Infigratinib for Achondroplasia

Recruiting in Palo Alto (17 mi)

+20 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: QED Therapeutics, Inc.

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Quality of Life assessments for this subject population will also be evaluated. Treatment-naïve subjects must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201 and subjects are not otherwise eligible to enroll in another QED-sponsored Phase 2 or Phase 3 ACH study.

Eligibility Criteria

This trial is for children with Achondroplasia (ACH) aged 3 to <18 who have growth potential and completed a previous infigratinib study or have been assessed for growth over 6 months. They must be able to swallow pills, comply with the study plan, and use contraception if applicable. Those with recent fractures, other short stature conditions, or using certain drugs can't join.

Inclusion Criteria

I can take pills by mouth.

I agree to use effective birth control during and for 1 month after the study.

Pediatric subjects with ACH who have completed study activities in a previous QED-sponsored interventional study with infigratinib

+6 more

Exclusion Criteria

I am not taking medication that strongly affects liver enzyme CYP3A4.

I have reached my full adult height.

I need to start a medication that is not allowed in the trial.

+8 more

Participant Groups

The trial tests the long-term safety and effectiveness of Infigratinib, an FGFR inhibitor, in kids who've taken it before or are new to it after a specific dose has been identified. It's an open-label extension study where all participants receive the drug and their quality of life is monitored.

2Treatment groups

Experimental Treatment

Group I: Arm 2: Treatment naïve subjectsExperimental Treatment1 Intervention

Children naïve to infigratinib

Group II: Arm 1: Rollover subjectsExperimental Treatment1 Intervention

Children who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3)