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Extension Study of Infigratinib in Children With Achondroplasia (ACH)

This study is a continuation of earlier research into a medicine called Infigratinib for children and teenagers aged 3 to 18 who have achondroplasia. Achondroplasia is the most common type of dwarfism, affecting how bones grow. The main goals of this study are to continue watching the long-term safety and effectiveness of Infigratinib. Researchers also want to see how the medicine might help with growth and improve the overall quality of life for those taking it. Some children in this study have taken Infigratinib before, while others might be new to the treatment. This is a "Phase 2" study, meaning it's still in the earlier stages of testing to find the best dose and understand the medicine better.

Em resumo

Status
Inscrição por convite
Fase
FASE2
Patrocinador
QED Therapeutics, a BridgeBio company
Meta de inscrição
325
Iniciar
06 Dec 2021
Conclusão estimada
01 Feb 2032

Sobre o que é este estudo?

This study is looking at a new medicine called Infigratinib for children and teenagers who have achondroplasia. Achondroplasia is a condition that affects how bones grow, leading to shorter limbs and other features. The medicine aims to help bone growth in young people with this condition.

Since this is an "extension study," it means many of the children involved have already taken Infigratinib in a previous study. This allows doctors to observe the long-term effects of the medicine. The study will carefully check if the medicine is safe to use over time and how well it continues to work. They will also look at how it might improve the daily lives and overall well-being of the children, which is often called "quality of life."

Some children in this study might be taking Infigratinib for the first time. For these new participants, the study will first make sure they've had their growth carefully measured for at least six months in a related study. This helps the doctors understand their natural growth patterns before starting the medicine. This is a Phase 2 study, which means it's focused on finding the right dose and further understanding the medicine's effects before it can move into larger studies.

Pontos principais

  • The study is for children and teenagers (3-18 years old) with achondroplasia.
  • It's testing a medicine called Infigratinib for its long-term safety and effectiveness on growth.
  • Some participants have already used the medicine; others may be new to it.
  • Quality of life will also be assessed.
  • Regular clinic visits and taking tablets are involved.
  • Você pode se retirar do estudo a qualquer momento.

Quem pode ser elegível?

This study is for children and teenagers with achondroplasia, generally between the ages of 3 and 18. Some participants will have already taken Infigratinib in a previous study and are continuing with it. Others may be new to the medicine, but they must have had their growth carefully monitored for at least six months in another study beforehand.

To join, participants and their parents or guardians must be able to attend study visits and follow the instructions. The child must also be able to swallow tablets. For girls aged 10 or older, or those who have started their periods, a negative pregnancy test is needed. If sexually active, effective contraception must be used during and for one month after the study.

Children cannot join if they have another health condition that might interfere with the study or make participation unsafe. They also can't be in another clinical study not sponsored by QED. Those who have already reached their full adult height are not eligible to join.

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.

  1. Is your child aged 3 to 18 years old?
  2. Does your child have achondroplasia?
  3. Can your child swallow tablets?
  4. Are you able to attend regular study visits?
  5. If your child is a girl aged 10 or over, or has started her periods, is she not pregnant?
  6. Has your child NOT reached their full adult height?
Responda a todas as perguntas para ver o seu resultado.

O que envolve a participação?

If you or your child decides to take part in this study, you'll have regular visits to the clinic. These visits will involve health checks, blood tests, and other assessments to see how the medicine is affecting growth and overall health. The medication, Infigratinib, will be taken as tablets.

Throughout the study, doctors will closely monitor for any side effects and check how well the medicine is working. Part of the study also involves assessing your child's quality of life, which means asking questions about how they feel and their daily activities. The total duration of participation will be discussed with you, as this is an extension study and participants may continue for a longer period.

Riscos e benefícios potenciais

Participating in a study like this might offer potential benefits, such as contributing to scientific understanding of achondroplasia and possibly helping your child's growth. However, there are also potential risks; the medicine might have side effects, and some benefits may not be seen. All possible risks and benefits will be fully explained before you agree to participate. Remember, joining a clinical study is always voluntary, and you have the right to withdraw at any time without affecting your or your child's medical care.

Locations (31)

  • UCSF Benioff Children's Hospital, Oakland
    Código postal verificado
    Oakland, Estados Unidos
  • Children's Hospital Colorado
    Código postal verificado
    Aurora, Estados Unidos
  • Nemours Alfred I. Dupont Hospital for Children
    Código postal verificado
    Wilmington, Estados Unidos
  • Johns Hopkins University
    Código postal verificado
    Baltimore, Estados Unidos
  • University of Missouri
    Código postal verificado
    Columbia, Estados Unidos
  • Cincinnati Children's Hospital Medical Center
    Código postal verificado
    Cincinnati, Estados Unidos
  • Vanderbilt University Medical Center
    Código postal verificado
    Nashville, Estados Unidos
  • University Hospital and UW Health Clinics
    Código postal verificado
    Madison, Estados Unidos
  • Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan
    Não verificado
    Cidade Autônoma de Buenos Aires, Argentina
  • Murdoch Children's Hospital
    Código postal verificado
    Parkville, Austrália
  • Stollery Children's Hospital
    Código postal verificado
    Edmonton, Canada
  • Children's Hospital - London Health Sciences Centre
    Código postal verificado
    Londres, Canadá

Perguntas frequentes

What is achondroplasia?

Achondroplasia is a genetic condition that affects how bones grow, leading to shorter limbs and other features, and is a common cause of dwarfism.

What is Infigratinib?

Infigratinib is a medicine being studied to see if it can help improve bone growth in children with achondroplasia.

Eu tenho que pagar para participar do estudo?

No, participation in a clinical study typically does not involve any costs for the participants.

What does 'extension study' mean?

An 'extension study' means that many participants have already taken this medicine in a previous study and are continuing it to see its long-term effects.

Posso parar de participar do estudo a qualquer momento?

Yes, you or your child can stop participating in the study at any time, for any reason, without affecting your medical care.

Como saber mais

Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.

Interessado em participar?

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