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).
Eligibility
Sex
ALL
Min age
3 Years
Max age
18 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Change from baseline (BL) in Annualized Height Velocity (AHV; cm/year) — 26 weeks
Incidence, severity, and seriousness of adverse events (AEs) that require dose reduction or discontinuation — at least 26 weeks
Trial sites (24)
Facility
City
Region
Status
UCSF Benioff Children's Hospital
Oakland
California
Childrens Hospital Colorado
Aurora
Colorado
Children's National Hospital
Washington D.C.
District of Columbia
Johns Hopkins School of Medicine
Baltimore
Maryland
University of Missouri
Columbia
Missouri
Cincinnati Children's Hospital Medical Center
Cincinnati
Ohio
Vanderbilt University Medical Center
Nashville
Tennessee
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison
Wisconsin
Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne
Parkville
Victoria
London Health Services Centre - Children's Hospital of Western Ontario
London
Ontario
Children's Hospital of Eastern Ontario Research Institute
Ottawa
Ontario
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
Montreal
Quebec
Hôpital Femme Mère Enfant
Bron
France
Hôpital Universitaire Necker-Enfants Malades
Paris
France
Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants
Toulouse
France
Haukeland University Hospital
Bergen
Norway
Paediatric Clinical Research Unit at Oslo University Hospital
Oslo
Norway
Hospital Pediátrico de Coimbra
Coimbra
Portugal
KK Women's and Children's Hospital
Singapore
Singapore
Unidad de Cirugía Artroscopica, Hopsital MIKS
Vitoria-Gasteiz
Spain
Astrid Lindgren Children's Hospital
Solna
Sweden
Manchester University
Manchester
England
Sheffield Children's Hospital
Sheffield
England
Glasgow Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow
Scotland
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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