Infigratinib for Dwarfism (HCH Trial)

Recruiting in Palo Alto (17 mi)

+20 other locations

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Waitlist Available

Sponsor: QED Therapeutics, Inc.

No Placebo Group

Prior Safety Data

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

I have been part of the QBGJ398-004 study for at least 26 weeks and am still participating.

I am between 5 and 11 years old.

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

I have been diagnosed with HCH due to my short stature, confirmed by a test.

I can stand and walk on my own without help.

I am a girl over 10 or have had my period and my pregnancy test is negative.

Exclusion Criteria

I have a serious eye condition confirmed by an eye doctor.

I have been on high-dose steroids for more than 3 weeks.

I have had or plan to have surgery to lengthen my limbs.

Participant Groups

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.

2Treatment groups

Experimental Treatment

Group I: Phase 2 Cohort 2Experimental Treatment1 Intervention

infigratinib (0.25 mg/kg/day)

Group II: Phase 2 Cohort 1Experimental Treatment1 Intervention

infigratinib (0.128 mg/kg/day)