Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
Start Date
11/19/2025
Completion Date
3/1/2032
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.
Eligibility Criteria
Age Range: No minimum to No maximum
Interventions
Infigratinib is provided as a single dose of minitablets for oral administration
Infigratinib is provided as sprinkle capsules for daily oral administration
Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
Infigratinib is provided as sprinkle capsules for daily oral administration
Conditions
Locations
UCSF Benioff Children's Hospital
Oakland, California 94609
United States
Johns Hopkins University
Baltimore, Maryland 21218
United States
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin 53705
United States
Murdoch Children's Research Institute
Parkville, Victoria 3052
Australia
Children's Hospital of Eastern Ontario Research Institute
Ottawa, Ontario K1H 8L1
Canada
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec H3T 1C5
Canada
Paediatric Clinical Research Unit at Oslo University Hospital
Oslo, 0372
Norway
KK Women's and Children's Hospital
Singapore, 2298899
Singapore
Unidad de Cirugía Artroscópica (UCA)
Vitoria-Gasteiz, 01010
Spain
NHS Greater Glasgow and Clyde
Glasgow, G3 8SJ
United Kingdom
Manchester University NHS Foundation Trust
Manchester, M13 9WL
United Kingdom
Sheffield Children's NHS Foundation Trust
Sheffield, S10 2TH
United Kingdom