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New pill could help kids with dwarfism grow better

NCT ID NCT07169279

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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jul 14, 2026 · Updated 3 times

Summary

This study tests an oral drug called infigratinib in infants and children under 3 years old with achondroplasia, the most common form of dwarfism. The goal is to see if it safely improves growth and body proportions. The trial has several phases to find the right dose and then compare the drug to a placebo over time.

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 daily oral treatment to improve growth and body proportions in young children with achondroplasia.
What could go wrong
This is an early Phase 2 trial with only 77 participants, so results may not confirm benefit. Side effects are still being studied, and the drug requires daily dosing.

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 77 people

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

Started

Nov 2025

Expected to finish

Mar 2032

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

0 years to 32 months

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: * Diagnosis of ACH confirmed by genetic testing. If prospective participants had prior genetic testing, the diagnosis must be confirmed by a report from a certified laboratory, documenting the specific mutation. * Age 0 to 32 months (2 years and 8 months) at screening. * Signed informed consent, which must be obtained from each participant's parent(s) or legal guardian. * Parent(s)/Guardian(s) willing and able to attend all study visits and comply with all study requirements. * Parent(s)/Guardian(s) willing and able to comply with the routine care of the study participants according to local guidance for the management of infants and young children with ACH. * Able to swallow age-appropriate oral medication. * In participants \<1 year old, be compliant with recommended vitamin D supplementation of 5 10 μg/day or higher (or as recommended by country specific guidelines). Exclusion Criteria: * Participants who have hypochondroplasia or diagnosis of genetic condition other than ACH, or any clinical condition that can affect growth. * Gestational age at birth \<37 weeks and/or birth weight \<2500 grams. * Gastroesophageal reflux disease requiring prolonged treatment (\>1 week) with prohibited medications. * Evidence of cervicomedullary compression, as defined by an Achondroplasia Foramen Magnum Score (AFMS) 4, symptomatic or asymptomatic, diagnosed during MRI done at screening or a previous MRI done at any time if the participant had not undergone decompression surgery. * History of fracture of a long bone or spine within 6 months prior to screening. * Any other significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib and/or would require treatment with a prohibited medication (per protocol), and/or would place the participant at high risk for poor treatment compliance or for failure to complete the study. * Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH or short stature, including (but not limited to) r-hGH, IGF-1, CNP analog, FGF ligand trap, or treatment targeting FGFR inhibition at any time. * Regular long-term (\>3 weeks; more than twice/year) treatment with supraphysiologic doses of glucocorticoid therapy (ie, \>15 mg/m2/day of hydrocortisone or equivalent) or treatment with glucocorticoids at anti-inflammatory doses (for over 3 weeks within 6 months of the screening visit. NOTE: Low-dose topical, inhaled, or intranasal corticosteroids are acceptable. * Significant abnormality in screening laboratory results, * Allergy or hypersensitivity to any components of the study drug.

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Conditions

The condition(s) this trial relates to.

achondroplasia bone disorder Dwarfism hereditary endocrine growth disease Mucopolysaccharidosis IV musculoskeletal system disorder osteochondrodysplasia

As listed by the trial registrant

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

Achondroplasia

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

    12 sites in 7 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

  • Children's Hospital of Eastern Ontario Research Institute

    RECRUITING

    Ottawa, Ontario, K1H 8L1, Canada

  • Johns Hopkins University

    RECRUITING

    Baltimore, Maryland, 21218, United States

  • KK Women's and Children's Hospital

    RECRUITING

    Singapore, 2298899, Singapore

  • Manchester University NHS Foundation Trust

    RECRUITING

    Manchester, M13 9WL, United Kingdom

  • Murdoch Children's Research Institute

    RECRUITING

    Parkville, Victoria, 3052, Australia

  • NHS Greater Glasgow and Clyde

    RECRUITING

    Glasgow, G3 8SJ, United Kingdom

  • Paediatric Clinical Research Unit at Oslo University Hospital

    RECRUITING

    Oslo, 0372, Norway

  • Sheffield Children's NHS Foundation Trust

    RECRUITING

    Sheffield, S10 2TH, United Kingdom

  • UCSF Benioff Children's Hospital

    RECRUITING

    Oakland, California, 94609, United States

  • Unidad de Cirugía Artroscópica (UCA)

    RECRUITING

    Vitoria-Gasteiz, 01010, Spain

  • 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

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