Extension Study of Infigratinib in Children With Achondroplasia (ACH)

Part of paid clinical trials in Oakland, California.

Sponsor
QED Therapeutics, a BridgeBio company
Study ID
NCT05145010
Phase
PHASE2
Status
Enrolling By Invitation

Conditions

  • Achondroplasia

Eligibility Criteria

Sex
ALL
Age
3 Years - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • Infigratinib — DRUG
    Infigratinib minitablets or sprinkle capsules to be administered by mouth. In subjects that completed a prior study with infigratinib, the starting dose will be the same as the last dose received in the prior interventional study with infigratinib. Infigratinib dose may be adjusted to 0.25 mg/kg/day (the dose selected to be explored further in the dose escalation portion of Phase 2 study QBGJ398-201 (PROPEL 2)).
  • Infigratinib — DRUG
    Infigratinib sprinkle capsules to be administered by mouth. Starting dose for the subjects naïve to Infigratinib will be 0.25 mg/kg/day (the dose selected to be explored further in the dose escalation portion of Phase 2 study QBGJ398-201 (PROPEL 2)).

Study Details

This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Quality of Life assessments for this subject population will also be evaluated. Treatment-naïve subjects must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201 and subjects are not otherwise eligible to enroll in another QED-sponsored Phase 2 or Phase 3 ACH study.

Key Dates

Start date
Dec 6, 2021
Status verified
Oct 2025
Primary completion
Dec 1, 2031
Completion
Feb 1, 2032

Study Design

Enrollment
300 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Arm 1: Rollover subjects
    Children who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3)
  • Experimental: Arm 2: Treatment naïve subjects
    Children naïve to infigratinib

Primary Outcome Measure

Incidence of treatment emergent adverse events (TEAE) and serious TEAE [ Time Frame: 10 years ]

Locations (8)

FacilityCityStateZIPSite coordinators
USCF Benioff Children's Hospital, OaklandOaklandCalifornia94609-
Children's Hospital ColoradoAuroraColorado80045-
Nemours Alfred I. Dupont Hospital for ChildrenWilmingtonDelaware19803-
Johns Hopkins UniversityBaltimoreMaryland21205-
University of MissouriColumbiaMissouri65212-
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229-
Vanderbilt University Medical CenterNashvilleTennessee37232-
University Hospital and UW Health ClinicsMadisonWisconsin53705-

Find similar trials in Oakland, CA

By research site
USCF Benioff Children's Hospital, Oakland· Oakland, CAChildren's Hospital Colorado· Aurora, CONemours Alfred I. Dupont Hospital for Children· Wilmington, DEJohns Hopkins University· Baltimore, MDUniversity of Missouri· Columbia, MOCincinnati Children's Hospital Medical Center· Cincinnati, OH

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