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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.

At a glance

Status
Recruiting
Sponsor
QED Therapeutics, a BridgeBio company
Enrolment target
150
Start
05 Jun 2024
Estimated completion
01 Oct 2026

What is this study about?

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.

Key takeaways

  • 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.

Who may be eligible?

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.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

  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?
Answer every question to see your result.

What does participation involve?

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.

Potential risks and benefits

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)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • UCSF Benioff Children's Hospital
    Verified postcode
    Oakland, United States· Recruiting
  • Childrens Hospital Colorado
    Verified postcode
    Aurora, United States· Recruiting
  • Children's National Hospital
    Verified postcode
    Washington D.C., United States· Recruiting
  • Johns Hopkins School of Medicine
    Verified postcode
    Baltimore, United States· Recruiting
  • University of Missouri
    Verified postcode
    Columbia, United States· Recruiting
  • Cincinnati Children's Hospital Medical Center
    Verified postcode
    Cincinnati, United States· Recruiting
  • Vanderbilt University Medical Center
    Verified postcode
    Nashville, United States· Recruiting
  • University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
    Verified postcode
    Madison, United States· Recruiting
  • Murdoch Children's Research Institute
    Verified postcode
    Parkville, Australia· Recruiting
  • London Health Sciences Centre - Children's Hospital of Western Ontario
    Verified postcode
    London, Canada· Recruiting
  • Children's Hospital of Eastern Ontario Research Institute
    Verified postcode
    Ottawa, Canada· Recruiting
  • Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
    Verified postcode
    Montreal, Canada· Recruiting

Common questions

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.

How to find out more

QED Therapeutics, Inc

Always speak to your GP or specialist before deciding to take part in a study.

Interested in taking part?

Register your interest

Share your details and the research team for "Prospective Clinical Assessment Study in Children With Hypoc…" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

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