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, of who have completed at least 26 weeks of participation in QED-sponsored ACCEL (QBGJ398-004).
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
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
24
Oral infigratinib 0.128 mg/kg/day
Oral infigratinib 0.25 mg/kg/day
UCSF Benioff Children's Hospital
Oakland, California, United States
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
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
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Aurora, Colorado, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
Johns Hopkins School of Medicine
Baltimore, Maryland, United States
University of Missouri
Columbia, Missouri, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
London Health Services Center - Children's Hospital of Western Ontario
London, Ontario, Canada
Children's Hospital of Eastern Ontario Research Institute
Ottawa, Ontario, Canada
...and 12 more locations