ACCEL 2/3
Research type
Research Study
Full 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
IRAS ID
1011971
Contact name
Elena Muslimova
Contact email
Sponsor organisation
QED Therapeutics, Inc.
Eudract number
2024-516822-67
Research summary
QED Therapeutics, Inc. (the Sponsor) is running a research study to see if a test medicine not yet approved for market, named infigratinib, will help in the treatment of hypochondroplasia (HCH) and to understand how safe it is for children and young people with HCH.
Hypochondroplasia is a rare genetic (inherited) bone disorder that is believed to occur in one in 15,000 to 40,000 live births. Currently, no drugs targeting the underlying causes of hypochondroplasia have been approved in North America, Asia Pacific, or Europe, though there are a few drugs in development for this condition. Current management of hypochondroplasia focuses on the prevention and treatment of its complications. Children with hypochondroplasia that have already completed at least 26 weeks in the observational QED Therapeutics, Inc. sponsored study – ACCEL (QBGJ398-004) will be invited to take part.
For the Phase 2 portion of the study, children will be randomly assigned to receive either a dose of 0.128 mg/kg/day (Group 1) or a dose of 0.25 mg/kg/day (Group 2) of infigratinib. There is an equal chance of being assigned to either of the groups. In the Phase 2 portion of the study, the child and the child’s study doctor and other study staff will know the dose of study medicine that thechild is taking.
For the Phase 3 portion of the study, the children will be randomly assigned to receive either infigratinib or placebo. A placebo is an inactive treatment that has no therapeutic medical effects, like a sugar pill. There is a 2 out of 3 chance of being assigned to infigratinib (the active medication) and a 1 out of 3 chance of being assigned to placebo (the inactive treatment). In the Phase 3 portion of the study, neither the child nor the child’s study doctor or other study staff will know what treatment the child is receiving.REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
25/EM/0101
Date of REC Opinion
10 Jun 2025
REC opinion
Further Information Favourable Opinion