FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W4387405277> ?p ?o ?g. }

• W4387405277 abstract "Abstract Disclosure: R. Savarirayan: Advisory Board Member; Self; Ascendis Pharma, BioMarin, QED Therapeutics, Sanofi. Consulting Fee; Self; BioMarin. Grant Recipient; Self; Ascendis Pharma, BioMarin, QED Therapeutics, Therachon. Research Investigator; Self; Ascendis Pharma, BioMarin, QED Therapeutics, Therachon. J. De Bergua: None. P. Arundel: None. H. McDevitt: None. V. Cormier-Daire: Advisory Board Member; Self; BioMarin. V. Saraff: None. M. Skae: None. B. Delgado: None. A. Leiva-Gea: None. M. Salcedo: None. J. Salles: None. M.P. Nicolino: None. M. Rossi: Advisory Board Member; Self; BioMarin. P. Kannu: Advisory Board Member; Self; Novartis, Ipsen. Grant Recipient; Self; CIHR. M. Bober: Advisory Board Member; Self; Biomarin. Consulting Fee; Self; Ascendis Pharma, BioMarin, Pfizer, QED Therapeutics. Grant Recipient; Self; Ascendis Pharma, BioMarin, Pfizer, QED Therapeutics. Research Investigator; Self; Ascendis Pharma, BioMarin, Pfizer, QED Therapeutics. J. Phillips III: None. H. Saal: Advisory Board Member; Self; Alexion. Grant Recipient; Self; Alexion, BioMarin, Pfizer, QED Therapeutics. Research Investigator; Self; Alexion, BioMarin, Pfizer, QED Therapeutics. P. Harmatz: Consulting Fee; Self; Audentes, Aeglea, Homology, JCR, Denali, Inventiva, Paradigm, Capsida, Chiesi, Avrobio. Grant Recipient; Self; BioMarin, Inventiva. Research Investigator; Self; BioMarin, Shire/Takeda, QED Therapeutics, RegenXbio, Denali, Ascendis, Amicus, Allievex, JCR, Orphazyme, Idorsia, Sangamo. C. Burren: Grant Recipient; Self; Amgen, Pfizer, QED Therapeutics. Research Investigator; Self; Amgen, Pfizer, QED Therapeutics. T. Candler: None. T. Cho: Employee; Self; QED Therapeutics. Stock Owner; Self; QED Therapeutics. E. Muslimova: Employee; Self; QED Therapeutics. Stock Owner; Self; QED Therapeutics. R. Weng: Employee; Self; QED Therapeutics. Stock Owner; Self; QED Therapeutics. D. Rogoff: Employee; Self; QED Therapeutics. Stock Owner; Self; QED Therapeutics. J. Hoover-Fong: Consulting Fee; Self; Pfizer/Therachon, BioMarin, QED Therapeutics, Sanofi, Ascendis Pharma. Grant Recipient; Self; Pfizer/Therachon, BioMarin, Ascendis Pharma. Research Investigator; Self; Pfizer/Therachon, BioMarin, Ascendis Pharma. M. Irving: Advisory Board Member; Self; Ascendis Pharma, BioMarin, QED Therapeutics, Sanofi, Therachon/Pfizer. Speaker; Self; BioMarin, QED Therapeutics. Background: Achondroplasia (ACH), the most common short-limbed 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 is a selective, orally bioavailable FGFR1-3 tyrosine kinase inhibitor being investigated for the treatment of ACH in the observational and 2 interventional studies, as detailed below. Methods: PROPEL (NCT04035811) is a non-interventional clinical assessment study designed to characterize the natural history of ∼250 children 2.5 to &lt;17 years of age with ACH over a 6−24-month period. Primary objective: collect baseline height velocity measurements in children who may participate in an interventional study with infigratinib. Primary endpoint: annualized growth velocity (AGV). Further objectives: collect other baseline growth measurements; evaluate exploratory biomarker indicators of growth; assess ACH-related medical events reported as medical history, or non-treatment adverse events (AEs), health-related quality of life, body pain, functional abilities and cognitive functions in children with ACH. PROPEL 2 (NCT04265651) is a phase 2, open-label study of infigratinib in children 3−11 years of age with ACH who completed ≥6 months of observation in PROPEL. This study includes dose-escalation (extended dose-finding treatment phase, n≥40), a pharmacokinetics sub-study (n≥18), and a dose-expansion phase (n≈20) to confirm the selected dose and provide evidence of efficacy. Primary endpoints: AEs; change from baseline in AGV; and infigratinib pharmacokinetics. Secondary endpoints: safety/tolerability of infigratinib; changes from baseline in anthropometric parameters. Exploratory outcomes: changes in quality of life (QoL) and other parameters of disease burden. PROPEL OLE (NCT05145010) is a phase 2, open-label extension study in ∼230 children who completed an interventional study with infigratinib and, potentially, in ≤50 infigratinib-naïve children. Primary objectives: safety, tolerability; and efficacy of long-term daily doses of infigratinib. Secondary objectives: changes in other indicators of growth/development, skeletal abnormalities, QoL/disease burden, and cognitive functions. Children may receive infigratinib until they reach final height. Summary: The PROPEL, PROPEL 2, and PROPEL OLE studies are ongoing. Together, they are intended to contribute to the understanding of the natural history of ACH, provide key evidence on the safety and efficacy of oral infigratinib, including QoL and skeletal changes in children with ACH, and inform the design of future studies in this setting. Presentation: Thursday, June 15, 2023" @default.
• W4387405277 created "2023-10-07" @default.
• W4387405277 creator A5000857707 @default.
• W4387405277 creator A5000891412 @default.
• W4387405277 creator A5001570533 @default.
• W4387405277 creator A5001767521 @default.
• W4387405277 creator A5004969502 @default.
• W4387405277 creator A5006532808 @default.
• W4387405277 creator A5006745801 @default.
• W4387405277 creator A5008365701 @default.
• W4387405277 creator A5025914068 @default.
• W4387405277 creator A5028724151 @default.
• W4387405277 creator A5031316607 @default.
• W4387405277 creator A5031964969 @default.
• W4387405277 creator A5033079105 @default.
• W4387405277 creator A5034336613 @default.
• W4387405277 creator A5038560947 @default.
• W4387405277 creator A5040237112 @default.
• W4387405277 creator A5042446335 @default.
• W4387405277 creator A5042951745 @default.
• W4387405277 creator A5043701965 @default.
• W4387405277 creator A5049904372 @default.
• W4387405277 creator A5061306814 @default.
• W4387405277 creator A5069130924 @default.
• W4387405277 creator A5072762008 @default.
• W4387405277 creator A5077589167 @default.
• W4387405277 creator A5078684328 @default.
• W4387405277 creator A5090295108 @default.
• W4387405277 date "2023-10-01" @default.
• W4387405277 modified "2023-10-17" @default.
• W4387405277 title "THU165 PROPEL, PROPEL 2 And PROPEL OLE Studies Of Infigratinib In Children With Achondroplasia: Design And Status Of 3 Ongoing Trials" @default.
• W4387405277 doi "https://doi.org/10.1210/jendso/bvad114.1416" @default.
• W4387405277 hasPublicationYear "2023" @default.
• W4387405277 type Work @default.
• W4387405277 citedByCount "0" @default.
• W4387405277 crossrefType "journal-article" @default.
• W4387405277 hasAuthorship W4387405277A5000857707 @default.
• W4387405277 hasAuthorship W4387405277A5000891412 @default.
• W4387405277 hasAuthorship W4387405277A5001570533 @default.
• W4387405277 hasAuthorship W4387405277A5001767521 @default.
• W4387405277 hasAuthorship W4387405277A5004969502 @default.
• W4387405277 hasAuthorship W4387405277A5006532808 @default.
• W4387405277 hasAuthorship W4387405277A5006745801 @default.
• W4387405277 hasAuthorship W4387405277A5008365701 @default.
• W4387405277 hasAuthorship W4387405277A5025914068 @default.
• W4387405277 hasAuthorship W4387405277A5028724151 @default.
• W4387405277 hasAuthorship W4387405277A5031316607 @default.
• W4387405277 hasAuthorship W4387405277A5031964969 @default.
• W4387405277 hasAuthorship W4387405277A5033079105 @default.
• W4387405277 hasAuthorship W4387405277A5034336613 @default.
• W4387405277 hasAuthorship W4387405277A5038560947 @default.
• W4387405277 hasAuthorship W4387405277A5040237112 @default.
• W4387405277 hasAuthorship W4387405277A5042446335 @default.
• W4387405277 hasAuthorship W4387405277A5042951745 @default.
• W4387405277 hasAuthorship W4387405277A5043701965 @default.
• W4387405277 hasAuthorship W4387405277A5049904372 @default.
• W4387405277 hasAuthorship W4387405277A5061306814 @default.
• W4387405277 hasAuthorship W4387405277A5069130924 @default.
• W4387405277 hasAuthorship W4387405277A5072762008 @default.
• W4387405277 hasAuthorship W4387405277A5077589167 @default.
• W4387405277 hasAuthorship W4387405277A5078684328 @default.
• W4387405277 hasAuthorship W4387405277A5090295108 @default.
• W4387405277 hasBestOaLocation W43874052771 @default.
• W4387405277 hasConcept C121332964 @default.
• W4387405277 hasConcept C71924100 @default.
• W4387405277 hasConceptScore W4387405277C121332964 @default.
• W4387405277 hasConceptScore W4387405277C71924100 @default.
• W4387405277 hasIssue "Supplement_1" @default.
• W4387405277 hasLocation W43874052771 @default.
• W4387405277 hasOpenAccess W4387405277 @default.
• W4387405277 hasPrimaryLocation W43874052771 @default.
• W4387405277 hasRelatedWork W1506200166 @default.
• W4387405277 hasRelatedWork W1995515455 @default.
• W4387405277 hasRelatedWork W2080531066 @default.
• W4387405277 hasRelatedWork W2748952813 @default.
• W4387405277 hasRelatedWork W2899084033 @default.
• W4387405277 hasRelatedWork W2935759653 @default.
• W4387405277 hasRelatedWork W3031052312 @default.
• W4387405277 hasRelatedWork W3032375762 @default.
• W4387405277 hasRelatedWork W3105167352 @default.
• W4387405277 hasRelatedWork W3108674512 @default.
• W4387405277 hasVolume "7" @default.
• W4387405277 isParatext "false" @default.
• W4387405277 isRetracted "false" @default.
• W4387405277 workType "article" @default.