NCT07169279

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

Phase 2b, Randomized, Double-Blind, Placebo-Controlled Clinical Trial, Preceded by a Single Ascending Dose Portion and a Phase 2 Open-Label Portion, to Evaluate the Safety and Efficacy of Oral Infigratinib in Infants and Young Children With Achondroplasia

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 77 (estimated)
• Sponsor: QED Therapeutics, a BridgeBio company · Industry
• Sex: All
• Age: 0 Years – 32 Months
• Healthy volunteers: Not accepted

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.

Detailed description

PROPEL Infant \& Toddler (I\&T) is a Phase 2, multicenter, randomized, placebo-controlled study that comprises 4 portions: the single ascending dose (SAD) portion (open-label), the Phase 2 portion (open-label), the Phase 2b portion (placebo-controlled), and an Extension Portion (open-label). The study will evaluate children with ACH \< 3 years old being administered oral infigratinib.

Conditions

• Achondroplasia

Interventions

Type	Name	Description
DRUG	Infigratinib is provided as a single dose of minitablets for oral administration	* The initial cohort dose of infigratinib will begin at the protocol-specified starting dose, with subsequent cohort escalation based on protocol specific criteria. * The target dose is the dose that will provide similar exposure to the dose of 0.25 mg/kg/day in participants 3 years old and older. * The dose and number of minitablets will be calculated based on individual participant age and weight.
DRUG	Infigratinib is provided as sprinkle capsules for daily oral administration	* The cohort dose of infigratinib will be the dose identified in the Single Ascending Dose portion for the age group. * The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
DRUG	Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration	* The cohort dose of infigratinib or placebo will be the dose confirmed in the Phase 2 portion for the age group. * The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
DRUG	Infigratinib is provided as sprinkle capsules for daily oral administration	* The dose of infigratinib will be the dose confirmed in the Phase 2 portion and used in the Phase 2b portion for the age group. * The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months for the first year and every 6 months thereafter.

Timeline

• Start date: 2025-11-19
• Primary completion: 2032-03-01
• Completion: 2032-03-01
• First posted: 2025-09-11
• Last updated: 2026-04-01

Locations

13 sites across 7 countries: United States, Australia, Canada, Norway, Singapore, Spain, United Kingdom

Regulatory

• FDA-regulated drug study