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Einschreibung auf EinladungPHASE2INTERVENTIONELL

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

This research is an ongoing study for children aged 3 to 18 who have a medical condition called Hypochondroplasia. The study is called an 'open-label, long-term extension' study and focuses on a medicine named Infigratinib. This means that everyone taking part will receive the medicine, and the study will continue for a long time to see its effects. The main goal is to check how safe Infigratinib is over time and how well it helps children with the condition. All participants in this study would have already taken part in a previous study about Infigratinib, mainly to continue observing its effects.

Auf einen Blick

Status
Einschreibung auf Einladung
Phase
PHASE2
Sponsor
QED Therapeutics, ein Unternehmen von BridgeBio
Einschreibeziel
135
Starten
23. Apr 2026
Geschätzte Fertigstellung
31 May 2036

Worum geht es in dieser Studie?

This study is a follow-up for children with Hypochondroplasia (HCH) who have already participated in an earlier research study about a medicine called Infigratinib. HCH is a type of short stature. The researchers want to learn more about how Infigratinib works over a longer period, specifically focusing on its safety and how effectively it helps these children.

Infigratinib is a type of medicine designed to target specific signals in the body that are involved in bone growth. By looking at patients over a longer time, the study aims to build a more complete picture of the medicine's effects, how the body handles it, and if it continues to be helpful for growth and overall health in children with HCH.

Understanding a medicine's effects in the long term is very important for doctors and patients. This type of study helps make sure that if Infigratinib becomes widely available, we have a good understanding of its benefits and any potential challenges it might present when used for many years.

Wichtige Erkenntnisse

  • This study is for children with Hypochondroplasia who previously took part in an Infigratinib study.
  • It aims to understand the long-term safety and benefits of the medicine, Infigratinib.
  • Participants will receive the study medicine and have regular health check-ups.
  • The study includes children aged 3 to 18 years.
  • Joining is voluntary, and you can withdraw at any time.
  • It helps gather important information for future treatments for Hypochondroplasia.

Wer könnte berechtigt sein?

This study is open to children aged between 3 and 18 years old who have Hypochondroplasia. A very important requirement is that they must have already completed a previous study called ACCEL 2/3, or in some cases, the observational ACCEL study, specifically those who had at least six months of growth monitoring in that study.

There are also some reasons why a child might not be able to join. For example, if they have another serious medical condition that could interfere with the study or make participation unsafe. Also, if they are already taking part in another medical study with a different organisation, or if they have already reached their full adult height, they would not be able to join. Girls who are 10 years or older, or any girl who has started her periods, will need to have a negative pregnancy test to make sure they are not pregnant.

Könnte diese Studie für Sie geeignet sein?

Beantworten Sie diese kurzen Fragen, um zu sehen, ob Sie möglicherweise berechtigt sind. Dies ist nur ein Leitfaden – das Forschungsteam trifft die endgültige Entscheidung.

  1. Is my child aged between 3 and 18 years old?
  2. Does my child have Hypochondroplasia?
  3. Did my child complete the previous ACCEL 2/3 study (or ACCEL with 6 months of growth tracking)?
  4. Does my child have any other serious medical conditions that could affect study participation?
  5. Is my child currently participating in another clinical study with a different company?
  6. Has my child reached their full adult height?
Beantworten Sie jede Frage, um Ihr Ergebnis zu sehen.

Was beinhaltet die Teilnahme?

If your child joins this study, they will receive the study medicine, Infigratinib, for an extended period. Since this is an extension study, the exact number and frequency of visits will be based on the established routine from the previous study your child participated in. Typically, this would involve regular clinic visits where doctors and nurses will monitor your child’s health, growth, and how the medicine is affecting them. These visits will include checks like measuring height and weight, and possibly blood tests or other assessments to keep track of their progress and safety. The total duration of participation aims to observe effects over a long period, but specific end points will be discussed with the study team.

Potenzielle Risiken und Vorteile

Participating in this study might offer potential benefits, such as continuing to receive the study medicine, Infigratinib, which could potentially help with your child's growth. It also contributes valuable information that helps medical science understand Hypochondroplasia better for all children. Potential risks could include side effects from the medicine, which would be monitored closely by the study team. You always have the right to withdraw your child from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (24)

  • UCSF Benioff Kinderkrankenhaus
    Verifizierte Postleitzahl
    Oakland, Vereinigte Staaten
  • Kinderkrankenhaus Colorado
    Verifizierte Postleitzahl
    Aurora, Vereinigte Staaten
  • Nationales Kinderkrankenhaus
    Verifizierte Postleitzahl
    Washington D.C., Vereinigte Staaten
  • Johns Hopkins Medizinische Fakultät
    Verifizierte Postleitzahl
    Baltimore, Vereinigte Staaten
  • Universität von Missouri
    Verifizierte Postleitzahl
    Columbia, Vereinigte Staaten
  • Cincinnati Kinderkrankenhaus Medizinisches Zentrum
    Verifizierte Postleitzahl
    Cincinnati, Vereinigte Staaten
  • Vanderbilt Universitätsklinikum
    Verifizierte Postleitzahl
    Nashville, Vereinigte Staaten
  • Universität von Wisconsin Madison - Waisman Center Knochen-Dysplasie-Klinik
    Verifizierte Postleitzahl
    Madison, Vereinigte Staaten
  • Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne
    Verifizierte Postleitzahl
    Parkville, Australien
  • London Health Sciences Centre - Kinderkrankenhaus von West-Ontario
    Verifizierte Postleitzahl
    London, Kanada
  • Forschungsinstitut des Kinderkrankenhauses von Ost-Ontario
    Verifizierte Postleitzahl
    Ottawa, Kanada
  • Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
    Verifizierte Postleitzahl
    Montreal, Kanada

Häufige Fragen

What is Hypochondroplasia?

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

What is Infigratinib?

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

Why is this study only for children who were in a previous study?

This study is designed to follow up on children who have already received Infigratinib in an earlier study (ACCEL 2/3) to understand its long-term effects on safety and growth.

Will my child automatically receive the medicine?

Yes, this is an 'open-label' study, meaning all participants who are eligible and enrol will receive the study medicine, Infigratinib.

Can we stop participating at any time?

Yes, you have the right to withdraw your child from the study at any point, and it will not affect their regular medical care.

Wie Sie mehr erfahren können

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