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

PHASE2Not yet recruitingINTERVENTIONAL
Enrollment

77

Participants

Timeline

Start Date

October 31, 2025

Primary Completion Date

March 31, 2032

Study Completion Date

March 31, 2032

Conditions
Achondroplasia
Interventions
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."

Trial Locations (13)

3052

Murdoch Children's Research Institute, Parkville

21218

Johns Hopkins University, Baltimore

53705

University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic, Madison

94609

UCSF Benioff Children's Hospital, Oakland

2298899

KK Women's and Children's Hospital, Singapore

K1H 8L1

Children's Hospital of Eastern Ontario Research Institute, Ottawa

H3T 1C5

Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine, Montreal

0372

Paediatric Clinical Research Unit at Oslo University Hospital, Oslo

01010

Unidad de Cirugia Artroscopica (UCA), Vitoria-Gasteiz

G3 8SJ

NHS Greater Glasgow and Clyde, Glasgow

SE1 7EH

Guy's and Saint Thomas' NHS Foundation Trust, London

M13 9WL

Manchester University NHS Foundation Trust, Manchester

S10 2TH

Sheffield Children's NHS Foundation Trust, Sheffield

All Listed Sponsors
lead

QED Therapeutics, Inc., a Bridgebio company

INDUSTRY