Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome
This study is an 'open-label extension' which means everyone taking part will receive the study medicine, DCCR. It's designed to check how safe DCCR is over a longer period in people with Prader-Willi syndrome. DCCR is a medicine in tablet form. This study is only for individuals who have previously participated in a specific DCCR study (Study C602) and met certain conditions after that study. The main goal is to gather more information about DCCR's long-term safety, helping doctors understand its effects better for patients with Prader-Willi syndrome. This type of study helps make sure new treatments are as safe as possible.
Em resumo
Sobre o que é este estudo?
This study is a continuation for people who have already taken part in a specific study looking at a medicine called DCCR. DCCR is a tablet given to individuals with Prader-Willi syndrome. The main purpose of this new study is to closely watch the long-term safety of DCCR. When new medicines are developed, doctors need to understand not only if they work but also if they are safe to use over many months or even years.
This particular study is called an 'open-label' study. This means that unlike some studies where people might get a dummy treatment (placebo), everyone in this study will receive the DCCR medicine. This allows researchers to gather more information on how the body reacts to DCCR over an extended period.
By taking part, you would be helping medical science gather vital information. This information helps doctors and researchers understand DCCR better, which is crucial for the potential future treatment of Prader-Willi syndrome. Your involvement helps improve care for people living with this condition.
Pontos principais
- This study is for people with Prader-Willi syndrome who have already been in a DCCR study.
- It aims to understand the long-term safety of the DCCR medicine.
- Everyone in the study will receive the active DCCR medicine.
- Participation involves regular health checks and taking DCCR tablets.
- Your involvement helps medical research for Prader-Willi syndrome.
- Você pode deixar o estudo a qualquer momento.
Quem pode ser elegível?
To join this study, a person must have already taken part in a previous DCCR study, specifically 'Study C602'. This includes people who completed the main part of that study, or those who left early but a certain amount of time has passed since then. It also includes people who were in the extended part of Study C602 but chose not to join the next stage, as long as enough time has gone by.
There are also some reasons why someone could not join. For example, if you are a female and are pregnant, breastfeeding, or planning to become pregnant or breastfeed during the study or shortly after, you would not be able to take part. Also, if you are currently participating in another study that involves a new drug or treatment, you would not be eligible for this study. All participants or their parents/guardians will need to give their informed consent, meaning they understand and agree to 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 I or my child taken part in Study C602 before?
- Have I completed the main part of C602, or has enough time passed since stopping it?
- Am I (or is the female intended participant) currently pregnant, breastfeeding, or planning to be?
- Am I (or is the participant) currently in another research study with a new medicine or device?
O que envolve a participação?
If you join this study, it would involve continuing to take the DCCR medicine as prescribed. The study team will regularly check your health to monitor the long-term safety of the medicine. This will include visits to the clinic where doctors and nurses will carry out various checks and assessments, though the detailed schedule isn't provided here. You'll be taking the DCCR tablets, and the team will keep a close eye on any effects, both positive and negative. The total duration of your participation isn't specified in detail but is focused on long-term safety, meaning it could last for an extended period.
Riscos e benefícios potenciais
Localizações (22)
- UC IrvineCódigo postal verificadoOrange, Estados Unidos
- Universidade de StanfordCódigo postal verificadoPalo Alto, Estados Unidos
- Rady Children's Hospital of San DiegoCódigo postal verificadoSan Diego, Estados Unidos
- U of Florida GainesvilleCódigo postal verificadoGainesville, Estados Unidos
- Emory Children's CenterCódigo postal verificadoAtlanta, Estados Unidos
- Indiana University School of MedicineCódigo postal verificadoIndianápolis, Estados Unidos
- National Institutes of HealthCódigo postal verificadoBethesda, Estados Unidos
- Hospital Infantil de BostonCódigo postal verificadoBoston, Estados Unidos
- Instituto de Pesquisa Clínica SparrowCódigo postal verificadoLansing, Estados Unidos
- Children's Hospital and Clinic MinnesotaCódigo postal verificadoSaint Paul, United States
- St. Joseph's University Medical CenterCódigo postal verificadoPaterson, United States
- NYU Winthrop HospitalCódigo postal verificadoMineola, United States
Perguntas frequentes
O que é a síndrome de Prader-Willi?
Prader-Willi syndrome is a complex genetic condition that affects many parts of the body. Key features include a constant feeling of hunger, which can lead to life-threatening obesity, as well as problems with learning and behaviour.
What is DCCR?
DCCR is the study medicine being looked at. It comes as tablets and is being investigated for use in people with Prader-Willi syndrome.
O que significa 'open-label'?
Open-label means that everyone taking part in the study will know they are receiving the study medicine, DCCR. No one will receive a dummy treatment (placebo).
Por que este estudo é apenas para pessoas que participaram de um estudo anterior?
This study is specifically designed to follow up on people who have already taken DCCR in a previous study (Study C602). This helps researchers understand the long-term effects on people who have already started the treatment.
What does 'long-term safety' mean?
It means the study will observe participants over an extended period to see how safe the medicine is when used over many months, and to identify any effects that might only appear after longer use.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
Discussão
Discussão da comunidade
Desenvolvido pelo nosso fórum em community.patient.info. Por favor, seja respeitoso — isto não é um conselho médico.