Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

Summary

This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \< 3 years old with ACH. The purposes of the SAD and Phase 2 portions are to identify and confirm the dose of infigratinib to be used in the Phase 2b portion, based on safety and PK. The purpose of the Phase 2b, placebo-controlled portion is to evaluate the safety and efficacy of infigratinib in children \< 3 years old with ACH at the selected dose.

Timeline

Start

2025-11-19

Primary completion

2030-03

Completion

2032-03

Drugs

Indications