LIPid Intensive Drug therapy for Sepsis Pilot (LIPIDS-P): Phase I/II clinical trial protocol of lipid emulsion therapy for stabilising cholesterol levels in sepsis and septic shock

Introduction Sepsis is a life-threatening, dysregulated response to infection. Both high-density lipoprotein and low-density lipoprotein cholesterol should protect against sepsis by several mechanisms; however, for partially unknown reasons, cholesterol levels become critically low in patients with...

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Main Authors: Marie Crandall, Lisa Jones, Faheem W Guirgis, Lauren Page Black, Martin Daniel Rosenthal, Morgan Henson, Jason Ferreira, Christiaan Leeuwenburgh, Colleen Kalynych, Lyle L Moldawer, Taylor Miller, Srinivasa T Reddy, Samuel S Wu, Frederick A Moore
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Language:English
Published: BMJ Publishing Group 2019-09-01
Series:BMJ Open
Online Access:https://bmjopen.bmj.com/content/9/9/e029348.full
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author Marie Crandall
Lisa Jones
Faheem W Guirgis
Lauren Page Black
Martin Daniel Rosenthal
Morgan Henson
Jason Ferreira
Christiaan Leeuwenburgh
Colleen Kalynych
Lyle L Moldawer
Taylor Miller
Srinivasa T Reddy
Samuel S Wu
Frederick A Moore
author_facet Marie Crandall
Lisa Jones
Faheem W Guirgis
Lauren Page Black
Martin Daniel Rosenthal
Morgan Henson
Jason Ferreira
Christiaan Leeuwenburgh
Colleen Kalynych
Lyle L Moldawer
Taylor Miller
Srinivasa T Reddy
Samuel S Wu
Frederick A Moore
author_sort Marie Crandall
collection DOAJ
description Introduction Sepsis is a life-threatening, dysregulated response to infection. Both high-density lipoprotein and low-density lipoprotein cholesterol should protect against sepsis by several mechanisms; however, for partially unknown reasons, cholesterol levels become critically low in patients with early sepsis who experience poor outcomes. An anti-inflammatory lipid injectable emulsion containing fish oil is approved by the Food and Drug Administration as parenteral nutrition for critically ill patients and may prevent this decrease in serum cholesterol levels by providing substrate for cholesterol synthesis and may favourably modulate inflammation. This LIPid Intensive Drug therapy for Sepsis Pilot clinical trial is the first study to attempt to stabilise early cholesterol levels using lipid emulsion as a treatment modality for sepsis.Methods and analysis This is a two-centre, phase I/II clinical trial. Phase I is a non-randomised dose-escalation study using a Bayesian optimal interval design in which up to 16 patients will be enrolled to evaluate the safest and most efficacious dose for stabilising cholesterol levels. Based on phase I results, the two best doses will be used to randomise 48 patients to either lipid injectable emulsion or active control (no treatment). Twenty-four patients will be randomised to one of two doses of the study drug, while 24 control group patients will receive no drug and will be followed during their hospitalisation. The control group will receive all standard treatments mandated by the institutional sepsis alert protocol. The phase II study will employ a permuted blocked randomisation technique, and the primary endpoint will be change in serum total cholesterol level (48 hours − enrolment). Secondary endpoints include change in cholesterol level from enrolment to 7 days, change in Sequential Organ Failure Assessment score over the first 48 hours and 7 days, in-hospital and 28-day mortality, lipid oxidation status, inflammatory biomarkers, and high-density lipoprotein function.Ethics and dissemination Investigators are trained and follow good clinical practices, and each phase of the study was reviewed and approved by the institutional review boards of each institution. Results of each phase will be disseminated through presentations at national meetings and publication in peer-reviewed journals. If promising, data from the pilot study will be used for a larger, multicentre, phase II clinical trial.Trial registration number NCT03405870.
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spelling doaj-art-930d668cd3464691a83b8d7095fa5bc62024-11-29T11:45:09ZengBMJ Publishing GroupBMJ Open2044-60552019-09-019910.1136/bmjopen-2019-029348LIPid Intensive Drug therapy for Sepsis Pilot (LIPIDS-P): Phase I/II clinical trial protocol of lipid emulsion therapy for stabilising cholesterol levels in sepsis and septic shockMarie Crandall0Lisa Jones1Faheem W Guirgis2Lauren Page Black3Martin Daniel Rosenthal4Morgan Henson5Jason Ferreira6Christiaan Leeuwenburgh7Colleen Kalynych8Lyle L Moldawer9Taylor Miller10Srinivasa T Reddy11Samuel S Wu12Frederick A Moore136 Surgery, University of Florida College of Medicine – Jacksonville, Jacksonville, Florida, USA5 Medicine, Division of Pulmonary and Critical Care Medicine, University of Florida College of Medicine – Jacksonville, Jacksonville, Florida, USA1 Emergency Medicine, University of Florida College of Medicine - Jacksonville, Jacksonville, Florida, USA1 Emergency Medicine, University of Florida College of Medicine - Jacksonville, Jacksonville, Florida, USA2 Surgery, University of Florida College of Medicine, Gainesville, Florida, USA1 Emergency Medicine, University of Florida College of Medicine - Jacksonville, Jacksonville, Florida, USA3 Pharmacy, University of Florida Health at Jacksonville, Jacksonville, Florida, USA4 Institute on Aging, University of Florida College of Medicine, Gainesville, Florida, USA1 Emergency Medicine, University of Florida College of Medicine - Jacksonville, Jacksonville, Florida, USA2 Surgery, University of Florida College of Medicine, Gainesville, Florida, USA1 Emergency Medicine, University of Florida College of Medicine - Jacksonville, Jacksonville, Florida, USA6 Department of Medicine, Division of Cardiology, UCLA, Los Angeles, California, USA8 Department of Biostatistics, University of Florida, Gainesville, Florida, USA2 Surgery, University of Florida College of Medicine, Gainesville, Florida, USAIntroduction Sepsis is a life-threatening, dysregulated response to infection. Both high-density lipoprotein and low-density lipoprotein cholesterol should protect against sepsis by several mechanisms; however, for partially unknown reasons, cholesterol levels become critically low in patients with early sepsis who experience poor outcomes. An anti-inflammatory lipid injectable emulsion containing fish oil is approved by the Food and Drug Administration as parenteral nutrition for critically ill patients and may prevent this decrease in serum cholesterol levels by providing substrate for cholesterol synthesis and may favourably modulate inflammation. This LIPid Intensive Drug therapy for Sepsis Pilot clinical trial is the first study to attempt to stabilise early cholesterol levels using lipid emulsion as a treatment modality for sepsis.Methods and analysis This is a two-centre, phase I/II clinical trial. Phase I is a non-randomised dose-escalation study using a Bayesian optimal interval design in which up to 16 patients will be enrolled to evaluate the safest and most efficacious dose for stabilising cholesterol levels. Based on phase I results, the two best doses will be used to randomise 48 patients to either lipid injectable emulsion or active control (no treatment). Twenty-four patients will be randomised to one of two doses of the study drug, while 24 control group patients will receive no drug and will be followed during their hospitalisation. The control group will receive all standard treatments mandated by the institutional sepsis alert protocol. The phase II study will employ a permuted blocked randomisation technique, and the primary endpoint will be change in serum total cholesterol level (48 hours − enrolment). Secondary endpoints include change in cholesterol level from enrolment to 7 days, change in Sequential Organ Failure Assessment score over the first 48 hours and 7 days, in-hospital and 28-day mortality, lipid oxidation status, inflammatory biomarkers, and high-density lipoprotein function.Ethics and dissemination Investigators are trained and follow good clinical practices, and each phase of the study was reviewed and approved by the institutional review boards of each institution. Results of each phase will be disseminated through presentations at national meetings and publication in peer-reviewed journals. If promising, data from the pilot study will be used for a larger, multicentre, phase II clinical trial.Trial registration number NCT03405870.https://bmjopen.bmj.com/content/9/9/e029348.full
spellingShingle Marie Crandall
Lisa Jones
Faheem W Guirgis
Lauren Page Black
Martin Daniel Rosenthal
Morgan Henson
Jason Ferreira
Christiaan Leeuwenburgh
Colleen Kalynych
Lyle L Moldawer
Taylor Miller
Srinivasa T Reddy
Samuel S Wu
Frederick A Moore
LIPid Intensive Drug therapy for Sepsis Pilot (LIPIDS-P): Phase I/II clinical trial protocol of lipid emulsion therapy for stabilising cholesterol levels in sepsis and septic shock
BMJ Open
title LIPid Intensive Drug therapy for Sepsis Pilot (LIPIDS-P): Phase I/II clinical trial protocol of lipid emulsion therapy for stabilising cholesterol levels in sepsis and septic shock
title_full LIPid Intensive Drug therapy for Sepsis Pilot (LIPIDS-P): Phase I/II clinical trial protocol of lipid emulsion therapy for stabilising cholesterol levels in sepsis and septic shock
title_fullStr LIPid Intensive Drug therapy for Sepsis Pilot (LIPIDS-P): Phase I/II clinical trial protocol of lipid emulsion therapy for stabilising cholesterol levels in sepsis and septic shock
title_full_unstemmed LIPid Intensive Drug therapy for Sepsis Pilot (LIPIDS-P): Phase I/II clinical trial protocol of lipid emulsion therapy for stabilising cholesterol levels in sepsis and septic shock
title_short LIPid Intensive Drug therapy for Sepsis Pilot (LIPIDS-P): Phase I/II clinical trial protocol of lipid emulsion therapy for stabilising cholesterol levels in sepsis and septic shock
title_sort lipid intensive drug therapy for sepsis pilot lipids p phase i ii clinical trial protocol of lipid emulsion therapy for stabilising cholesterol levels in sepsis and septic shock
url https://bmjopen.bmj.com/content/9/9/e029348.full
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