FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W3088970141> ?p ?o ?g. }

• W3088970141 endingPage "115664" @default.
• W3088970141 startingPage "115664" @default.
• W3088970141 abstract "Hypophosphatasia (HPP) is the rare, inherited, metabolic bone disease characterized by low activity of the tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP) leading to excess extracellular inorganic pyrophosphate (PPi) and pyridoxal 5'-phosphate (PLP). Asfotase alfa is the human recombinant enzyme-replacement therapy that replaces deficient TNSALP. However, there is limited information concerning the appropriate dose of asfotase alfa for adult patients with pediatric-onset HPP. Thus, we evaluated the pharmacodynamics and safety/tolerability of different doses of asfotase alfa in such patients.This 13-week, Phase 2a, open-label study enrolled adults (aged ≥18 years) with pediatric-onset HPP. They were randomized 1:1:1 to receive a single subcutaneous dose of asfotase alfa (0.5, 2.0, or 3.0 mg/kg) at Week 1, then 3 times per week (ie, 1.5, 6.0, or 9.0 mg/kg/wk) starting at Week 3 for 7 weeks. Key outcome measures included change from Baseline to before the third dose during Week 9 (trough) in plasma PPi (primary outcome measure) and PLP (secondary outcome measure).Twenty-seven adults received asfotase alfa 0.5 (n = 8), 2.0 (n = 10), and 3.0 (n = 9) mg/kg; all completed the study. Median (range) age was 45 (18-77) years; most patients were white (96%) and female (59%). Median plasma PPi and PLP concentrations decreased from Baseline to Week 9 in all 3 cohorts. Differences in least squares mean (LSM) changes in PPi were significant with 2.0 mg/kg (p = 0.0008) and 3.0 mg/kg (p < 0.0001) vs. 0.5 mg/kg. Differences in LSM changes in PLP were also significant for 2.0 mg/kg (p = 0.0239) and 3.0 mg/kg (p = 0.0128) vs. 0.5 mg/kg. Injection site reactions were the most frequent treatment-emergent adverse event (78%), showing increasing frequency with increasing dose.Adults with pediatric-onset HPP receiving asfotase alfa at 6.0 mg/kg/wk (the recommended dose) or 9.0 mg/kg/wk had greater reductions in circulating PPi and PLP concentrations compared with a lower dose of 1.5 mg/kg/wk.Clinicaltrials.gov identifier NCT02797821." @default.
• W3088970141 created "2020-10-01" @default.
• W3088970141 creator A5000903477 @default.
• W3088970141 creator A5005473186 @default.
• W3088970141 creator A5006014530 @default.
• W3088970141 creator A5021401921 @default.
• W3088970141 creator A5035870078 @default.
• W3088970141 creator A5036775753 @default.
• W3088970141 creator A5037775852 @default.
• W3088970141 date "2021-01-01" @default.
• W3088970141 modified "2023-09-25" @default.
• W3088970141 title "Pharmacodynamics of asfotase alfa in adults with pediatric-onset hypophosphatasia" @default.
• W3088970141 cites W1970270213 @default.
• W3088970141 cites W1973772928 @default.
• W3088970141 cites W1985358886 @default.
• W3088970141 cites W1994460887 @default.
• W3088970141 cites W2005513865 @default.
• W3088970141 cites W2006848294 @default.
• W3088970141 cites W2049802698 @default.
• W3088970141 cites W2050045002 @default.
• W3088970141 cites W2102235351 @default.
• W3088970141 cites W2107554167 @default.
• W3088970141 cites W2131795083 @default.
• W3088970141 cites W2141225628 @default.
• W3088970141 cites W2145360615 @default.
• W3088970141 cites W2150512825 @default.
• W3088970141 cites W2169170480 @default.
• W3088970141 cites W2208579015 @default.
• W3088970141 cites W2280954766 @default.
• W3088970141 cites W2437385547 @default.
• W3088970141 cites W2489042166 @default.
• W3088970141 cites W2513922842 @default.
• W3088970141 cites W2559874490 @default.
• W3088970141 cites W2738193737 @default.
• W3088970141 cites W2890457058 @default.
• W3088970141 cites W2904850670 @default.
• W3088970141 cites W2905259247 @default.
• W3088970141 cites W2910000388 @default.
• W3088970141 cites W2919472853 @default.
• W3088970141 cites W2924411106 @default.
• W3088970141 cites W2937942213 @default.
• W3088970141 cites W4240468194 @default.
• W3088970141 doi "https://doi.org/10.1016/j.bone.2020.115664" @default.
• W3088970141 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/32987199" @default.
• W3088970141 hasPublicationYear "2021" @default.
• W3088970141 type Work @default.
• W3088970141 sameAs 3088970141 @default.
• W3088970141 citedByCount "13" @default.
• W3088970141 countsByYear W30889701412021 @default.
• W3088970141 countsByYear W30889701412022 @default.
• W3088970141 countsByYear W30889701412023 @default.
• W3088970141 crossrefType "journal-article" @default.
• W3088970141 hasAuthorship W3088970141A5000903477 @default.
• W3088970141 hasAuthorship W3088970141A5005473186 @default.
• W3088970141 hasAuthorship W3088970141A5006014530 @default.
• W3088970141 hasAuthorship W3088970141A5021401921 @default.
• W3088970141 hasAuthorship W3088970141A5035870078 @default.
• W3088970141 hasAuthorship W3088970141A5036775753 @default.
• W3088970141 hasAuthorship W3088970141A5037775852 @default.
• W3088970141 hasBestOaLocation W30889701411 @default.
• W3088970141 hasConcept C111113717 @default.
• W3088970141 hasConcept C112705442 @default.
• W3088970141 hasConcept C126322002 @default.
• W3088970141 hasConcept C134018914 @default.
• W3088970141 hasConcept C160160445 @default.
• W3088970141 hasConcept C181199279 @default.
• W3088970141 hasConcept C185592680 @default.
• W3088970141 hasConcept C197934379 @default.
• W3088970141 hasConcept C2778375690 @default.
• W3088970141 hasConcept C2778471162 @default.
• W3088970141 hasConcept C2779134260 @default.
• W3088970141 hasConcept C2779969927 @default.
• W3088970141 hasConcept C55493867 @default.
• W3088970141 hasConcept C71924100 @default.
• W3088970141 hasConcept C90924648 @default.
• W3088970141 hasConceptScore W3088970141C111113717 @default.
• W3088970141 hasConceptScore W3088970141C112705442 @default.
• W3088970141 hasConceptScore W3088970141C126322002 @default.
• W3088970141 hasConceptScore W3088970141C134018914 @default.
• W3088970141 hasConceptScore W3088970141C160160445 @default.
• W3088970141 hasConceptScore W3088970141C181199279 @default.
• W3088970141 hasConceptScore W3088970141C185592680 @default.
• W3088970141 hasConceptScore W3088970141C197934379 @default.
• W3088970141 hasConceptScore W3088970141C2778375690 @default.
• W3088970141 hasConceptScore W3088970141C2778471162 @default.
• W3088970141 hasConceptScore W3088970141C2779134260 @default.
• W3088970141 hasConceptScore W3088970141C2779969927 @default.
• W3088970141 hasConceptScore W3088970141C55493867 @default.
• W3088970141 hasConceptScore W3088970141C71924100 @default.
• W3088970141 hasConceptScore W3088970141C90924648 @default.
• W3088970141 hasFunder F4320309052 @default.
• W3088970141 hasLocation W30889701411 @default.
• W3088970141 hasLocation W30889701412 @default.
• W3088970141 hasOpenAccess W3088970141 @default.
• W3088970141 hasPrimaryLocation W30889701411 @default.
• W3088970141 hasRelatedWork W1537289008 @default.
• W3088970141 hasRelatedWork W1934667138 @default.
• W3088970141 hasRelatedWork W2014468166 @default.

• next