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

Part of paid clinical trials in Oakland, California.

Sponsor
QED Therapeutics, a BridgeBio company
Study ID
NCT07169279
Phase
PHASE2
Status
Recruiting
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Conditions

  • Achondroplasia

Eligibility Criteria

Sex
ALL
Age
0 Years - 32 Months
Healthy Volunteers
Not accepted

Interventions

  • Infigratinib is provided as a single dose of minitablets for oral administration — DRUG
    * 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.
  • Infigratinib is provided as sprinkle capsules for daily oral administration — DRUG
    * 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.
  • Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration — DRUG
    * 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.
  • Infigratinib is provided as sprinkle capsules for daily oral administration — DRUG
    * 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.

Study Details

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.

Key Dates

Start date
Nov 19, 2025
Status verified
May 2026
Primary completion
Mar 31, 2032
Completion
Mar 31, 2032

Study Design

Enrollment
77 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: SAD Cohort 1 infigratinib (2 to less than 3 years old)
    Single Ascending Dose Escalation and PK Portion
  • Experimental: SAD Cohort 2 infigratinib (1 to less than 2 years old)
    Single Ascending Dose Escalation and PK Portion
  • Experimental: SAD Cohort 3 infigratinib (6 months to less than 1 year old)
    Single Ascending Dose Escalation and PK Portion
  • Experimental: SAD Cohort 4 infigratinib (0 to less than 6 months old)
    Single Ascending Dose Escalation and PK Portion
  • Experimental: Phase 2 Cohort 1 infigratinib (2 to less than 3 years old)
    Open-label Safety and PK Portion
  • Experimental: Phase 2 Cohort 2 infigratinib (1 to less than 2 years old)
    Open-label Safety and PK Portion
  • Experimental: Phase 2 Cohort 3 infigratinib (6 months to less than 1 year old)
    Open-label Safety and PK Portion
  • Experimental: Phase 2 Cohort 4 infigratinib (0 to less than 6 months)
    Open-label Safety and PK Portion
  • Experimental: Phase 2B Cohort 1 infigratinib (2 to less than 3 years old)
    Randomized Safety and Efficacy Portion
  • Experimental: Phase 2B Cohort 2 infigratinib (6 months to less than 2 years old)
    Randomized Safety and Efficacy Portion
  • Experimental: Phase 2B Cohort 3 infigratinib (0 to less than 6 months old)
    Randomized Safety and Efficacy Portion
  • Experimental: Open- label Extension infigratinib (0 months to 3 years [+6 months old])
    Open-label extension portion continuing to assess safety and efficacy in children until they reach 3 years old (+6 months)
  • Placebo Comparator: Phase 2B Cohort 1 Placebo (2 to less than 3 years old)
    Randomized Safety and Efficacy study
  • Placebo Comparator: Phase 2B Cohort 2 placebo (6 months to less than 2 years old)
    Randomized Safety and Efficacy study
  • Placebo Comparator: Phase 2B Cohort 3 Placebo (0 to less than 6 months old)
    Randomized Safety and Efficacy study

Primary Outcome Measure

Single Ascending Dose Portion: Identify the dose of infigratinib to be used in each age cohort of the Phase 2 potion of the study (by assessing safety and PK of infigratinib and its active metabolites) [ Time Frame: 2 weeks ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
UCSF Benioff Children's HospitalOaklandCalifornia94609-
Johns Hopkins UniversityBaltimoreMaryland21218-
University of Wisconsin Madison - Waisman Center Bone Dysplasia ClinicMadisonWisconsin53705-

Find similar trials in Oakland, CA

By research site
UCSF Benioff Children's Hospital· Oakland, CAJohns Hopkins University· Baltimore, MDUniversity of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic· Madison, WI

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