Infigratinib for Dwarfism

(HCH Trial)

Recruiting at 21 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: QED Therapeutics, Inc.

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 0

24 Participants Needed

Trial Summary

What is the purpose of this trial?

ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety, of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib, at one of two doses, of who have completed at least 26 weeks of participation in QED-sponsored ACCEL (QBGJ398-004).

Eligibility Criteria

This trial is for children with Hypochondroplasia (HCH), a form of dwarfism that affects bone growth. Participants must have completed at least 26 weeks in a prior ACCEL study and are now continuing treatment to assess further effects.

Inclusion Criteria

Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures

Signed informed consent

I am between 3 and 18 years old and still growing.

Exclusion Criteria

Participants who have ACH or a short stature condition other than HCH

Allergy to any components of the study drug

Pregnant or breastfeeding at the screening visit or planning to become pregnant (self or partner) at any time during the study

Treatment Details

Interventions

• Infigratinib

Trial Overview The trial is testing two different doses of Infigratinib, an experimental medication, to see how effective and safe it is for treating HCH in children. It's part of a larger Phase 2/3 study following initial treatment results.

Participant Groups

Group I: Phase 2 Cohort 2 Experimental Treatment 1 Intervention

infigratinib (0.25 mg/kg/day)

Group II: Phase 2 Cohort 1 Experimental Treatment 1 Intervention

infigratinib (0.128 mg/kg/day)

Who Is Running the Clinical Trial?

QED Therapeutics, Inc.

Lead Sponsor

Trials

11

Recruited

1,200+