Infigratinib for Achondroplasia

Recruiting in Palo Alto (17 mi)

+22 other locations

Age: < 18

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 trial is testing a medicine called infigratinib in children aged 3 to 11 years with Achondroplasia. The medicine works by blocking proteins that cause abnormal bone growth. The children have already participated in an earlier study.

Eligibility Criteria

This trial is for children aged 3-11 with Achondroplasia who were part of the PROPEL study. They must be able to swallow pills, stand unassisted, and not have started puberty. Kids can't join if they've had certain treatments like growth hormones recently or have eye disorders, a history of cancer, or been on long-term steroids.

Inclusion Criteria

Signed informed consent by participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the participant (when applicable).

At least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry.

I can walk and stand up on my own without help.

+2 more

Exclusion Criteria

You have another serious health condition that could make it difficult to evaluate the effectiveness or safety of infigratinib.

I have had a bone break in the last year.

I have a condition causing short stature, but it's not achondroplasia.

+10 more

Participant Groups

The trial tests different doses of Infigratinib (a drug that targets specific growth receptors) in kids with Achondroplasia to see which dose is safe and works best. It's an open-label study where everyone knows what treatment they're getting, involving both increasing the dose over time and expanding it to more patients.

5Treatment groups

Experimental Treatment

Group I: Infigratinib 0.25 mg/kgExperimental Treatment1 Intervention

Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months. Dose Expansion: Upon identification of the recommended dose from all cohorts analyzed, an expansion cohort of 20 subjects may begin enrollment to further determine safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of the selected dose.

Group II: Infigratinib 0.128 mg/kgExperimental Treatment1 Intervention

Group III: Infigratinib 0.064 mg/kgExperimental Treatment1 Intervention

Group IV: Infigratinib 0.032 mg/kgExperimental Treatment1 Intervention

Group V: Infigratinib 0.016 mg/kgExperimental Treatment1 Intervention

Dose Escalation: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.