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New study tracks growth in kids with rare bone disorder

NCT ID NCT06410976

Knowledge-focused Sponsor: QED Therapeutics, a BridgeBio company Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 17, 2026 · Updated 2 times

Summary

This study watches children aged 2.5 to 17 with hypochondroplasia, a condition causing short stature, to track their growth and health over time. No treatments or drugs are given. The goal is to learn more about how the condition affects growth, body proportions, thinking skills, and quality of life. About 150 children will take part.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 150 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2024

Expected to finish

Oct 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Children with hypochondroplasia

Ages

30 months to 16 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

bone disorder Dwarfism hereditary disease hypochondroplasia Mucopolysaccharidosis IV musculoskeletal system disorder osteochondrodysplasia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hypochondroplasia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    25 sites in 10 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Astrid Lindgren Children's Hospital

    RECRUITING

    Solna, 17164, Sweden

  • Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants

    RECRUITING

    Toulouse, 31059, France

  • Children's Hospital of Eastern Ontario Research Institute

    RECRUITING

    Ottawa, Ontario, K1H 8L1, Canada

  • Children's National Hospital

    RECRUITING

    Washington D.C., District of Columbia, 20010, United States

  • Childrens Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Glasgow Clinical Research Facility, Queen Elizabeth University Hospital

    RECRUITING

    Glasgow, Scotland, G51 4TF, United Kingdom

  • Haukeland University Hospital

    RECRUITING

    Bergen, 5021, Norway

  • Hospital Pediátrico de Coimbra

    RECRUITING

    Coimbra, 3000-602, Portugal

  • Hôpital Femme Mère Enfant HCL

    RECRUITING

    Bron, 69677, France

  • Hôpital Necker-Enfants Malades

    RECRUITING

    Paris, 75015, France

  • Johns Hopkins School of Medicine

    RECRUITING

    Baltimore, Maryland, 21287, United States

  • KK Women's and Children's Hospital

    RECRUITING

    Singapore, 229899, Singapore

  • London Health Sciences Centre - Children's Hospital of Western Ontario

    RECRUITING

    London, Ontario, N6C 2R5, Canada

  • Manchester University

    RECRUITING

    Manchester, England, M13 9WL, United Kingdom

  • Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne

    RECRUITING

    Parkville, Victoria, 3052, Australia

  • Paediatric Clinical Research Unit at Oslo University Hospital

    RECRUITING

    Oslo, 0372, Norway

  • Sheffield Children's NHS Foundation Trust

    RECRUITING

    Sheffield, England, S10 2TH, United Kingdom

  • The Portland Hospital for Women and Children

    RECRUITING

    London, England, W1W 5AH, United Kingdom

  • UCSF Benioff Children's Hospital

    RECRUITING

    Oakland, California, 94609, United States

  • Unidad de Cirugía Artroscopica, Hopsital MIKS

    RECRUITING

    Vitoria-Gasteiz, 01010, Spain

  • University of Missouri

    RECRUITING

    Columbia, Missouri, 65201, United States

  • University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

    RECRUITING

    Madison, Wisconsin, 53705, United States

  • Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine

    RECRUITING

    Montreal, Quebec, H3T 1C5, Canada

  • Vanderbilt University Medical Center

    RECRUITING

    Nashville, Tennessee, 37232, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.