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Prospective Clinical Assessment Study in Children With Hypochondroplasia

This is a long-term study for children aged 2.5 to 16 years who have hypochondroplasia, a condition that causes short stature. The main goal is to understand how this condition impacts children's lives over several years. Researchers will observe their growth patterns and look at any health problems related to hypochondroplasia. They also want to see how the condition affects their everyday activities, general well-being, and thinking skills. This study is observational, meaning no new medicines or treatments are being tested or given. It simply involves gathering information to help improve our understanding of hypochondroplasia and how best to support children living with it.

Em resumo

Status
Recrutamento
Patrocinador
QED Therapeutics, a BridgeBio company
Meta de inscrição
150
Iniciar
05 Jun 2024
Conclusão estimada
01 Out 2026

Sobre o que é este estudo?

This study is called a "natural history" study, which means doctors want to learn more about how hypochondroplasia (HCH) usually develops and affects children over time. HCH is a type of short stature, meaning children with this condition are typically shorter than others their age due to problems with bone growth.

The researchers will follow children with HCH from ages 2.5 up to 16 years old. They will carefully observe and collect information about several things: how the children grow, any health problems they might develop because of HCH, how they get on with day-to-day activities, their general quality of life, and their thinking and learning abilities. This information will help doctors and scientists gain a much better understanding of HCH.

By gathering all this detail, the study aims to build a clearer picture of what it's like to live with HCH as a child. This improved understanding can then help develop better ways to support and care for children with the condition in the future. It's important to know that this study doesn't involve giving any new medications; it's purely about observing and collecting information.

Pontos principais

  • This study helps doctors learn more about hypochondroplasia, a type of short stature.
  • It involves observing children aged 2.5 to 16; no new medicines are given.
  • Information gathered will help improve future care for children with hypochondroplasia.
  • Participation includes regular clinic visits to track growth and overall health.
  • You can stop taking part at any time without affecting your child's medical care.

Quem pode ser elegível?

To join this study, children need to be between two and a half and sixteen years old. They must have a clear diagnosis of hypochondroplasia, which means doctors have confirmed it both by their physical appearance (being shorter than average) and by a genetic test.

It's important that the child can walk and stand on their own without help. Also, the child and their parents or guardians must be willing and able to attend all the study appointments and follow the study instructions.

Children generally can't join if they have other conditions causing short stature, have already started puberty (if they are a girl), have recently had bone-lengthening surgery, or have certain other significant health problems that might make taking part unsafe or interfere with the study's results.

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 between 2 and a half and 16 years old?
  2. Has your child been diagnosed with hypochondroplasia by both a doctor and a genetic test?
  3. Can your child walk and stand on their own without help?
  4. Are you and your child able to attend study appointments regularly?
  5. Has your child NOT had puberty if they are a girl?
  6. Has your child NOT had significant limb-lengthening surgery recently?
Responda a todas as perguntas para ver o seu resultado.

O que envolve a participação?

If you decide to take part, your child will have regular visits to the study clinic. During these visits, doctors and nurses will measure your child's growth (height, weight, etc.), monitor any health problems, and ask questions about their daily life, how they feel, and what they can do. There will also be some simple tests to check their thinking skills. No new medicines or treatments will be given as part of this study. The total duration of participation will depend on how long the study continues, but it is a long-term study that aims to follow children over several years to understand the condition's progression.

Riscos e benefícios potenciais

There are no medical treatments given in this study, so there are no direct risks from new medications. The main "risk" is the time commitment required for clinic visits and the potential for some minor discomfort from standard measurements or examinations. The potential benefit is that the information gathered will help doctors and researchers better understand hypochondroplasia, which could lead to improved care and support for children with the condition in the future. You are always free to withdraw from the study at any time for any reason without it affecting your child's medical care.

Locations (25)

  • UCSF Benioff Children's Hospital
    Código postal verificado
    Oakland, Estados Unidos· Recrutamento
  • Childrens Hospital Colorado
    Código postal verificado
    Aurora, Estados Unidos· Recrutamento
  • Children's National Hospital
    Código postal verificado
    Washington D.C., Estados Unidos· Recrutamento
  • Johns Hopkins School of Medicine
    Código postal verificado
    Baltimore, Estados Unidos· Recrutamento
  • University of Missouri
    Código postal verificado
    Columbia, Estados Unidos· Recrutamento
  • Cincinnati Children's Hospital Medical Center
    Código postal verificado
    Cincinnati, Estados Unidos· Recrutamento
  • Vanderbilt University Medical Center
    Código postal verificado
    Nashville, Estados Unidos· Recrutamento
  • University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
    Código postal verificado
    Madison, Estados Unidos· Recrutamento
  • Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne
    Código postal verificado
    Parkville, Austrália· Recrutamento
  • London Health Sciences Centre - Children's Hospital of Western Ontario
    Código postal verificado
    Londres, Canadá· Recrutamento
  • Children's Hospital of Eastern Ontario Research Institute
    Código postal verificado
    Ottawa, Canadá· Recrutamento
  • Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
    Código postal verificado
    Montreal, Canadá· Recrutamento

Perguntas frequentes

What is hypochondroplasia?

It's a condition that causes shorter than average height, affecting bone growth but usually less severely than some other similar conditions.

Will my child receive any new medicine in this study?

No, this study does not involve giving any new medicines or treatments; it's purely about observing and collecting information.

How long will my child be in the study?

This is a long-term study designed to follow children for several years to understand how the condition changes over time.

What kind of appointments will my child have?

Appointments will involve measuring growth, checking for health issues, and asking questions about daily life and learning, similar to regular check-ups.

Can we stop participating if we change our mind?

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

Como saber mais

QED Therapeutics, Inc

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