Alla studier
RekryteringOBSERVATIONELL

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.

Överblick

Status
Rekrytering
Sponsor
QED Therapeutics, a BridgeBio company
Inskrivningsmål
150
Starta
05 Jun 2024
Beräknad slutförande
01 Okt 2026

Vad handlar denna studie om?

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.

Viktiga punkter

  • 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.

Vem kan vara berättigad?

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.

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. 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?
Svara på varje fråga för att se ditt resultat.

Vad innebär deltagande?

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.

Potentiella risker och fördelar

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.

Platser (25)

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

Vanliga frågor

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.

Hur länge kommer mitt barn att vara med i studien?

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.

Hur du får reda på mer

QED Therapeutics, Inc

Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.

Intresserad av att delta?

Registrera ditt intresse

Dela dina uppgifter och forskarteamet för "Prospective Clinical Assessment Study in Children With Hypoc…" kommer att kontakta dig om du kan vara berättigad. Prata alltid med din läkare innan du går med på att delta.

Diskussion

Gemenskapsdiskussion

Drivs av vårt forum på community.patient.info. Var vänlig och respektfull — detta är inte medicinsk rådgivning.