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Could a daily pill boost growth in kids with hypochondroplasia?

NCT ID NCT06873035

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 17, 2026 · Updated 2 times

Summary

This study tests an oral drug called infigratinib in children with hypochondroplasia, a condition that causes short stature. The trial has two parts: first, all children receive the drug to see if it safely increases their growth rate; then, some will switch to a placebo to confirm the effect. Up to 24 children aged 3 to 11 will take part.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
infigratinib (oral drug)
What this could lead to
If successful, this could provide a treatment option to improve growth in children with hypochondroplasia.
What could go wrong
This is an early-to-mid-stage trial with only 24 participants. The drug may not work as hoped, and side effects could require dose changes or stopping treatment.

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 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 24 people

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

Started

Apr 2025

Expected to finish

Jan 2027

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.

Key Inclusion Criteria: * Participants must have completed at least 26 weeks and still be on the observational study (QBGJ398-004). * Phase 2 portion: Participants 5-11 years of age (inclusive). * Phase 3 portion: Participants 3 to \<18 years of age at screening with growth potential * Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test. * Participants are able to swallow oral medication. * Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures. * Participants are ambulatory and able to stand without assistance. Sex and Contraceptive/Barrier Requirements * Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche. * If sexually active, participants whether male or female, must be willing to use a highly effective method of contraception, as relevant, while taking study drug and for 1 month after the last dose of study drug. * Signed informed consent. Key Exclusion Criteria: * Participants who have ACH or a short stature condition other than HCH. * Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib. * Current evidence of clinically significant corneal or retinal disorder/keratopathy confirmed by ophthalmic examination. * Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations. * History and/or current evidence of extensive ectopic tissue calcification. * History of malignancy. * Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH, HCH, or short stature. * Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid. * Previous limb-lengthening surgery at any time or planned/expected to have limb-lengthening or guided growth surgery while participating in the study. * Participants receiving medications which could increase serum phosphorus and/or calcium concentrations * Clinically significant abnormality in any laboratory test result at screening. * Pregnant or breastfeeding at the screening visit or planning to become pregnant (self or partner) at any time during the study. * Allergy to any components of the study drug. * Concurrent circumstance, disease, or condition that would interfere with study participation.

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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, Sweden, 17164, Sweden

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

    Toulouse, France, 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, Norway, 5021, Norway

  • Hospital Pediátrico de Coimbra

    Coimbra, Portugal, 3000-602, Portugal

  • Hôpital Femme Mère Enfant

    Bron, France, 69677, France

  • Hôpital Universitaire Necker-Enfants Malades

    Paris, France, 75015, France

  • Johns Hopkins School of Medicine

    Baltimore, Maryland, 21287, United States

  • KK Women's and Children's Hospital

    Singapore, Singapore, 229899, Singapore

  • London Health Services 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, Norway, 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, Spain, 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

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