Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

Condition(s) studied

Investigational drug(s) / intervention(s)

Infigratinib: Infigratinib to be administered by mouth and initiated at the last dose level received in the ACCEL 2/3 study or at the dose selected to be further evaluated after proof-of-concept is established for Phase 2 portion of ACCEL 2/3.

Study summary

Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with Hypochondroplasia (HCH) who previously completed ACCEL 2/3, and potentially additional participants who completed ACCEL. Participants rolling over directly from the observational ACCEL study must have had at least a 6-month period of growth assessment in that study.

Eligibility

Primary outcome measure(s)

• Incidence of treatment emergent adverse events (TEAE) and serious TEAE — 10 years
• Changes over time in standing height Z-score in relation to HCH and non-HCH growth charts — 10 years

Trial sites (24)