NCT05145010

Extension Study of Infigratinib in Children With Achondroplasia (ACH)

Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL OLE

• Status: Enrolling By Invitation
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 300 (estimated)
• Sponsor: QED Therapeutics, a BridgeBio company · Industry
• Sex: All
• Age: 3 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Quality of Life assessments for this subject population will also be evaluated. Treatment-naïve subjects must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201 and subjects are not otherwise eligible to enroll in another QED-sponsored Phase 2 or Phase 3 ACH study.

Conditions

• Achondroplasia

Interventions

Type	Name	Description
DRUG	Infigratinib	Infigratinib minitablets or sprinkle capsules to be administered by mouth. In subjects that completed a prior study with infigratinib, the starting dose will be the same as the last dose received in the prior interventional study with infigratinib. Infigratinib dose may be adjusted to 0.25 mg/kg/day (the dose selected to be explored further in the dose escalation portion of Phase 2 study QBGJ398-201 (PROPEL 2)).
DRUG	Infigratinib	Infigratinib sprinkle capsules to be administered by mouth. Starting dose for the subjects naïve to Infigratinib will be 0.25 mg/kg/day (the dose selected to be explored further in the dose escalation portion of Phase 2 study QBGJ398-201 (PROPEL 2)).

Timeline

• Start date: 2021-12-06
• Primary completion: 2031-12-01
• Completion: 2032-02-01
• First posted: 2021-12-06
• Last updated: 2025-10-31

Locations

31 sites across 10 countries: United States, Argentina, Australia, Canada, France, Italy, Norway, Singapore, Spain, United Kingdom

Regulatory

• FDA-regulated drug study