Study of Infigratinib in Children With Achondroplasia
ClinicalTrials.gov Identifier: NCT04265651
Phase 2 trial, multicenter, open-label, dose-escalation, and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment and dose expansion.
| Actual Study Start Date : | March 10, 2020 |
| Estimated Primary Completion Date : | October 2021 |
| Estimated Study Completion Date : | February 2023 |
| Ages Eligible for Study: | 3 Years to 11 Years (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Signed informed consent by participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the participant (when applicable).
- Diagnosis of ACH documented clinically and confirmed by genetic testing.
- At least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry.
- Ambulatory and able to stand without assistance
- Able to swallow oral medication.
Recruitment centers
| Australia, Victoria | |
| Murdoch Children's Hosptial | |
| Parkville, Victoria, Australia, 3052 | |
