Literature DB >> 24372304

Phase 1 safety, tolerability and pharmacokinetics of 3K3A-APC in healthy adult volunteers.

Patrick Lyden, Howard Levy, Sara Weymer, Kent Pryor, William Kramer, John H Griffin, Thomas P Davis, Berislav Zlokovic1.   

Abstract

BACKGROUND AND
PURPOSE: Activated Protein C (APC) stimulates multiple cytoprotective pathways via the protease activated receptor-1 (PAR-1) and promotes anticoagulation. 3K3A-APC was designed for preserved activity at PAR-1 with reduced anticoagulation. This Phase 1 trial characterized pharmacokinetics and anticoagulation effects of 3K3A-APC.
METHODS: Subjects (n=64) were randomly assigned to receive 3K3A-APC (n=4) at 6, 30, 90, 180, 360, 540 or 720 µg/kg or placebo (n=6) and were observed for 24 hr. After safety review additional subjects received drug every 12 hr for 5 doses (n=6 per group) at 90, 180, 360, or 540 µg/kg or placebo (n=8) and were observed for 24 hr.
RESULTS: All subjects returned for safety assessments at 72 hours and 15 days. We found few adverse events in all groups. Systolic blood pressure increased in both active and placebo groups. Moderately severe headache, nausea and vomiting were reported in one of two subjects treated with 720 µg/kg so 540 µg/kg was considered the highest tolerated dose. Mean plasma concentrations increased in proportion to dose. Clearance ranged from 11,693 ± 807 to 18,701 ± 4,797 mL/hr, volume of distribution ranged from 4,873±828 to 6,971 ± 1,169 mL, and elimination half-life ranged from 0.211 ± 0.097 to 0.294 ± 0.054 hours. Elevations in aPTT were minimal.
CONCLUSIONS: 3K3A-APC was well tolerated at multiple doses as high as 540 µg/kg. These results should be confirmed in stroke patients with relevant co-morbidities. Clinical Trial Registration-URL: http://www.clinicaltrials.gov. Unique identifier: NCT01660230.

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Year:  2013        PMID: 24372304      PMCID: PMC4040367          DOI: 10.2174/1381612819666131230131454

Source DB:  PubMed          Journal:  Curr Pharm Des        ISSN: 1381-6128            Impact factor:   3.116


  32 in total

1.  Activated protein C variants with normal cytoprotective but reduced anticoagulant activity.

Authors:  Laurent O Mosnier; Andrew J Gale; Subramanian Yegneswaran; John H Griffin
Journal:  Blood       Date:  2004-06-03       Impact factor: 22.113

2.  Direct detection of activated protein C in blood from human subjects.

Authors:  A Gruber; J H Griffin
Journal:  Blood       Date:  1992-05-01       Impact factor: 22.113

3.  Low molecular weight heparinoid, ORG 10172 (danaparoid), and outcome after acute ischemic stroke: a randomized controlled trial. The Publications Committee for the Trial of ORG 10172 in Acute Stroke Treatment (TOAST) Investigators.

Authors: 
Journal:  JAMA       Date:  1998 Apr 22-29       Impact factor: 56.272

4.  Efficacy and safety of recombinant human activated protein C for severe sepsis.

Authors:  G R Bernard; J L Vincent; P F Laterre; S P LaRosa; J F Dhainaut; A Lopez-Rodriguez; J S Steingrub; G E Garber; J D Helterbrand; E W Ely; C J Fisher
Journal:  N Engl J Med       Date:  2001-03-08       Impact factor: 91.245

5.  Antithrombotic treatment of ischemic stroke among patients with occlusion or severe stenosis of the internal carotid artery: A report of the Trial of Org 10172 in Acute Stroke Treatment (TOAST).

Authors:  H P Adams; B H Bendixen; E Leira; K C Chang; P H Davis; R F Woolson; W R Clarke; M D Hansen
Journal:  Neurology       Date:  1999-07-13       Impact factor: 9.910

6.  Activated protein C alters cytosolic calcium flux in human brain endothelium via binding to endothelial protein C receptor and activation of protease activated receptor-1.

Authors:  Eszter Dömötör; Omar Benzakour; John H Griffin; David Yule; Kenji Fukudome; Berislav V Zlokovic
Journal:  Blood       Date:  2003-02-13       Impact factor: 22.113

7.  Endothelial barrier protection by activated protein C through PAR1-dependent sphingosine 1-phosphate receptor-1 crossactivation.

Authors:  Clemens Feistritzer; Matthias Riewald
Journal:  Blood       Date:  2004-12-30       Impact factor: 22.113

8.  Activated protein C blocks p53-mediated apoptosis in ischemic human brain endothelium and is neuroprotective.

Authors:  Tong Cheng; Dong Liu; John H Griffin; José A Fernández; Francis Castellino; Elliot D Rosen; Kenji Fukudome; Berislav V Zlokovic
Journal:  Nat Med       Date:  2003-02-03       Impact factor: 53.440

9.  Activated protein C prevents neuronal apoptosis via protease activated receptors 1 and 3.

Authors:  Huang Guo; Dong Liu; Harris Gelbard; Tong Cheng; Rae Insalaco; José A Fernández; John H Griffin; Berislav V Zlokovic
Journal:  Neuron       Date:  2004-02-19       Impact factor: 17.173

10.  RIGOR guidelines: escalating STAIR and STEPS for effective translational research.

Authors:  Paul A Lapchak; John H Zhang; Linda J Noble-Haeusslein
Journal:  Transl Stroke Res       Date:  2012-09-05       Impact factor: 6.829

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  42 in total

Review 1.  From blood-brain barrier to blood-brain interface: new opportunities for CNS drug delivery.

Authors:  William A Banks
Journal:  Nat Rev Drug Discov       Date:  2016-01-22       Impact factor: 84.694

2.  Activated protein C inhibits neutrophil extracellular trap formation in vitro and activation in vivo.

Authors:  Laura D Healy; Cristina Puy; José A Fernández; Annachiara Mitrugno; Ravi S Keshari; Nyiawung A Taku; Tiffany T Chu; Xiao Xu; András Gruber; Florea Lupu; John H Griffin; Owen J T McCarty
Journal:  J Biol Chem       Date:  2017-04-13       Impact factor: 5.157

Review 3.  Activated protein C: biased for translation.

Authors:  John H Griffin; Berislav V Zlokovic; Laurent O Mosnier
Journal:  Blood       Date:  2015-03-30       Impact factor: 22.113

Review 4.  Activated protein C in neuroprotection and malaria.

Authors:  Laurent O Mosnier
Journal:  Curr Opin Hematol       Date:  2019-09       Impact factor: 3.284

5.  Neuroprotection and vasculoprotection using genetically targeted protease-ligands.

Authors:  Padmesh S Rajput; Jessica A Lamb; Jose Á Fernández; Jilin Bai; Benedict R Pereira; I-Farn Lei; Jennifer Leung; John H Griffin; Patrick D Lyden
Journal:  Brain Res       Date:  2019-03-14       Impact factor: 3.252

6.  Protease activated receptor-1 mediates cytotoxicity during ischemia using in vivo and in vitro models.

Authors:  P S Rajput; P D Lyden; B Chen; J A Lamb; B Pereira; A Lamb; L Zhao; I-F Lei; J Bai
Journal:  Neuroscience       Date:  2014-09-28       Impact factor: 3.590

Review 7.  Cytoprotective-selective activated protein C therapy for ischaemic stroke.

Authors:  Laurent O Mosnier; Berislav V Zlokovic; John H Griffin
Journal:  Thromb Haemost       Date:  2014-09-18       Impact factor: 5.249

8.  Physiological cerebrovascular remodeling in response to chronic mild hypoxia: A role for activated protein C.

Authors:  Laurent Burnier; Amin Boroujerdi; Jose A Fernández; Jennifer V Welser-Alves; John H Griffin; Richard Milner
Journal:  Exp Neurol       Date:  2016-07-10       Impact factor: 5.330

Review 9.  2016 Scientific Sessions Sol Sherry Distinguished Lecturer in Thrombosis: Thrombotic Stroke: Neuroprotective Therapy by Recombinant-Activated Protein C.

Authors:  John H Griffin; Laurent O Mosnier; José A Fernández; Berislav V Zlokovic
Journal:  Arterioscler Thromb Vasc Biol       Date:  2016-10-06       Impact factor: 8.311

10.  Final Results of the RHAPSODY Trial: A Multi-Center, Phase 2 Trial Using a Continual Reassessment Method to Determine the Safety and Tolerability of 3K3A-APC, A Recombinant Variant of Human Activated Protein C, in Combination with Tissue Plasminogen Activator, Mechanical Thrombectomy or both in Moderate to Severe Acute Ischemic Stroke.

Authors:  Patrick Lyden; Kent E Pryor; Christopher S Coffey; Merit Cudkowicz; Robin Conwit; Ashutosh Jadhav; Robert N Sawyer; Jan Claassen; Opeolu Adeoye; Shlee Song; Peter Hannon; Natalia S Rost; Archana Hinduja; Michel Torbey; Jin-Moo Lee; Curtis Benesch; Michael Rippee; Marilyn Rymer; Michael T Froehler; E Clarke Haley; Mark Johnson; Jon Yankey; Kim Magee; Julie Qidwai; Howard Levy; E Mark Haacke; Miller Fawaz; Thomas P Davis; Arthur W Toga; John H Griffin; Berislav V Zlokovic
Journal:  Ann Neurol       Date:  2019-01-07       Impact factor: 10.422

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