An Interventional Study of Infigratinib in Children With Hypochondroplasia (HCH)

July 14, 2026 updated by: QED Therapeutics, a BridgeBio company

A Phase 2/3, Multicenter, Open-Label Phase Followed by a Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children With Hypochondroplasia: ACCEL 2/3

ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib at one of two doses, who have completed at least 26 weeks of participation in QED-sponsored ACCEL (QBGJ398-004).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Enrolling by invitation

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

ACCEL 2/3 is a Phase 2/3 study that comprises of 2 portions. The Phase 2 portion is an open-label, portion in children with HCH aged 5 to 11 years old followed by a Phase 3 portion which is double-blind, placebo-controlled in children with HCH aged >3 years old to <18 years old.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
24

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 3

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 Services 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
    • France
      • Bron, France, France, 69677
        • Hopital Femme Mere Enfant
      • Paris, France, France, 75015
        • Hopital universitaire Necker-Enfants Malades
      • Toulouse, France, France, 31059
        • Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants
  • Norway
    • Norway
      • Bergen, Norway, Norway, 5021
        • Haukeland University Hospital
      • Oslo, Norway, Norway, 0372
        • Paediatric Clinical Research Unit at Oslo University Hospital
  • Portugal
    • Portugal
      • Coimbra, Portugal, Portugal, 3000-602
        • Hospital Pediátrico de Coimbra
  • Singapore
    • Singapore
      • Singapore, Singapore, Singapore, 229899
        • KK Women's and Children's Hospital
  • Spain
    • Spain
      • Vitoria-Gasteiz, Spain, Spain, 01010
        • Unidad de Cirugía Artroscopica, Hopsital MIKS
  • Sweden
    • Sweden
      • Solna, Sweden, 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Participants must have completed at least 26 weeks and still be on the observational study (QBGJ398-004).
  • Phase 2 portion: Participants 5-11 years of age (inclusive).
  • Phase 3 portion: Participants 3 to <18 years of age at screening with growth potential
  • Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
  • Participants are able to swallow oral medication.
  • Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
  • Participants are ambulatory and able to stand without assistance. Sex and Contraceptive/Barrier Requirements
  • Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
  • If sexually active, participants whether male or female, must be willing to use a highly effective method of contraception, as relevant, while taking study drug and for 1 month after the last dose of study drug.
  • Signed informed consent.

Key Exclusion Criteria:

  • Participants who have ACH or a short stature condition other than HCH.
  • Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib.
  • Current evidence of clinically significant corneal or retinal disorder/keratopathy confirmed by ophthalmic examination.
  • Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations.
  • History and/or current evidence of extensive ectopic tissue calcification.
  • History of malignancy.
  • Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH, HCH, or short stature.
  • Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid.
  • Previous limb-lengthening surgery at any time or planned/expected to have limb-lengthening or guided growth surgery while participating in the study.
  • Participants receiving medications which could increase serum phosphorus and/or calcium concentrations
  • Clinically significant abnormality in any laboratory test result at screening.
  • Pregnant or breastfeeding at the screening visit or planning to become pregnant (self or partner) at any time during the study.
  • Allergy to any components of the study drug.
  • Concurrent circumstance, disease, or condition that would interfere with study participation.

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: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Phase 2 Cohort 1
Oral infigratinib 0.128 mg/kg/day
Drug: infigratinib 0.128 mg/kg/day
Oral infigratinib 0.128 mg/kg/day
Experimental: Phase 2 Cohort 2
Oral infigratinib 0.25 mg/kg/day
Drug: infigratinib 0.25 mg/kg/day
Oral infigratinib 0.25 mg/kg/day

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Change from baseline (BL) in Annualized Height Velocity (AHV; cm/year)
Time Frame: 26 weeks
26 weeks
Incidence, severity, and seriousness of adverse events (AEs) that require dose reduction or discontinuation
Time Frame: at least 26 weeks
at least 26 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Changes in cognitive function (as assessed by age appropriate computerized tests)
Time Frame: 52 weeks
52 weeks
Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)
Time Frame: 52 weeks
52 weeks
Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)
Time Frame: 52 weeks
52 weeks
Change from BL in collagen X marker (CXM) levels
Time Frame: 52 weeks
52 weeks
Change from BL in height Z-score (in relation to both HCH and average height tables)
Time Frame: 52 weeks
52 weeks
Change from BL in upper to lower body segment ratio (cm)
Time Frame: 52 weeks
52 weeks

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Time Frame
Change from BL in comorbidities associated with HCH
Time Frame: 52 weeks
52 weeks
Change from BL in Health-related Quality of Life (HRQoL) (as assessed by Pediatric Quality of Life Inventory [PedsQL tool])
Time Frame: 52 weeks
52 weeks
Evaluate treatment benefit as assessed by qualitative interviews
Time Frame: 52 weeks
52 weeks

Collaborators and Investigators

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

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 22, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 18, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 6, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 12, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 16, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 14, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • QBGJ398-304

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

product manufactured in and exported from the U.S.

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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