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Prospective Clinical Assessment Study in Children With Hypochondroplasia

Sponsor: QED Therapeutics, a BridgeBio company

NCT ID: NCT06410976

View on ClinicalTrials.gov ↗

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.

Hypochondroplasia

How this trial compares with your answers

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What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

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
Inclusion Criteria: Signed informed consent. 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 molecular test. 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 study procedures. Exclusion Criteria: 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 to screening. Having a clinically significant disease or condition that in view of the investigator or Sponsor will interfere with the evaluation of growth, with study participation or not be in the best interest of the participant. 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 for ≥ 30 days or with the last dose \<6 months before screening. Have had regular long-term treatment (\>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable). Previous limb-lengthening surgery or guided growth surgery with plates still in place or removed within the 6 months prior to screening. Having had a fracture of the long bones or spine within 12 months of screening. History and/or current evidence of extensive ectopic tissue calcification. History of malignancy. Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation, and/or would place the participant at high risk for poor compliance with study activities or for not completing the study. Current participation in any other ongoing clinical study with another sponsor.

Where Is This Study? (4 UK sites)

The Portland Hospital for Women and Children

London W1W 5AH, United Kingdom

Recruiting

Manchester University

Manchester M13 9WL, United Kingdom

Recruiting
Hospital R&D contact (matched)

Elizabeth Mainwaring

R&D.applications@mft.nhs.uk0161 276 3340

Sheffield Children's NHS Foundation Trust

Sheffield S10 2TH, United Kingdom

Recruiting
Hospital R&D contact (matched)

Sarah Flude

scn-tr.research.governance@nhs.net0114 226 7980

Glasgow Clinical Research Facility, Queen Elizabeth University Hospital

Glasgow G51 4TF, United Kingdom

Recruiting

How to Get in Touch

QED Therapeutics, Inc

Sponsor contact

CONTACT

1-877-280-5655 medinfo@qedtx.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-05