Study of Infigratinib in Children With Achondroplasia

Last updated: October 19, 2025
Sponsor: QED Therapeutics, Inc., a Bridgebio company
Overall Status: Completed

Phase

2

Condition

Severe Short Stature

Bone Diseases

Musculoskeletal Diseases

Treatment

Infigratinib 0.032 mg/kg

Infigratinib 0.016 mg/kg

Infigratinib 0.064 mg/kg

Clinical Study ID

NCT04265651
QBGJ398-201
  • Ages 3-11
  • All Genders

Study Summary

This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment, and dose expansion. The study also includes a PK Substudy to fully characterize the pharmacokinetics of infigratinib in children with ACH.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Signed informed consent by participant or parent(s) or legally authorizedrepresentative (LAR) and signed informed assent by the participant (whenapplicable).

  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 (ProtocolQBGJ398-001) before study entry.

  4. Ambulatory and able to stand without assistance

  5. Able to swallow oral medication.

Exclusion

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 ongrowth in children with ACH.

  4. Significant concurrent disease or condition that, in the view of the Investigatorand/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 orinhibitors of CYP3A4 and medications which increase serum phosphorus and/or calciumconcentration.

  8. Treatment with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolicsteroids 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 ongoinginhaled steroid for asthma is acceptable).

  11. Treatment with any other investigational product or investigational medical devicefor the treatment of ACH or short stature.

  12. Previous limb-lengthening surgery or guided growth surgery.

  13. Fracture within 12 months of screening.

Study Design

Total Participants: 84
Treatment Group(s): 5
Primary Treatment: Infigratinib 0.032 mg/kg
Phase: 2
Study Start date:
March 10, 2020
Estimated Completion Date:
October 21, 2024

Connect with a study center

  • Murdoch Children's Hospital

    Parkville, Victoria 3052
    Australia

    Site Not Available

  • Murdoch Children's Hospital

    Parkville 2153770, Victoria 2145234 3052
    Australia

    Site Not Available

  • Stollery Children's Hospital

    Edmonton, Alberta T6G 2H7
    Canada

    Site Not Available

  • Stollery Children's Hospital

    Edmonton 5946768, Alberta 5883102 T6G 2H7
    Canada

    Site Not Available

  • Hopital Femme Mere Enfant

    Lyon,
    France

    Site Not Available

  • Hopital Femme Mere Enfant

    Lyon 2996944,
    France

    Site Not Available

  • Hopital Necker-Enfants Malades

    Paris,
    France

    Site Not Available

  • Hopital Necker-Enfants Malades

    Paris 2988507,
    France

    Site Not Available

  • Hopital des Enfants

    Toulouse,
    France

    Site Not Available

  • Hopital des Enfants

    Toulouse 2972315,
    France

    Site Not Available

  • Vithas Hospital San José

    Vitoria-Gasteiz, Álava 01012
    Spain

    Site Not Available

  • Vithas Hospital San José

    Vitoria-Gasteiz 3104499, Álava 01012
    Spain

    Site Not Available

  • Hospital Universitario La Paz

    Madrid, 24086
    Spain

    Site Not Available

  • Hospital Universitario La Paz

    Madrid 3117735, 24086
    Spain

    Site Not Available

  • Hospital Universitario Virgen de la Victoria

    Málaga,
    Spain

    Site Not Available

  • Hospital Universitario Virgen de la Victoria

    Málaga 2514256,
    Spain

    Site Not Available

  • Sheffield Children's Hospital

    Sheffield, England S10 2TH
    United Kingdom

    Site Not Available

  • Sheffield Children's Hospital

    Sheffield 2638077, England 6269131 S10 2TH
    United Kingdom

    Site Not Available

  • Birmingham Children's Hospital

    Birmingham,
    United Kingdom

    Site Not Available

  • Birmingham Children's Hospital

    Birmingham 2655603,
    United Kingdom

    Site Not Available

  • University Hospitals Bristol and Weston NHS Foundation Trust

    Bristol, BS1 3NU
    United Kingdom

    Site Not Available

  • University Hospitals Bristol and Weston NHS Foundation Trust

    Bristol 2654675, BS1 3NU
    United Kingdom

    Site Not Available

  • Queen Elizabeth University Hospital

    Glasgow,
    United Kingdom

    Site Not Available

  • Queen Elizabeth University Hospital

    Glasgow 2648579,
    United Kingdom

    Site Not Available

  • Evelina London Children's Hospital

    London,
    United Kingdom

    Site Not Available

  • Evelina London Children's Hospital

    London 2643743,
    United Kingdom

    Site Not Available

  • Manchester University Children's Hospital

    Manchester,
    United Kingdom

    Site Not Available

  • Manchester University Children's Hospital

    Manchester 2643123,
    United Kingdom

    Site Not Available

  • UCSF Benioff Children's Hospital

    Oakland, California 94618
    United States

    Site Not Available

  • UCSF Benioff Children's Hospital

    Oakland 5378538, California 5332921 94618
    United States

    Site Not Available

  • Nemours Alfred I. Dupont Hospital for Children

    Wilmington, Delaware 19803
    United States

    Site Not Available

  • Nemours Alfred I. Dupont Hospital for Children

    Wilmington 4145381, Delaware 4142224 19803
    United States

    Site Not Available

  • Johns Hopkins School of Medicine

    Baltimore, Maryland 21211
    United States

    Site Not Available

  • Johns Hopkins School of Medicine

    Baltimore 4347778, Maryland 4361885 21211
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati 4508722, Ohio 5165418 45229
    United States

    Site Not Available

  • Vanderbilt University Medical Center

    Nashville, Tennessee 37232
    United States

    Site Not Available

  • Vanderbilt University Medical Center

    Nashville 4644585, Tennessee 4662168 37232
    United States

    Site Not Available

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