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 28 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 study is looking at children aged 2.5 to under 17 with hypochondroplasia, a genetic condition that causes short stature. It is run by QED Therapeutics, a BridgeBio company. The study checks participants' health and growth over time.
Who can take part
- Aged 2.5 to under 17 years
- Diagnosed with hypochondroplasia (HCH) through short stature checks and a genetic test
- Able to walk and stand without help
- The child and their parent, guardian or carer can attend study visits and follow study steps
- Signed informed consent
Who may not be able to
- Has achondroplasia or another short stature condition instead of HCH
- Girls who have started their periods
- Growing 1.5 cm per year or less over at least 6 months before joining
- Another illness or condition that could affect growth checks or taking part
- Very abnormal screening blood or lab test results
- Eye problems affecting the cornea or retina
- Used another treatment for HCH or short stature for 30 days or more, or within 6 months before joining
- Long-term steroid tablets in the past month (low-dose asthma inhalers are OK)
- Previous limb-lengthening surgery, or guided growth surgery with plates still in place or removed in the past 6 months
- A broken long bone or spine fracture within the past 12 months
- History of widespread abnormal calcium deposits in body tissue
- History of cancer
- Anything else that doctors or the sponsor think would make taking part unsafe or too hard
- Currently taking part in another clinical study
What taking part involves
- • Not stated — ask the trial team
Time commitment: Not stated — ask the trial team about how long the study lasts, how many visits there are and what happens at them.
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
- 30 Months to 16 Years
- Who
- All
- Number of participants
- 150
- Started
- 2024-06-05
- Last checked
- 2026-05
Plain English Summary
What is this study?
- • Testing a new treatment for hypochondroplasia
- • Clinical study - 150 participants
- • This is a long-term, multicenter, non-interventional study of children ages 2
Who can take part?
- • Ages 30 Months to 16 Years
- • Diagnosed with hypochondroplasia
Where?
- • London - The Portland Hospital for Women and Children
- • Manchester - Manchester University
- • Sheffield - Sheffield Children's NHS Foundation Trust
- • Glasgow - Glasgow Clinical Research Facility, Queen Elizabeth University Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This is a long-term, multicenter, non-interventional study of children ages 2.5 to \<17 years with hypochondroplasia (HCH).
More detail
The objective is to evaluate growth, HCH-related medical complications, health-related quality of life, functional abilities and cognitive functions of study participants. Data collected will contribute to the characterization of the natural history of children with HCH. No study medication will be administered.
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: 30 Months - 16 Years
- Who can join: All genders
What the study is looking for
- ✓Signed agreement to take part.
- ✓Aged 2.5 to \<17 years at study entry.
- ✓Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a...
- ✓Participants are ambulatory and able to stand without assistance.
- ✓Study participants and parent(s), guardian(s), or caregiver(s) are willing and able to comply with study visits and...
Who cannot take part
- ✗Have ACH or short stature condition other than HCH.
- ✗In females, having had their menarche. Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior...
- ✗Clinically significant abnormality in any laboratory test result at screening
- ✗Current evidence of corneal or retinal disorders.
- ✗Have used any other investigational or approved product or medical device for the treatment of HCH or short stature...
See the full criteria
Where Is This Study? (4 UK sites)
The Portland Hospital for Women and Children
London W1W 5AH, United Kingdom
Manchester University
Manchester M13 9WL, United Kingdom
Sheffield Children's NHS Foundation Trust
Sheffield S10 2TH, United Kingdom
Glasgow Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow G51 4TF, United Kingdom
How to Get in Touch
QED Therapeutics, Inc
Sponsor contactCONTACT
