QED Therapeutics, a BridgeBio company
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).
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.
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…
Oral infigratinib 0.128 mg/kg/day
Oral infigratinib 0.25 mg/kg/day