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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.

En un coup d'œil

Statut
Inscription sur invitation
Phase
PHASE2
Sponsor
QED Therapeutics, a BridgeBio company
Objectif d'inscription
325
Démarrer
06 Dec 2021
Achèvement estimé
01 Feb 2032

De quoi parle cette étude ?

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.

Points clés

  • 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.
  • Vous pouvez vous retirer de l'étude à tout moment.

Qui peut être éligible ?

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.

Cet étude pourrait-elle vous convenir ?

Répondez à ces questions rapides pour voir si vous êtes éligible. Ceci est un guide uniquement — l'équipe de recherche prend la décision finale.

  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?
Répondez à chaque question pour voir votre résultat.

En quoi consiste la participation ?

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.

Risques et avantages potentiels

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.

Emplacements (31)

  • UCSF Benioff Children's Hospital, Oakland
    Code postal vérifié
    Oakland, États-Unis
  • Children's Hospital Colorado
    Code postal vérifié
    Aurora, États-Unis
  • Nemours Alfred I. Dupont Hospital for Children
    Code postal vérifié
    Wilmington, United States
  • Université Johns Hopkins
    Code postal vérifié
    Baltimore, États-Unis
  • University of Missouri
    Code postal vérifié
    Columbia, United States
  • Cincinnati Children's Hospital Medical Center
    Code postal vérifié
    Cincinnati, United States
  • Vanderbilt University Medical Center
    Code postal vérifié
    Nashville, United States
  • University Hospital and UW Health Clinics
    Code postal vérifié
    Madison, United States
  • Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan
    Non vérifié
    Ciudad Autonoma Buenos Aires, Argentina
  • Murdoch Children's Hospital
    Code postal vérifié
    Parkville, Australie
  • Stollery Children's Hospital
    Code postal vérifié
    Edmonton, Canada
  • Children's Hospital - London Health Sciences Centre
    Code postal vérifié
    Londres, Canada

Questions fréquentes

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.

Do I have to pay to be in the study?

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.

Can I stop participating in the study at any time?

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

Comment en savoir plus

Parlez toujours à votre médecin généraliste ou spécialiste avant de décider de participer à une étude.

Intéressé à participer ?

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Partagez vos coordonnées et l'équipe de recherche pour "Extension Study of Infigratinib in Children With Achondropla… vous contactera si vous êtes éligible. Consultez toujours votre médecin avant d'accepter de participer.

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