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• W4308138730 abstract "Abstract Background Achondroplasia (ACH), the most common non-lethal form of skeletal dysplasia, is characterized by defective endochondral ossification resulting from gain-of-function mutations in the fibroblast growth factor receptor 3 (FGFR3) gene, a negative regulator of endochondral bone formation. Infigratinib, a selective, orally bioavailable FGFR1–3 tyrosine kinase inhibitor, has been shown to reverse established growth arrest in chondrocytes and improve foramen magnum and long bone length in Fgfr3Y367C/+ mice. Infigratinib is being investigated for the treatment of ACH in the PROPEL program of three clinical trials: 1) PROPEL, designed to collect information on the natural history of ACH; 2) PROPEL2, designed to obtain preliminary evidence of efficacy and safety and to identify the dose of infigratinib to investigate further; 3) PROPEL OLE, which is designed to evaluate the long-term efficacy and safety of infigratinib in children with ACH. Methods PROPEL (NCT04035811) is a non-interventional clinical assessment study designed to characterize the natural history of up to 200 children aged 2.5–10 years with ACH over a 6−24-month period. The primary objective is to collect baseline height velocity measurements in children who may participate in an interventional study with infigratinib. The primary endpoint is the annualized growth velocity (AGV). Further objectives are to collect other baseline growth measurements, evaluate exploratory biomarker indicators of growth, and assess ACH-related medical events reported as medical history, or non-treatment adverse events (AEs). PROPEL2 (NCT04265651) is a phase 2, open-label study of infigratinib in children aged 3−11 years with ACH who completed ≥6 months observation in PROPEL. PROPEL2 includes dose-escalation with an extended dose-finding treatment phase (n≈40), a pharmacokinetics sub-study (n≈18), followed by a dose-expansion phase (n≈20) in which children receive infigratinib for 12 months to confirm the selected dose and provide evidence of efficacy. Primary endpoints are treatment-emergent AEs, change from baseline in AGV, and infigratinib pharmacokinetics. Secondary endpoints include safety/tolerability of infigratinib and changes from baseline in anthropometric parameters, including body proportions. Exploratory outcomes include changes in QoL and other parameters of disease burden. PROPEL OLE (NCT05145010) is a phase 2, open-label extension study in up to 230 children who completed an interventional study with infigratinib and, potentially, in ≤50 who are infigratinib-naive. The primary objectives are to evaluate safety, tolerability, and efficacy of long-term daily doses of infigratinib. Secondary objectives include evaluation of changes in other indicators of growth and development, and evaluation of QoL and disease burden. Children will receive infigratinib until they reach final/near final height. Summary PROPEL, PROPEL2, and PROPEL OLE are currently ongoing. Together, these studies are intended to provide key evidence on the safety and efficacy of oral infigratinib in children with ACH and will inform the design of future studies in this setting. Presentation: Saturday, June 11, 2022 1:00 p.m. - 3:00 p.m." @default.
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• W4308138730 date "2022-11-01" @default.
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• W4308138730 title "PSAT106 Infigratinib in Children with Achondroplasia: Design of the PROPEL, PROPEL2 and PROPEL OLE Studies" @default.
• W4308138730 doi "https://doi.org/10.1210/jendso/bvac150.1311" @default.
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