Pharmacogenomic Biomarker Study for Recombinant Human Activated Protein C Treatment in Severe Sepsis

NCT ID: NCT01486524

Last Updated: 2011-12-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

3000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2011-10-31

Study Completion Date

2012-04-30

Brief Summary

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The overall purpose of the study is to determine whether either of the Improved Response Polymorphisms (IRPs) individually predicts a differential DrotAA treatment effect in patients with severe sepsis and high risk of death. This will be an international, multicenter, "prospective-retrospective", nonrandomized, controlled, outcome-blinded, genotype-blinded, matched-patients study. No prospective enrollment or treatment of patients will occur under this protocol. Retrospectively collected clinical data and DNA samples will be analyzed for existing cohorts of patients with severe sepsis who were previously treated with DrotAA (treatment group) or not (control group) as part of their standard care in an ICU.

Detailed Description

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This will be a multicenter, "prospective-retrospective", controlled, matched-patients study. Retrospective phenotypic data and DNA samples will be obtained from patient registries and clinical trials where the study hypotheses were not related to DrotAA treatment. The prospective aspect of this study will be the statistical testing of prespecified hypotheses regarding the IRP genotype as a predictive biomarker for differential DrotAA treatment effects.

To control for differences in standard of care in different countries and medical centers, the selection of matched control patients will be performed within each cohort. Control patients will be selected to match the DrotAA-treated patients using an algorithm that matches on baseline demographic and disease characteristics that may have influenced the decision to give DrotAA or that may impact survival. A propensity score (the likelihood for having received DrotAA treatment) will be derived using the matching variables that are common in all cohorts. The number of matched control patients for each treated patient will be variable, up to a maximum of 3.

The selection of the control patients via the matching algorithm will be conducted by an independent clinical research organization (CRO) in a blinded manner - specifically without knowledge of survival outcome, other outcome data, and genotype. A two-phase transfer of data from each center will be implemented to ensure that the selection of matched control patients is implemented in a blinded manner. The first step will involve the transfer of the baseline data for all variables needed to conduct the matching. Once the control patients have been identified for each cohort, the outcomes data will be transferred to the CRO in the second phase of data transfer.

Centralized genotyping using a validated Taqman®-based analytical method will be conducted on the DNA samples for all matched patients. The genotyping laboratory will be blinded to treatment and outcome.

The total number of patients in the available cohorts is \>23,000, with approximately 800 who have received DrotAA as part of their standard ICU-based care. After applying eligibility criteria to all patients and selecting the matched control patients, it is expected that the final analysis will include approximately 3000 patients.

Conditions

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Severe Sepsis Septic Shock

Keywords

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pharmacogenomic biomarker predictive marker prospective-retrospective propensity score matched-patients trial

Study Design

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Study Time Perspective

RETROSPECTIVE

Study Groups

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DrotAA Treatment Group

Patients with severe sepsis at high risk of death (INDICATED patients) who received treatment with drotrecogin alfa (activated (DrotAA) as part of standard care in ICU. The standard dosing regimen for DrotAA is 96 hours of continuous infusion at a dose of 24 ug/kg/hour. DrotAA is also known as recombinant human activated protein C.

No interventions assigned to this group

Control Group (non-DrotAA treated)

Patients with severe sepsis at high risk of death (INDICATED patients) who did not receive DrotAA treatment as part of their standard care in an ICU. The Control group patients will be selected to match the DrotAA-treated patients based on numerous clinical covariates, including propensity score (for DrotAA treatment).

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

1. Age ≥ 18 years
2. Severe sepsis (must meet a, b, and c below)

* Suspected or proven infection
* Systemic Inflammatory Response Syndrome (SIRS)(must meet 2 of 4 criteria)

* Temperature \< 36°C or \> 38°C
* Heart rate \> 90 beats/minute
* Respiratory rate \> 20 breaths/minute or PaC02 \< 32 mm Hg) or on mechanical ventilation
* White blood cell count \< 4,000/mm3 or \> 12,000/mm3
* At least one organ dysfunction due to sepsis based on definitions of clinically significant organ dysfunction

* Cardiovascular dysfunction \[must meet one of (1), (2), or (3) below\]:

* Systolic blood pressure ≤ 90 mmHg and pH ≤ 7.3
* Mean arterial pressure ≤ 70 mmHg and pH ≤ 7.3
* Reported use of a vasopressor alone is sufficient evidence of shock
* Pulmonary dysfunction: PaO2/FiO2 ≤ 300 mmHg
* Central Nervous System dysfunction: Glasgow Coma Scale ≤ 12
* Coagulation dysfunction: platelets ≤ 80,000/mm3
* Renal dysfunction: creatinine ≥ 2.0 mg/dL
* Hepatic dysfunction: bilirubin ≥ 2.0 mg/dL
3. High risk of death (one of a, b, or c below)

* APACHE II ≥ 25
* SAPS II ≥ 54
* Multiple organ dysfunction - two or more clinically significant organ dysfunctions (as defined above), which have occurred within 2 days of each other
4. Platelet counts ≥ 30,000/mm3
5. DrotAA status known

Exclusion Criteria

1. Patients with no DNA
2. Patients enrolled in local cohort more than 2 years before Xigris \[drotrecogin alfa activated)\] was commercially available
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sirius Genomics Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Djillali Annane, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

University of Versailles

Alexandra DJ Mancini, MSc

Role: STUDY_DIRECTOR

Sirius Genomics Inc.

Locations

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Emory University School of Medicine

Atlanta, Georgia, United States

Site Status

Johns Hopkins University, Bayview Medical Center

Baltimore, Maryland, United States

Site Status

Harvard University School of Public Health

Boston, Massachusetts, United States

Site Status

Vanderbilt University Schoo of Medicine

Nashville, Tennessee, United States

Site Status

University of British Columbia and Providence Health Care, St. Paul's Hospital

Vancouver, British Columbia, Canada

Site Status

University of Versailles, Hospital Raymond Poincaré (AP-HP)

Garches, , France

Site Status

Université Paris Descartes, Sorbonne Paris Cité, Cochin Hotel-Dieu University Hospital

Paris, , France

Site Status

Imperial College London, Charing Cross Hospital

London, , United Kingdom

Site Status

Countries

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United States Canada France United Kingdom

References

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Annane D, Mira JP, Ware LB, Gordon AC, Hinds CJ, Christiani DC, Sevransky J, Barnes K, Buchman TG, Heagerty PJ, Balshaw R, Lesnikova N, de Nobrega K, Wellman HF, Neira M, Mancini ADJ, Walley KR, Russell JA. Pharmacogenomic biomarkers do not predict response to drotrecogin alfa in patients with severe sepsis. Ann Intensive Care. 2018 Jan 31;8(1):16. doi: 10.1186/s13613-018-0353-2.

Reference Type DERIVED
PMID: 29388048 (View on PubMed)

Other Identifiers

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SGX301

Identifier Type: -

Identifier Source: org_study_id