RecruitingPhase 2NCT07169279

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

Sponsor

QED Therapeutics, a BridgeBio company

Enrollment

77 participants

Start Date

Nov 19, 2025

Study Type

INTERVENTIONAL

Conditions

Achondroplasia

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.

Eligibility

Min Age: 0 YearsMax Age: 32 Months

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Infigratinib is provided as a single dose of minitablets for oral administration, a drug called Infigratinib is provided as sprinkle capsules for daily oral administration, and others for people with achondroplasia. The study is currently recruiting participants at 12 locations. People eligible for this study include aged 0 Years to 32 Months.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGInfigratinib 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 dose and number of minitablets will be calculated based on individual participant age and weight.

DRUGInfigratinib 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.

DRUGInfigratinib 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.

DRUGInfigratinib 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.

Locations(12)

UCSF Benioff Children's Hospital

Oakland, California, United States

Johns Hopkins University

Baltimore, Maryland, United States

University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

Madison, Wisconsin, United States

Murdoch Children's Research Institute

Parkville, Victoria, Australia

Children's Hospital of Eastern Ontario Research Institute

Ottawa, Ontario, Canada

Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, Canada

Paediatric Clinical Research Unit at Oslo University Hospital

Oslo, Norway

KK Women's and Children's Hospital

Singapore, Singapore

Unidad de Cirugía Artroscópica (UCA)

Vitoria-Gasteiz, Spain

NHS Greater Glasgow and Clyde

Glasgow, United Kingdom

Manchester University NHS Foundation Trust

Manchester, United Kingdom

Sheffield Children's NHS Foundation Trust

Sheffield, United Kingdom

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT07169279

Related Trials

A Study of TYRA-300 in Children With Achondroplasia: BEACH301

NCT0684235522 locations

Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

NCT0744187619 locations

VIrtual STudy in Achondroplasia for the US (VISTA)

NCT061682018 locations

A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.

NCT067328955 locations

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

NCT0607939818 locations