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Study of Infigratinib in Children With Achondroplasia

NCT04265651 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 84

Last updated 2025-10-22

No results posted yet for this study

Summary

This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment, and dose expansion. The study also includes a PK Substudy to fully characterize the pharmacokinetics of infigratinib in children with ACH.

Conditions

Interventions

DRUG

Infigratinib 0.016 mg/kg

Initial cohort dose of infigratinib at the protocol-specified starting dose, with subsequent cohort escalations based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.

DRUG

Infigratinib 0.032 mg/kg

Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.

DRUG

Infigratinib 0.064 mg/kg

Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.

DRUG

Infigratinib 0.128 mg/kg

Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.

DRUG

Infigratinib 0.25 mg/kg

Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.

Sponsors & Collaborators

  • QED Therapeutics, Inc., a Bridgebio company

    lead INDUSTRY

Principal Investigators

  • QED Therapeutics VP, Clinical Development · QED Therapeutics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
3 Years
Max Age
11 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-03-10
Primary Completion
2024-10-21
Completion
2024-10-21
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • France
  • 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 NCT04265651 on ClinicalTrials.gov