HypochondroplasiaPhase 2/3HCHNCT06873035
An Interventional Study of Infigratinib in Children With Hypochondroplasia
A Phase 2/3 study testing infigratinib 0.128 mg/kg/day and infigratinib 0.25 mg/kg/day in people with Hypochondroplasia, run by QED Therapeutics, a BridgeBio company at 24 sites in 10 countries.
Trial summary
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… 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 0.128 mg/kg/day and infigratinib 0.25 mg/kg/day
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 24 sitesin 10 countries
- Study dates
- 22 Apr 2025to Jan 2027
- Phase
- Phase 2/3
- 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
- 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.
Show all 10Show fewer
- 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.
Who cannot join
- 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.
Show all 14Show fewer
- 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.
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 Services 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, France, 69677
Hôpital Universitaire Necker-Enfants Malades
Paris, France, 75015
Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants
Toulouse, France, 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, Norway, 5021
Paediatric Clinical Research Unit at Oslo University Hospital
Oslo, Norway, 0372
Australia1 site
Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne
Parkville, Victoria, 3052
Singapore1 site
KK Women's and Children's Hospital
Singapore, Singapore, 229899
Spain1 site
Unidad de Cirugía Artroscopica, Hopsital MIKS
Vitoria-Gasteiz, Spain, 01010
About this study
Official title: 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).
Read the 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.
- Conditions
- Hypochondroplasia
- Being tested
- infigratinib 0.128 mg/kg/day, infigratinib 0.25 mg/kg/day
- 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.
infigratinib 0.128 mg/kg/dayDrug
Oral infigratinib 0.128 mg/kg/day
infigratinib 0.25 mg/kg/dayDrug
Oral infigratinib 0.25 mg/kg/day
| Study group | Role | What they receive |
|---|---|---|
| Phase 2 Cohort 1 | Experimental | Oral infigratinib 0.128 mg/kg/day Receives: Drug: infigratinib 0.128 mg/kg/day |
| Phase 2 Cohort 2 | Experimental | Oral infigratinib 0.25 mg/kg/day Receives: Drug: infigratinib 0.25 mg/kg/day |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline (BL) in Annualized Height Velocity (AHV; cm/year)
Measured: 26 weeks
Incidence, severity, and seriousness of adverse events (AEs) that require dose reduction or discontinuation
Measured: at least 26 weeks
Secondary outcomes (6)
Change from BL in height Z-score (in relation to both HCH and average height tables)
Measured: 52 weeks
Change from BL in upper to lower body segment ratio (cm)
Measured: 52 weeks
Changes in cognitive function (as assessed by age appropriate computerized tests)
Measured: 52 weeks
Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)
Measured: 52 weeks
Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)
Measured: 52 weeks
Change from BL in collagen X marker (CXM) levels
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2025
- Study start22 Apr 2025
- Primary completionJan 2027 (estimated)
- Study completionJan 2027 (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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This study comprises an open-label Phase 2 portion to evaluate safety and efficacy in participants receiving infigratinib at one of two doses followed by a Phase 3 randomized, double-blind portion of the dose selected from the Phase 2 portion versus placebo.
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06873035
- Sponsor's protocol ID
- QBGJ398-304
- Acronym
- HCH
- Status last verified
- May 2026
- First submitted
- 18 Feb 2025
- Last updated
- 16 Jul 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 16 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06873035
- Acronym / sponsor ID
- HCH / QBGJ398-304
- Conditions
- Hypochondroplasia
- Keywords
- skeletal dysplasia, endochondral ossification, hypochondroplasia, HCH, shortened proximal limbs, fibroblast growth factor receptor 3, FGFR3, endochondral bone formation
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- QED Therapeutics, a BridgeBio companyIndustry (pharma, biotech, devices)
- Study start
- 22 Apr 2025 (actual)
- Primary completion
- Jan 2027 (estimated)
- Study completion
- Jan 2027 (estimated)
- First posted
- 12 Mar 2025 (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.
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).
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- This study comprises an open-label Phase 2 portion to evaluate safety and efficacy in participants receiving infigratinib at one of two doses followed by a Phase 3 randomized, double-blind portion of the dose selected from the Phase 2 portion versus placebo.
| Arm | Type | Receives |
|---|---|---|
| Phase 2 Cohort 1 | Experimental | Drug: infigratinib 0.128 mg/kg/dayOral infigratinib 0.128 mg/kg/day |
| Phase 2 Cohort 2 | Experimental | Drug: infigratinib 0.25 mg/kg/dayOral infigratinib 0.25 mg/kg/day |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- 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.
Key exclusion criteria (14 listed)
- 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.
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 |
|---|---|---|
| infigratinib 0.128 mg/kg/dayOral infigratinib 0.128 mg/kg/day | Drug | Phase 2 Cohort 1 |
| infigratinib 0.25 mg/kg/dayOral infigratinib 0.25 mg/kg/day | Drug | Phase 2 Cohort 2 |
Primary outcome
Change from baseline (BL) in Annualized Height Velocity (AHV; cm/year)
Time frame: 26 weeks
Incidence, severity, and seriousness of adverse events (AEs) that require dose reduction or discontinuation
Time frame: at least 26 weeks
6 secondary outcomes
Change from BL in height Z-score (in relation to both HCH and average height tables)
Time frame: 52 weeks
Change from BL in upper to lower body segment ratio (cm)
Time frame: 52 weeks
Changes in cognitive function (as assessed by age appropriate computerized tests)
Time frame: 52 weeks
Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)
Time frame: 52 weeks
Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)
Time frame: 52 weeks
Change from BL in collagen X marker (CXM) levels
Time frame: 52 weeks
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 16 Jul 2026.
- Individual participant dataSharing planned: No
Related trials for Hypochondroplasia
Not yet recruitingPhase 3
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide Versus Placebo in Children and Adolescents With Hypochondroplasia
Hypochondroplasia
RecruitingObservational study
Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
Achondroplasia, Hypochondroplasia
Enrolling by invitationPhase 3
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
Hypochondroplasia
Enrolling by invitationPhase 2
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
Hypochondroplasia
RecruitingObservational study
Prospective Clinical Assessment Study in Children With Hypochondroplasia
Hypochondroplasia
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.
The record lists no contact. Use the official record to reach the sponsor.
24 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing infigratinib 0.128 mg/kg/day and infigratinib 0.25 mg/kg/day 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 10 inclusion and 14 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 16 Jul 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Change from baseline (BL) in Annualized Height Velocity (AHV; cm/year) (time frame: 26 weeks). 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 16 Jul 2026.