This study is accepting new patients by invitation only

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

a study on Hypochondroplasia FGFR Gene Alteration Mucopolysaccharidosis

Summary

Eligibility
for people ages 3-18 (full criteria)
Location
at Oakland, California and other locations
Dates
study started
study ends around

Description

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.

Official Title

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

Keywords

Hypochondroplasia, skeletal dysplasia, endochondral ossification, HCH, shortened proximal limbs, fibroblast growth factor receptor 3, FGFR3, endochondral bone formation, disproportionate short stature, quality of life, dwarfism, bone diseases, musculoskeletal diseases, osteochondrodysplasia, functional abilities, annualized growth velocity, annualized height velocity, growth, genetic diseases, congenital, AHV, AGV, Pathogenic variants, Mucopolysaccharidosis IV, Osteochondrodysplasias, Inborn Genetic Diseases, infigratinib

Eligibility

You can join if…

Open to people ages 3-18

  • 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

You CAN'T join if...

  • 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

  • UCSF Benioff Children's Hospital
    Oakland California 94609 United States
  • Childrens Hospital Colorado
    Aurora Colorado 80045 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
QED Therapeutics, a BridgeBio company
ID
NCT07393373
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 135 study participants
Last Updated