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Enrolling by invitationNCT07393373Updated Aug 26, 2026

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

A Phase 2 interventional study of Infigratinib in Hypochondroplasia, sponsored by QED Therapeutics, a BridgeBio company. Enrolling by invitation at 24 sites in 10 countries. Open to participants aged 3 Years to 18 Years. Per ClinicalTrials.gov, last updated 2026-08-26.

Sponsored by QED Therapeutics, a BridgeBio company (part of BridgeBio) · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
135
Allocation
Not applicable
Ages
3 Years to 18 Years
Sex
All
01

Study 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.

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Conditions studied

  • Hypochondroplasia

Keywords

  • skeletal dysplasia
  • endochondral ossification
  • hypochondroplasia
  • 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
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In context

Mucopolysaccharidosis IV

101 studies on the registry are indexed under Mucopolysaccharidosis IV; 27 are open to participants now.

This study's planned enrollment of 135 is above the median of 61 across 56 interventional studies indexed under Mucopolysaccharidosis IV.

Browse Mucopolysaccharidosis IV studies →

Lead sponsor

QED Therapeutics, a BridgeBio company is the lead sponsor of 8 studies on the registry; 5 are open to participants now.

Browse QED Therapeutics studies →All BridgeBio studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
3 Years to 18 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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:

  • 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
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Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
135 participants (estimated)

Study arms

  • Experimental
    Arm 1: Rollover subjects

    Children who have completed QED-sponsored interventional study with infigratinib

    Drug: Infigratinib

Interventions

  • DrugInfigratinib

    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.

06

What researchers measure

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

07

Study locations

24 sites
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References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 26, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT07393373
Lead sponsor
QED Therapeutics, a BridgeBio company
Responsible party
Sponsor
First posted
Feb 6, 2026
Start date
Apr 23, 2026
Primary completion
May 31, 2036 (estimated)
Completion
May 31, 2036 (estimated)
Last update
Aug 26, 2026

Study contacts

QED Therapeutics SVP, Clinical Development
study director · QED Therapeutics, a BridgeBio company

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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No contact was published for this record. The registry link below has the sponsor’s details.

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Studies of the drug tested here: Infigratinib

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