Infigratinib for Dwarfism
(HCH Trial)
Trial Summary
What is the purpose of this trial?
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).
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are taking medications that could increase serum phosphorus or calcium levels, or if you are on long-term high doses of glucocorticoids.
What data supports the effectiveness of the drug Infigratinib for treating dwarfism?
Research shows that Infigratinib, a drug that targets specific growth factor receptors, has been effective in improving bone growth in animal models of achondroplasia, a common form of dwarfism. This suggests it may help counteract the overactivity of growth factor receptors that cause the condition.12345
Is Infigratinib safe for humans?
How does the drug Infigratinib differ from other treatments for dwarfism?
Infigratinib is unique because it directly targets the overactive FGFR3 gene, which is responsible for achondroplasia, a common form of dwarfism. Unlike most treatments that only address symptoms, Infigratinib is an oral medication that aims to counteract the underlying genetic cause of the condition.12368
Eligibility Criteria
This trial is for children with Hypochondroplasia (HCH), a form of dwarfism that affects bone growth. Participants must have completed at least 26 weeks in a prior ACCEL study and are now continuing treatment to assess further effects.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Phase 2 Treatment
Open-label treatment with infigratinib in children with hypochondroplasia, receiving either 0.128 mg/kg/day or 0.25 mg/kg/day
Phase 3 Treatment
Double-blind, placebo-controlled treatment with infigratinib in children with hypochondroplasia
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Infigratinib
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Who Is Running the Clinical Trial?
QED Therapeutics, Inc.
Lead Sponsor