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

NCT07169279 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 77

Last updated 2026-05-12

No results posted yet for this study

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.

Conditions

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.

Sponsors & Collaborators

  • QED Therapeutics, a BridgeBio company

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
0 Years
Max Age
32 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-11-19
Primary Completion
2032-03-31
Completion
2032-03-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • Norway
  • Singapore
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07169279 on ClinicalTrials.gov