantil de BostonCódigo postal verificadoBoston, Estados Unidos
- Weill Cornell MedicineCódigo postal verificadoNova Iorque, Estados Unidos
- Hospital Infantil de QueenslandCódigo postal verificadoSouth Brisbane, Australia
- The Royal Children's HospitalCódigo postal verificadoMelbourne, Austrália
- MAGIC Clinic LtdCódigo postal verificadoCalgary, Canadá
- British Columbia Children's HospitalCódigo postal verificadoVancouver, Canadá
- Childrens Hospital London Health Sciences CentreCódigo postal verificadoLondres, Canadá
- Childrens Hospital of Eastern OntarioCódigo postal verificadoOttawa, Canadá
Perguntas frequentes
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.
How long will I need to be in the study?
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.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
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