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Enrolling by Invitation

NCT Number: NCT07393373

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

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.

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Key information

Conditions

Hypochondroplasia Bone Diseases Bone Diseases, Developmental Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Dwarfism Endocrine System Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis IV Musculoskeletal Diseases Nutritional and Metabolic Diseases Osteochondrodysplasias Skin and Connective Tissue Diseases

Age range

3 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne, Parkville, Victoria, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Infigratinib

Drug

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.

Primary outcomes

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

    Time frame: 10 years

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

    Time frame: 10 years

Secondary outcomes

  1. Changes over time in AHV Z-score

    Time frame: 10 years

  2. Changes over time in body proportions

    Time frame: 10 years

  3. Changes over time in weight Z-score

    Time frame: 10 years

  4. Changes overtime in BMI

    Time frame: 10 years

  5. Age of puberty onset and time to Tanner stage ≥4

    Time frame: 10 years

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

    Time frame: 10 years

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

    Time frame: 10 years

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

    Time frame: 10 years

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

    Time frame: 10 years

  10. Change in visual learning assessed by age-appropriate computerized tests (One Card Learning Test)

    Time frame: 10 years

  11. Change in working memory assessed by age-appropriate computerized tests (One Back Test)

    Time frame: 10 years

  12. Changes over time in severity of epilepsy measured by frequency and adverse event grading

    Time frame: 10 years

  13. Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)

    Time frame: 10 years

  14. Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)

    Time frame: 10 years

  15. Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)

    Time frame: 10 years

  16. Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)

    Time frame: 10 years

  17. Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview

    Time frame: 10 years

Sponsors and collaborators

Lead sponsor

QED Therapeutics, a BridgeBio company

Industry

Registry information

Official study 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

Important dates

Study start
2026
Primary completion
2036
Study completion
2036
First posted
Feb 6, 2026
Registry last updated
May 26, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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