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New oral drug shows promise for helping children with dwarfism grow

NCT ID NCT05145010

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a daily oral drug called infigratinib in 300 children with achondroplasia, the most common form of dwarfism. The drug aims to improve bone growth by targeting a specific protein. Researchers will monitor safety and growth over several years, including children who have taken the drug before and new participants.

What this could mean

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

Active substance
Infigratinib (an oral drug that targets a specific protein to promote bone growth)
What this could lead to
If successful, this could provide a long-term treatment option to improve growth and quality of life for children with achondroplasia.
What could go wrong
This is an early-phase extension study, so results are preliminary. The drug may cause side effects, and not all children may benefit. Long-term safety is still being evaluated.

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 300 people

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

Started

Dec 2021

Expected to finish

Feb 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

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 Rollover Subjects Inclusion Criteria: 1. Pediatric subjects with ACH who have completed a previous QED-sponsored interventional study with infigratinib. 2. Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures. 3. Subjects are able to swallow oral medication. 4. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche. 5. If sexually active, subject must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug. 6. The PI, or a person designated by the PI, will obtain written informed consent from each subject's parent(s), legal guardian(s), or caregiver(s) and the subject's assent, when applicable, before any study-specific activity is performed. Key Rollover Subjects Exclusion Criteria: 1. Subject has concurrent circumstance, disease, or condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations. 2. Subjects who developed a medical condition that will require the initiation of treatment with a prohibited medication. 3. Subjects prematurely discontinued a prior QED-sponsored interventional study with infigratinib 4. Current participation in an ongoing clinical study with a sponsor other than QED 5. Subjects that have reached final height or near final height. Key Inclusion Criteria for Treatment Naïve Subjects 1. Subject must be 3 to \<18 years of age at screening and have growth potential. 2. Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures. 3. Subjects are able to swallow oral medication. 4. Subjects who have a diagnosis of ACH, documented clinically and confirmed by genetic testing. 5. Subjects have at least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398 001) before study entry. 6. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche. 7. If sexually active, subject must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug. 8. The PI, or a person designated by the PI, will obtain written informed consent from each subject's parent(s), legal guardian(s), or caregiver(s) and the subject's assent, when applicable, before any study-specific activity is performed. Key Exclusion Criteria for Treatment Naïve Subjects 1. Subjects who have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia, psychosocial short stature). 2. Subjects who have significant concurrent disease or condition that, in the view of the PI and/or sponsor, would represent an increased risk to the subject or would interfere with study participation or safety evaluations. 3. Subjects who have a history of malignancy. 4. Subjects who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A4. 5. Subjects who discontinued treatment with prohibited medications for at least 5 half-lives before screening are eligible. 6. Subjects who have received treatment with growth hormone, insulin-like growth factor 1 (IGF 1), anabolic steroids or any investigational or approved drug for the treatment of ACH in the previous 6 months. 7. Subjects who have significant abnormality in screening laboratory results. 8. Subjects who have had a fracture within 12 months of screening.

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Conditions

The condition(s) this trial relates to.

achondroplasia bone disorder hereditary 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 places running it

    31 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

  • Birmingham Women's and Children's NHS Foundation Trust

    Birmingham, B5 6NH, United Kingdom

  • Centre Hospitalier Universitaire Sainte-Justine

    Montreal, Quebec, H3T 1C5, Canada

  • Children's Hospital - London Health Sciences Centre

    London, Ontario, N6A 5W9, Canada

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1, Canada

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • Haukeland University Hospital

    Bergen, 5021, Norway

  • Hopital Femme Mere Enfant

    Lyon, 69500, France

  • Hopital Necker-Enfants Malades

    Paris, 75743, France

  • Hopital des Enfants

    Toulouse, 31300, France

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital Universitario Virgen de la Victoria

    Málaga, 29010, Spain

  • Hospital Vithas San José

    Vitoria-Gasteiz, Álava, 01012, Spain

  • Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan

    Ciudad Autonoma Buenos Aires, Buenos Aires, C1245AAM, Argentina

  • Johns Hopkins University

    Baltimore, Maryland, 21205, United States

  • KK Women's and Children's Hosptial

    Singapore, 229899, Singapore

  • Manchester University Children's Hospital

    Manchester, M13 9WL, United Kingdom

  • Murdoch Children's Hospital

    Parkville, Victoria, 3052, Australia

  • Nemours Alfred I. Dupont Hospital for Children

    Wilmington, Delaware, 19803, United States

  • Oslo University Hospital

    Oslo, 0372, Norway

  • Policlinico A. Gemelli IRCCS

    Roma, 00168, Italy

  • Queen Elizabeth University Hospital

    Glasgow, G12 0XH, United Kingdom

  • Sheffield Children's Hospital

    Sheffield, S10 2TH, United Kingdom

  • St. Thomas' Hospital

    London, SE1 7EH, United Kingdom

  • Stollery Children's Hospital

    Edmonton, Alberta, T6C 2B7, Canada

  • USCF Benioff Children's Hospital, Oakland

    Oakland, California, 94609, United States

  • University Hospital and UW Health Clinics

    Madison, Wisconsin, 53705, United States

  • University Hospitals Bristol and Weston NHS Foundation Trust

    Bristol, BS2 8AE, United Kingdom

  • University of Missouri

    Columbia, Missouri, 65212, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

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