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New drug shows promise for boosting growth in kids with dwarfism

NCT ID NCT04265651

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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

Summary

This study tested an oral drug called infigratinib in 84 children aged 3 to 11 with achondroplasia, the most common form of dwarfism. The goal was to see if it could safely increase their growth rate. Children took the drug daily, and researchers measured height changes and side effects. While the drug may help improve height, it is not a cure and requires ongoing 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

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

Participants

84 people

The number who actually took part.

Started

Mar 2020

Finished

Oct 2024

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 11 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. Signed informed consent by participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the participant (when applicable). 2. Diagnosis of ACH, documented clinically and confirmed by genetic testing. 3. At least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry. 4. Ambulatory and able to stand without assistance 5. Able to swallow oral medication. Exclusion Criteria: 1. Hypochondroplasia or short stature condition other than ACH. 2. In females, having had their menarche. 3. Height \< -2 or \> +2 standard deviations for age and sex based on reference tables on growth in children with ACH. 4. Significant concurrent disease or condition that, in the view of the Investigator and/or Sponsor, would confound assessment of efficacy or safety of infigratinib. 5. Current evidence of corneal or retinal disorder/keratopathy. 6. History of malignancy. 7. Currently receiving treatment with agents that are known strong inducers or inhibitors of CYP3A4 and medications which increase serum phosphorus and/or calcium concentration. 8. Treatment with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or long-term treatment (\>3 months) at any time. 9. Treatment with a C-type natriuretic peptide (CNP) analog, fibroblast growth factor (FGF) ligand trap, or treatment targeting FGFR inhibition at any time. 10. Regular long-term treatment (\>3 weeks) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable). 11. Treatment with any other investigational product or investigational medical device for the treatment of ACH or short stature. 12. Previous limb-lengthening surgery or guided growth surgery. 13. Fracture within 12 months of screening.

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Conditions

The condition(s) this trial relates to.

achondroplasia Bone Diseases, Developmental 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

  • Birmingham Children's Hospital

    Birmingham, United Kingdom

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Evelina London Children's Hospital

    London, United Kingdom

  • Hopital Femme Mere Enfant

    Lyon, France

  • Hopital Necker-Enfants Malades

    Paris, France

  • Hopital des Enfants

    Toulouse, France

  • Hospital Universitario La Paz

    Madrid, 24086, Spain

  • Hospital Universitario Virgen de la Victoria

    Málaga, Spain

  • Johns Hopkins School of Medicine

    Baltimore, Maryland, 21211, United States

  • Manchester University Children's Hospital

    Manchester, United Kingdom

  • Murdoch Children's Hospital

    Parkville, Victoria, 3052, Australia

  • Nemours Alfred I. Dupont Hospital for Children

    Wilmington, Delaware, 19803, United States

  • Queen Elizabeth University Hospital

    Glasgow, United Kingdom

  • Sheffield Children's Hospital

    Sheffield, England, S10 2TH, United Kingdom

  • Stollery Children's Hospital

    Edmonton, Alberta, T6G 2H7, Canada

  • UCSF Benioff Children's Hospital

    Oakland, California, 94618, United States

  • University Hospitals Bristol and Weston NHS Foundation Trust

    Bristol, BS1 3NU, United Kingdom

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

  • Vithas Hospital San José

    Vitoria-Gasteiz, Álava, 01012, Spain

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