Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

Phase 2Enrolling By InvitationNCT07393373

QED Therapeutics, a BridgeBio company135 enrolled

Overview

Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with Hypochondroplasia (HCH) who previously completed ACCEL 2/3, and potentially additional participants who completed ACCEL. Participants rolling over directly from the observational ACCEL study must have had at least a 6-month period of growth assessment in that study.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

135

Conditions

Hypochondroplasia

Interventions

InfigratinibDRUG

Infigratinib to be administered by mouth and initiated at the last dose level received in the ACCEL 2/3 study or at the dose selected to be further evaluated after proof-of-concept is established for Phase 2 portion of ACCEL 2/3.

Eligibility

Sex: ALLMin age: 3 YearsMax age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Inclusion Criteria for Participants Rolling Over from ACCEL 2/3 1. Pediatric participants with HCH who have completed ACCEL 2/3 2. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche Exclusion Criteria: * Exclusion Criteria for Participants Rolling Over from ACCEL 2/3 1. Participant has concurrent medical condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations 2. Participants who developed a medical condition that requires the initiation of treatment with a prohibited medication 3. Participants who prematurely discontinued ACCEL 2/3 4. Participants who have reached final height or near final height 5. Current participation in an ongoing clinical study with a sponsor other than QED

Locations (25)

Outcomes

Primary Outcomes

Incidence of treatment emergent adverse events (TEAE) and serious TEAE

Time frame: 10 years

Changes over time in standing height Z-score in relation to HCH and non-HCH growth charts

Time frame: 10 years

Secondary Outcomes

Changes over time in AHV Z-score

Time frame: 10 years

Changes over time in body proportions

Time frame: 10 years

Changes over time in weight Z-score

Time frame: 10 years

Changes overtime in BMI

Time frame: 10 years

Age of puberty onset and time to Tanner stage ≥4

Time frame: 10 years

Changes over time in body composition as assessed by DXA (dual x-ray absorptiometry) scans

Time frame: 10 years

Changes over time in bone morphology/density by x-ray and DXA

Time frame: 10 years

Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test)

Time frame: 10 years

Change in attention assessed by age-appropriate computerized tests (Identification Test)

Time frame: 10 years

Data from ClinicalTrials.gov

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