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Hope for kids with rare bone disorder: new drug shows promise in Long-Term trial

NCT ID NCT07393373

Disease control 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 trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 27, 2026 · Updated 2 times

Summary

This study is for children with hypochondroplasia, a condition that causes short stature and bone differences. Researchers are testing a drug called infigratinib to see if it is safe and helps improve growth over the long term. Participants must have already completed a previous study of the same drug. The goal is to understand if the drug can help children grow taller and improve body proportions.

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.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 135 people

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

Started

Apr 2026

Expected to finish

May 2036

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.

Ages

3 to 18 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: * Inclusion Criteria for Participants Rolling Over from ACCEL 2/3 1. Pediatric participants with HCH who have completed ACCEL 2/3 2. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche Exclusion Criteria: * Exclusion Criteria for Participants Rolling Over from ACCEL 2/3 1. Participant has concurrent medical condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations 2. Participants who developed a medical condition that requires the initiation of treatment with a prohibited medication 3. Participants who prematurely discontinued ACCEL 2/3 4. Participants who have reached final height or near final height 5. Current participation in an ongoing clinical study with a sponsor other than QED

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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 places running it

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

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. 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

Locations

  • Astrid Lindgren Children's Hospital

    Solna, 17164, Sweden

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

    Toulouse, 31059, France

  • Children's Hospital of Eastern Ontario Research Institute

    Ottawa, Ontario, K1H 8L1, Canada

  • Children's National Hospital

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

  • Childrens Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Glasgow Clinical Research Facility, Queen Elizabeth University Hospital

    Glasgow, Scotland, G51 4TF, United Kingdom

  • Haukeland University Hospital

    Bergen, 5021, Norway

  • Hospital Pediátrico de Coimbra

    Coimbra, 3000-602, Portugal

  • Hôpital Femme Mère Enfant

    Bron, 69677, France

  • Hôpital Universitaire Necker-Enfants Malades

    Paris, 75015, France

  • Johns Hopkins School of Medicine

    Baltimore, Maryland, 21287, United States

  • KK Women's and Children's Hospital

    Singapore, 229899, Singapore

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

    London, Ontario, N6C 2R5, Canada

  • Manchester University

    Manchester, England, M13 9WL, United Kingdom

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

    Parkville, Victoria, 3052, Australia

  • Paediatric Clinical Research Unit at Oslo University Hospital

    Oslo, 0372, Norway

  • Sheffield Children's Hospital

    Sheffield, England, S10 2TH, United Kingdom

  • UCSF Benioff Children's Hospital

    Oakland, California, 94609, United States

  • Unidad de Cirugía Artroscopica, Hopsital MIKS

    Vitoria-Gasteiz, 01010, Spain

  • University of Missouri

    Columbia, Missouri, 65201, United States

  • University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

    Madison, Wisconsin, 53705, United States

  • Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine

    Montreal, Quebec, H3T 1C5, Canada

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

More trials for these conditions

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