At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Not specified by the sponsor
- How long the study runs
- Study runs about 241 months (dates as stated)
- About the drug or intervention
- Not specified by the sponsor
- Patient visit burden
- Not specified by the sponsor
In plain English
This is an observational study of children with hypochondroplasia, a genetic condition that causes short stature. 'Observational' means the study team collects information rather than testing a treatment. The study runs at many centres in more than one country and is sponsored (paid for) by BioMarin Pharmaceutical.
Who can take part
- The child must be aged 15 or younger when the informed consent (permission to take part) is signed.
- The child must have their hypochondroplasia diagnosis confirmed by a genetic test.
Who may not be able to
- Having another genetic condition that causes short stature, or a gene change known to cause another genetic syndrome linked to short stature.
- Having received a medicine or device being tested in research (an investigational product) within 6 months before the screening visit (the first study check-up).
What taking part involves
- • This is an observational study, so the team watches and records information rather than giving a treatment.
- • Any further details are not stated — ask the trial team.
Time commitment: How many visits there are, how long the study lasts, and what taking part involves are not stated — ask the trial team.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Observing health over time
- Ages
- Up to 15 Years
- Who
- All
- Number of participants
- 400
- Started
- 2023-11-27
- Last checked
- 2025-04
Plain English Summary
What is this study?
- • Testing a new treatment for hypochondroplasia
- • Clinical study - 400 participants
- • This study will assess growth over time and the clinical course of HCH in children by collecting growth measurements and other variables of interest
Who can take part?
- • Ages Up to 15 Years
- • Diagnosed with hypochondroplasia
Where?
- • London - Great Ormond Street Hospital for Children NHS Foundation Trust
- • London - Myriad Trials Ltd
- • Glasgow - NHS Greater Glasgow and Clyde
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This study will assess growth over time and the clinical course of HCH in children by collecting growth measurements and other variables of interest.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: Up to 15 Years
- Who can join: All genders
Treatment history
Treatments you must have had:
- ✓ genetic confirmation of Hypochondroplasia diagnosis
What the study is looking for
- ✓Participants must be ≤ 15 years old at the time of signing the agreement to take part
- ✓Participants must have genetic confirmation of Hypochondroplasia diagnosis
Who cannot take part
- ✗Have a diagnosis of another genetic short stature condition other than Hypochondroplasia or a genetic variant known...
- ✗Received an the the study treatment or medical device within 6 months before the Screening visit
See the full criteria
Where Is This Study? (3 UK sites)
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700Myriad Trials Ltd
London, United Kingdom
NHS Greater Glasgow and Clyde
Glasgow, United Kingdom
How to Get in Touch
Trial Specialist
Sponsor contactCONTACT