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Registrering genom inbjudanPHASE2, PHASE3INTERVENTIONELL

An Interventional Study of Infigratinib in Children With Hypochondroplasia

This study is investigating a new medication, infigratinib, to see how well it works and if it's safe for children with hypochondroplasia (a condition that causes shorter height). It's for children aged between 3 and 18 years old. The study has two main parts: one open-label part where everyone knows they are receiving the medicine, and a second part where some children will receive the medicine and others a dummy treatment (placebo) without anyone knowing who gets what. This helps researchers understand the medicine's true effects. To join, children must have been part of a previous related study and meet certain health requirements.

Överblick

Status
Registrering genom inbjudan
Fas
PHASE2, PHASE3
Sponsor
QED Therapeutics, a BridgeBio company
Inskrivningsmål
24
Starta
22 Apr 2025
Beräknad slutförande
01 Jan 2027

Vad handlar denna studie om?

This study, called ACCEL2/3, is looking into a new medicine called infigratinib for children who have a condition called hypochondroplasia. Children with hypochondroplasia are shorter than average due to an issue with how their bones grow. The main goal of this study is to find out if infigratinib is safe and helps children with hypochondroplasia to grow better.

The study is split into two main sections. The first part involves children aged 5 to 11, and everyone in this group will receive the medicine. This helps the researchers get an initial idea of how the medicine works. The second, larger part of the study includes children aged 3 to 18. In this part, some children will receive the study medicine, while others will get a placebo (a dummy treatment with no medicine in it). Neither the participants nor their doctors will know who is getting which treatment. This is a common way to test new medicines fairly.

Taking part in a study like this is important because it helps scientists understand new treatments and potentially improve the lives of children with hypochondroplasia. All studies are carefully reviewed to ensure they are safe and ethical for the participants involved.

Viktiga punkter

  • This study is for children aged 3 to 18 with hypochondroplasia.
  • It's testing a new medicine called infigratinib to see if it's safe and helps growth.
  • Some children will receive the medicine, others may receive a dummy treatment.
  • Participation involves regular hospital visits for check-ups and tests.
  • It's a chance to access a new potential treatment, but risks are possible.
  • You can withdraw your child from the study at any time.

Vem kan vara berättigad?

To join this study, children must already be taking part in another related study (QBGJ398-004) and have completed at least 26 weeks. They also need to have hypochondroplasia, which has been confirmed by a doctor and a special test.

For the first part of the study, children need to be between 5 and 11 years old. For the second, larger part, children can be from 3 up to 18 years old and still have the potential to grow. It’s also important that they can swallow medicine by mouth and are able to stand and walk on their own.

There are also some things that would prevent a child from joining. For example, if they have another reason for being short other than hypochondroplasia, or if they have certain serious health problems, they wouldn't be able to take part. Also, if they've had specific eye problems or certain types of previous surgery, they might not be suitable.

Kan denna studie passa dig?

Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.

  1. Är ditt barn mellan 3 och 18 år gammalt?
  2. Does your child have hypochondroplasia confirmed by a doctor and a test?
  3. Has your child been part of a previous related study for at least 26 weeks?
  4. Can your child swallow medicine by mouth and stand or walk without help?
  5. Does your child not have any serious eye problems or other major health conditions that would interfere?
Svara på varje fråga för att se ditt resultat.

Vad innebär deltagande?

If your child takes part in this study, they would receive a medicine called infigratinib, which is given by mouth. The study involves regular visits to the hospital or clinic. These visits will likely include growth measurements, general health checks, and possibly blood tests or eye exams to monitor how your child is doing and to check for any side effects.

The study will last for a period of time, and the research team will explain exactly how long your child would need to be involved and what each visit would entail. It's important that participants and their parents or guardians are able and willing to attend all scheduled appointments and follow the study instructions carefully. If your child is old enough to have periods, or if they are sexually active, there will be specific requirements about pregnancy tests and using contraception during and for a month after the study medicine.

Potentiella risker och fördelar

Taking part in this study may offer some potential benefits, such as access to a new medicine that could help bone growth in children with hypochondroplasia. However, as with any new medicine, there are also potential risks, including side effects that are not yet fully known. The research team will carefully monitor your child for any problems during the study. It's important to remember that you and your child have the right to withdraw from the study at any time, for any reason, without it affecting your child's future medical care.

Platser (24)

  • UCSF Benioff Children's Hospital
    Verifierat postnummer
    Oakland, USA
  • Barnsjukhuset Colorado
    Verifierat postnummer
    Aurora, USA
  • Barnens Nationella Sjukhus
    Verifierat postnummer
    Washington D.C., USA
  • Johns Hopkins School of Medicine
    Verifierat postnummer
    Baltimore, USA
  • University of Missouri
    Verifierat postnummer
    Columbia, United States
  • Cincinnati Barnsjukhus Medicinska Center
    Verifierat postnummer
    Cincinnati, USA
  • Vanderbilt University Medical Center
    Verifierat postnummer
    Nashville, USA
  • University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
    Verifierat postnummer
    Madison, USA
  • Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne
    Verifierat postnummer
    Parkville, Australien
  • London Health Services Centre - Children's Hospital of Western Ontario
    Verifierat postnummer
    London, Kanada
  • Children's Hospital of Eastern Ontario Research Institute
    Verifierat postnummer
    Ottawa, Kanada
  • Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
    Verifierat postnummer
    Montreal, Kanada

Vanliga frågor

What is hypochondroplasia?

Hypochondroplasia is a genetic condition that affects bone growth, leading to shorter stature, especially in the arms and legs.

What is infigratinib?

Infigratinib is the name of the new medicine being tested in this study to see if it can help children with hypochondroplasia grow.

Will my child get the actual medicine or a dummy pill?

In the first part of the study (for children aged 5-11), everyone receives the medicine. In the second part (for ages 3-18), some children will get the medicine and others a dummy pill (placebo) to fairly test its effects.

What happens after the study ends?

The study team will review all the information to understand if the medicine is safe and effective. Your child's doctor can discuss future treatment options based on their needs.

Kan jag ändra mig angående mitt barns deltagande?

Yes, you can decide to withdraw your child from the study at any time, for any reason, and it won't affect their regular medical care.

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