NCT04265651

Study of Infigratinib in Children With Achondroplasia

Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL 2

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 84 (actual)
• Sponsor: QED Therapeutics, a BridgeBio company · Industry
• Sex: All
• Age: 3 Years – 11 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment, and dose expansion. The study also includes a PK Substudy to fully characterize the pharmacokinetics of infigratinib in children with ACH.

Conditions

• Achondroplasia

Interventions

Type	Name	Description
DRUG	Infigratinib 0.016 mg/kg	Initial cohort dose of infigratinib at the protocol-specified starting dose, with subsequent cohort escalations based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
DRUG	Infigratinib 0.032 mg/kg	Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
DRUG	Infigratinib 0.064 mg/kg	Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
DRUG	Infigratinib 0.128 mg/kg	Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
DRUG	Infigratinib 0.25 mg/kg	Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.

Timeline

• Start date: 2020-03-10
• Primary completion: 2024-10-21
• Completion: 2024-10-21
• First posted: 2020-02-11
• Last updated: 2025-10-22

Locations

19 sites across 6 countries: United States, Australia, Canada, France, Spain, United Kingdom

Regulatory

• FDA-regulated drug study