HypochondroplasiaPhase 2NCT07393373
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
A Phase 2 study testing Infigratinib in people with Hypochondroplasia, run by QED Therapeutics, a BridgeBio company at 24 sites in 10 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Hypochondroplasia
- What is being tested?
- Infigratinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 135 participants
- Where
- 24 sitesin 10 countries
- Study dates
- 23 Apr 2026to 31 May 2036
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Inclusion Criteria for Participants Rolling Over from ACCEL 2/3
- Pediatric participants with HCH who have completed ACCEL 2/3
- Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche
Who cannot join
- Exclusion Criteria for Participants Rolling Over from ACCEL 2/3
- Participant has concurrent medical condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations
- Participants who developed a medical condition that requires the initiation of treatment with a prohibited medication
- Participants who prematurely discontinued ACCEL 2/3
- Participants who have reached final height or near final height
- Current participation in an ongoing clinical study with a sponsor other than QED
Trial locations and contacts
24 sites in 10 countries. Contact the study team or a site near you to ask about taking part.
24 sites in 10 countries
United States8 sites
Childrens Hospital Colorado
Aurora, Colorado, 80045
Johns Hopkins School of Medicine
Baltimore, Maryland, 21287
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Missouri
Columbia, Missouri, 65201
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin, 53705
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
UCSF Benioff Children's Hospital
Oakland, California, 94609
Children's National Hospital
Washington D.C., District of Columbia, 20010
Canada3 sites
London Health Sciences Centre - Children's Hospital of Western Ontario
London, Ontario, N6C 2R5
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, H3T 1C5
Children's Hospital of Eastern Ontario Research Institute
Ottawa, Ontario, K1H 8L1
France3 sites
Hôpital Femme Mère Enfant
Bron, 69677
Hôpital Universitaire Necker-Enfants Malades
Paris, 75015
Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants
Toulouse, 31059
United Kingdom3 sites
Glasgow Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, Scotland, G51 4TF
Manchester University
Manchester, England, M13 9WL
Sheffield Children's Hospital
Sheffield, England, S10 2TH
Norway2 sites
Haukeland University Hospital
Bergen, 5021
Paediatric Clinical Research Unit at Oslo University Hospital
Oslo, 0372
Australia1 site
Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne
Parkville, Victoria, 3052
Spain1 site
Unidad de Cirugía Artroscopica, Hopsital MIKS
Vitoria-Gasteiz, 01010
About this study
Official title: 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.
- Conditions
- Hypochondroplasia
- Being tested
- Infigratinib
- Keywords
- skeletal dysplasia, endochondral ossification, hypochondroplasia, HCH, shortened proximal limbs, fibroblast growth factor receptor 3, FGFR3, endochondral bone formation, disproportionate short stature, quality of life, dwarfism, bone diseases, musculoskeletal diseases, osteochondrodysplasia, functional abilities, annualized growth velocity, annualized height velocity, growth, genetic diseases, congenital
- Medical subject headings
- Hypochondroplasia, Mucopolysaccharidosis IV, Dwarfism, Bone Diseases, Musculoskeletal Diseases, Osteochondrodysplasias, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InfigratinibDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1: Rollover subjects | Experimental | Children who have completed QED-sponsored interventional study with infigratinib Receives: Drug: Infigratinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment emergent adverse events (TEAE) and serious TEAE
Measured: 10 years
Changes over time in standing height Z-score in relation to HCH and non-HCH growth charts
Measured: 10 years
Secondary outcomes (17)
Changes over time in AHV Z-score
Measured: 10 years
Changes over time in body proportions
Measured: 10 years
Changes over time in weight Z-score
Measured: 10 years
Changes overtime in BMI
Measured: 10 years
Age of puberty onset and time to Tanner stage ≥4
Measured: 10 years
Changes over time in body composition as assessed by DXA (dual x-ray absorptiometry) scans
Measured: 10 years
Changes over time in bone morphology/density by x-ray and DXA
Measured: 10 years
Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test)
Measured: 10 years
Change in attention assessed by age-appropriate computerized tests (Identification Test)
Measured: 10 years
Change in visual learning assessed by age-appropriate computerized tests (One Card Learning Test)
Measured: 10 years
Change in working memory assessed by age-appropriate computerized tests (One Back Test)
Measured: 10 years
Changes over time in severity of epilepsy measured by frequency and adverse event grading
Measured: 10 years
Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)
Measured: 10 years
Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)
Measured: 10 years
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)
Measured: 10 years
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)
Measured: 10 years
Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2026
- Study start23 Apr 2026
- Primary completion31 May 2036 (estimated)
- Study completion31 May 2036 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HypochondroplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 135 participants (estimated)
Sponsor and investigators
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- QED Therapeutics SVP, Clinical Development, Study director, QED Therapeutics, a BridgeBio company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07393373
- Sponsor's protocol ID
- QBGJ398-205
- Status last verified
- May 2026
- First submitted
- 19 Jan 2026
- Last updated
- 26 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07393373
- Acronym / sponsor ID
- QBGJ398-205
- Conditions
- Hypochondroplasia
- Keywords
- skeletal dysplasia, endochondral ossification, hypochondroplasia, HCH, shortened proximal limbs, fibroblast growth factor receptor 3, FGFR3, endochondral bone formation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 135 participants (estimated)
- Lead sponsor
- QED Therapeutics, a BridgeBio companyIndustry (pharma, biotech, devices)
- Study start
- 23 Apr 2026 (actual)
- Primary completion
- 31 May 2036 (estimated)
- Study completion
- 31 May 2036 (estimated)
- First posted
- 6 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm 1: Rollover subjects | Experimental | Drug: InfigratinibChildren who have completed QED-sponsored interventional study with infigratinib |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Inclusion Criteria for Participants Rolling Over from ACCEL 2/3
Key exclusion criteria (6 listed)
- Exclusion Criteria for Participants Rolling Over from ACCEL 2/3
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| InfigratinibInfigratinib 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. | Drug | Arm 1: Rollover subjects |
Primary outcome
Incidence of treatment emergent adverse events (TEAE) and serious TEAE
Time frame: 10 years
Changes over time in standing height Z-score in relation to HCH and non-HCH growth charts
Time frame: 10 years
17 secondary outcomes
Changes over time in AHV Z-score
Time frame: 10 years
Changes over time in body proportions
Time frame: 10 years
Changes over time in weight Z-score
Time frame: 10 years
Changes overtime in BMI
Time frame: 10 years
Age of puberty onset and time to Tanner stage ≥4
Time frame: 10 years
Changes over time in body composition as assessed by DXA (dual x-ray absorptiometry) scans
Time frame: 10 years
Changes over time in bone morphology/density by x-ray and DXA
Time frame: 10 years
Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test)
Time frame: 10 years
Change in attention assessed by age-appropriate computerized tests (Identification Test)
Time frame: 10 years
Change in visual learning assessed by age-appropriate computerized tests (One Card Learning Test)
Time frame: 10 years
Change in working memory assessed by age-appropriate computerized tests (One Back Test)
Time frame: 10 years
Changes over time in severity of epilepsy measured by frequency and adverse event grading
Time frame: 10 years
Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)
Time frame: 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
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)
Time frame: 10 years
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)
Time frame: 10 years
Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview
Time frame: 10 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
QED Therapeutics SVP, Clinical Development study director
QED Therapeutics, a BridgeBio company
24 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Infigratinib in people with Hypochondroplasia, run by QED Therapeutics, a BridgeBio company at 24 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in United States, Canada, France, United Kingdom, Norway and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment emergent adverse events (TEAE) and serious TEAE (time frame: 10 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2026.