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New drug shows promise for boosting growth in kids with dwarfism

NCT ID NCT06164951

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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

Summary

This study tested a daily oral drug called infigratinib in 114 children aged 3 to 18 with achondroplasia, the most common form of dwarfism. The goal was to see if it could safely increase their growth rate compared to a placebo. The trial is now complete, and results will show whether this drug can become a new treatment option.

What this could mean

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

Active substance
Infigratinib (a daily oral drug)
What this could lead to
If it works, this could become a new treatment to help children with achondroplasia grow taller.
What could go wrong
This is a completed Phase 3 trial, but results are not yet public. The drug may not work better than placebo, and side effects are possible.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

114 people

The number who actually took part.

Started

Nov 2023

Finished

Dec 2025

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 17 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: 1. Subject must be 3 to \<18 years of age at screening with growth potential defined as annualized height velocity of \>1.5 cm/year over a period of at least 6 months of participation in the PROPEL observational study (QBGJ398-001), pubertal Tanner stage ≤4, and bone age ≤13 years in females and ≤15 years in males. Type of Subject and Disease Characteristics 2. Subjects who have a diagnosis of ACH that has been documented clinically and confirmed by genetic testing. 3. Subjects must have completed at least 26 weeks in the PROPEL (QBGJ398-001) study before screening. 4. Subjects are able to swallow oral medication. 5. Subjects and parent(s), legal guardian(s), or caregivers are willing and able to comply with study visits and study procedures. 6. Subjects are ambulatory and able to stand without assistance. Sex and Contraceptive/Barrier Requirements 7. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche. 8. If sexually active, subjects, whether male or female, must be willing to use a highly effective method of contraception while taking study drug and for 3 months after the last dose of study drug. Informed Consent 9. Signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol, must be obtained for each subject from their parent(s) or legal guardian and signed informed consent/assent must be obtained from the subject (when applicable) Exclusion Criteria: Medical Conditions 1. Subjects who have hypochondroplasia or short stature condition other than ACH. 2. Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib. 3. Current evidence of clinically significant corneal or retinal disorder/keratopathy -confirmed by ophthalmic examination. 4. Concurrent circumstance, disease or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations and/or would require treatment with a prohibited medication, and/or would place the subject at high risk for poor treatment compliance or for not completing the study. 5. History and/or current evidence of extensive ectopic tissue calcification. 6. History of malignancy. Prior/Concomitant Therapy 7. Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH or short stature. 8. Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid therapy (ie, \>15 mg/m2/day of hydrocortisone or equivalent) or treatment with glucocorticoids at anti-inflammatory doses (ie, 2.5-10 mg/kg/day of hydrocortisone or equivalent) for over 3 weeks within 6 months of the screening visit (low-dose local preparations including inhaled steroid for asthma, intranasal sprays for allergies, and topical steroids are allowed). 9. Previous limb-lengthening surgery at any time or planned/expected to have limb-lengthening surgery or guided growth surgery during the study period. Guided growth surgery with plates removed at least 12 months prior to screening is allowed. 10. Currently receiving treatment with agents that are known strong inducers or inhibitors of CYP3A4 or prolonged treatment (\>1 week) with medications that alter the pH of the gastrointestinal tract including antacids, H2 antagonists (eg, ranitidine, famotidine), and proton-pump inhibitors (eg, omeprazole). 11. Current evidence of endocrine alterations of calcium/phosphorus homeostasis. Diagnostic assessments 12. Subjects who have significant abnormality in screening laboratory results. Other Exclusions 13. Having had a fracture of the long bones (ie, extremities) or spine within 12 months prior to screening. 14. Pregnant or breastfeeding at the screening visit or planning to become pregnant (self or partner) at any time during the study. 15. 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 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

Contacts and locations

Locations

  • QED Investigative Site

    San Francisco, California, 94609, United States

  • QED Investigative Site

    Aurora, Colorado, 80045, United States

  • QED Investigative Site

    Baltimore, Maryland, 21287, United States

  • QED Investigative Site

    Columbia, Missouri, 65212, United States

  • QED Investigative Site

    Cincinnati, Ohio, 45229, United States

  • QED Investigative Site

    Nashville, Tennessee, 37232, United States

  • QED Investigative Site

    Madison, Wisconsin, 53792, United States

  • QED Investigative Site

    Buenos Aires, Buenos Aires F.D., C1245AAM, Argentina

  • QED Investigative Site

    Parkville, Victoria, 3052, Australia

  • QED Investigative Site

    Edmonton, Alberta, T6G 2B7, Canada

  • QED Investigative Site

    London, Ontario, N6A 5W9, Canada

  • QED Investigative Site

    Ottawa, Ontario, K1H 8L1, Canada

  • QED Investigative Site

    Montreal, Quebec, H3T 1C5, Canada

  • QED Investigative Site

    Bron, 69677, France

  • QED Investigative Site

    Paris, 75015, France

  • QED Investigative Site

    Toulouse, 31059, France

  • QED Investigative Site

    Rome, 00168, Italy

  • QED Investigative Site

    Bergen, 5009, Norway

  • QED Investigative Site

    Oslo, 0372, Norway

  • QED Investigative Site

    Singapore, 229899, Singapore

  • QED Investigative Site

    Málaga, 29010, Spain

  • QED Investigative Site

    Vitoria-Gasteiz, 01008, Spain

  • QED Investigative Site

    Bristol, BS2 8BJ, United Kingdom

  • QED Investigative Site

    Glasgow, G51 4TF, United Kingdom

  • QED Investigative Site

    London, SE1 7EH, United Kingdom

  • QED Investigative Site

    Manchester, M13 9WL, United Kingdom

  • QED Investigative Site

    Sheffield, S10 2TH, United Kingdom

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