Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

May 21, 2026 updated by: QED Therapeutics, a BridgeBio company

Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Hypochondroplasia: ACCEL OLE

Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with Hypochondroplasia (HCH) who previously completed ACCEL 2/3, and potentially additional participants who completed ACCEL. Participants rolling over directly from the observational ACCEL study must have had at least a 6-month period of growth assessment in that study.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

135

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne
  • Canada
    • Ontario
      • London, Ontario, Canada, N6C 2R5
        • London Health Sciences Centre - Children's Hospital of Western Ontario
      • Ottawa, Ontario, Canada, K1H 8L1
        • Children's Hospital of Eastern Ontario Research Institute
    • Quebec
      • Montreal, Quebec, Canada, H3T 1C5
        • Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
  • France
      • Bron, France, 69677
        • Hopital Femme Mere Enfant
      • Paris, France, 75015
        • Hôpital Universitaire Necker-Enfants Malades
      • Toulouse, France, 31059
        • Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants
  • Norway
      • Bergen, Norway, 5021
        • Haukeland University Hospital
      • Oslo, Norway, 0372
        • Paediatric Clinical Research Unit at Oslo University Hospital
  • Portugal
      • Coimbra, Portugal, 3000-602
        • Hospital Pediátrico de Coimbra
  • Singapore
      • Singapore, Singapore, 229899
        • KK Women's and Children's Hospital
  • Spain
      • Vitoria-Gasteiz, Spain, 01010
        • Unidad de Cirugía Artroscopica, Hopsital MIKS
  • Sweden
      • Solna, Sweden, 17164
        • Astrid Lindgren Children's Hospital
  • United Kingdom
    • England
      • Manchester, England, United Kingdom, M13 9WL
        • Manchester University
      • Sheffield, England, United Kingdom, S10 2TH
        • Sheffield Children's Hospital
    • Scotland
      • Glasgow, Scotland, United Kingdom, G51 4TF
        • Glasgow Clinical Research Facility, Queen Elizabeth University Hospital
  • United States
    • California
      • Oakland, California, United States, 94609
        • UCSF Benioff Children's Hospital
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Childrens Hospital Colorado
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Children's National Hospital
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins School of Medicine
    • Missouri
      • Columbia, Missouri, United States, 65201
        • University of Missouri
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center
    • Wisconsin
      • Madison, Wisconsin, United States, 53705
        • University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Inclusion Criteria for Participants Rolling Over from ACCEL 2/3

    1. Pediatric participants with HCH who have completed ACCEL 2/3
    2. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche

Exclusion Criteria:

  • Exclusion Criteria for Participants Rolling Over from ACCEL 2/3

    1. Participant has concurrent medical condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations
    2. Participants who developed a medical condition that requires the initiation of treatment with a prohibited medication
    3. Participants who prematurely discontinued ACCEL 2/3
    4. Participants who have reached final height or near final height
    5. Current participation in an ongoing clinical study with a sponsor other than QED

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Arm 1: Rollover subjects
Children who have completed QED-sponsored interventional study with infigratinib
Drug: Infigratinib
Infigratinib to be administered by mouth and initiated at the last dose level received in the ACCEL 2/3 study or at the dose selected to be further evaluated after proof-of-concept is established for Phase 2 portion of ACCEL 2/3.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of treatment emergent adverse events (TEAE) and serious TEAE
Time Frame: 10 years
10 years
Changes over time in standing height Z-score in relation to HCH and non-HCH growth charts
Time Frame: 10 years
10 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Changes over time in body proportions
Time Frame: 10 years
10 years
Changes overtime in BMI
Time Frame: 10 years
10 years
Age of puberty onset and time to Tanner stage ≥4
Time Frame: 10 years
10 years
Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)
Time Frame: 10 years
10 years
Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)
Time Frame: 10 years
10 years
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)
Time Frame: 10 years
10 years
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)
Time Frame: 10 years
10 years
Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview
Time Frame: 10 years
10 years
Changes over time in AHV Z-score
Time Frame: 10 years
10 years
Changes over time in weight Z-score
Time Frame: 10 years
10 years
Changes over time in body composition as assessed by DXA (dual x-ray absorptiometry) scans
Time Frame: 10 years
10 years
Changes over time in bone morphology/density by x-ray and DXA
Time Frame: 10 years
10 years
Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test)
Time Frame: 10 years
10 years
Change in attention assessed by age-appropriate computerized tests (Identification Test)
Time Frame: 10 years
10 years
Change in visual learning assessed by age-appropriate computerized tests (One Card Learning Test)
Time Frame: 10 years
10 years
Change in working memory assessed by age-appropriate computerized tests (One Back Test)
Time Frame: 10 years
10 years
Changes over time in severity of epilepsy measured by frequency and adverse event grading
Time Frame: 10 years
10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

QED Therapeutics, a BridgeBio company

Investigators

  • Study Director: QED Therapeutics SVP, Clinical Development, QED Therapeutics, a BridgeBio company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 23, 2026

Primary Completion (Estimated)

May 31, 2036

Study Completion (Estimated)

May 31, 2036

Study Registration Dates

First Submitted

January 19, 2026

First Submitted That Met QC Criteria

January 30, 2026

First Posted (Actual)

February 6, 2026

Study Record Updates

Last Update Posted (Actual)

May 26, 2026

Last Update Submitted That Met QC Criteria

May 21, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • QBGJ398-205

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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