NCT07393373
Enrolling By Invitation
Phase 2
Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Hypochondroplasia: ACCEL OLE
QED Therapeutics, a BridgeBio company25 sites in 10 countries135 target enrollmentStarted: April 21, 2026Last updated:
InterventionsInfigratinib
Overview
- Phase
- Phase 2
- Status
- Enrolling By Invitation
- Sponsor
- QED Therapeutics, a BridgeBio company
- Enrollment
- 135
- Locations
- 25
- Primary Endpoint
- Incidence of treatment emergent adverse events (TEAE) and serious TEAE
Overview
Brief Summary
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 Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 3 Years to 18 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Inclusion Criteria for Participants Rolling Over from ACCEL 2/3
- •Pediatric participants with HCH who have completed ACCEL 2/3
- •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
- •Participant has concurrent medical condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations
- •Participants who developed a medical condition that requires the initiation of treatment with a prohibited medication
- •Participants who prematurely discontinued ACCEL 2/3
- •Participants who have reached final height or near final height
- •Current participation in an ongoing clinical study with a sponsor other than QED
Arms & Interventions
Arm 1: Rollover subjects
Experimental
Children who have completed QED-sponsored interventional study with infigratinib
Intervention: Infigratinib (Drug)
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(10 years)
- Changes over time in body proportions(10 years)
- Changes over time in weight Z-score(10 years)
- Changes overtime in BMI(10 years)
- Age of puberty onset and time to Tanner stage ≥4(10 years)
- Changes over time in body composition as assessed by DXA (dual x-ray absorptiometry) scans(10 years)
- Changes over time in bone morphology/density by x-ray and DXA(10 years)
- Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test)(10 years)
- Change in attention assessed by age-appropriate computerized tests (Identification Test)(10 years)
- Change in visual learning assessed by age-appropriate computerized tests (One Card Learning Test)(10 years)
- Change in working memory assessed by age-appropriate computerized tests (One Back Test)(10 years)
- Changes over time in severity of epilepsy measured by frequency and adverse event grading(10 years)
- Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)(10 years)
- Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)(10 years)
- Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)(10 years)
- Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)(10 years)
- Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview(10 years)
Investigators
Study Sites (25)
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