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    Summary
    EudraCT Number:2011-005815-10
    Sponsor's Protocol Code Number:AJSEB001
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-04-03
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2011-005815-10
    A.3Full title of the trial
    Developmental Clinical Sciences: Does GM-CSF restore effective neutrophil function in critically ill patients?
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Does GM-CSF ( a drug which stimulates the production of white blood cells) restore effective neutrophil (the key white blood cell in fighting infection) function in critically ill patients?
    A.3.2Name or abbreviated title of the trial where available
    Does GM-CSF restore neutrophil phagocytosis in critical illness?
    A.4.1Sponsor's protocol code numberAJSEB001
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNewcastle Upon Tyne Hospitals NHS Foundation Trust
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportMedical Research Council
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNewcastle Upon Tyne Hospitals NHS Foundation Trust
    B.5.2Functional name of contact pointProfessor John Simpson,
    B.5.3 Address:
    B.5.3.1Street AddressInst of Cellular Med,4th Floor,William Leech Building, Medical School,Newcastle University
    B.5.3.2Town/ cityFramlington Place, Newcastle Upon Tyne
    B.5.3.3Post codeNE2 4HH
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number01912227770
    B.5.5Fax number01912220723
    B.5.6E-mailj.simpson@ncl.ac.uk
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Leukine
    D.2.1.1.2Name of the Marketing Authorisation holderBayer Health Care Pharmaceuticals, LLC. Seattle. WA 98101
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameLeukine (Sargramostim)
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSargramostim
    D.3.9.1CAS number 123774-72-1
    D.3.9.3Other descriptive nameYeast derived recombinant human GM-CSF
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Complement mediated dysfunctional neutrophil phagocytosis in critical illness.
    E.1.1.1Medical condition in easily understood language
    The reduced ability of neutrophils (a type of white blood cell)to eat bacteria (phagocytosis)in patients on intensive care.
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10061314
    E.1.2Term Neutrophil function disorder
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level LLT
    E.1.2Classification code 10017500
    E.1.2Term Functional disorders of polymorphonuclear neutrophils
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10061867
    E.1.2Term Neutrophil function test abnormal
    E.1.2System Organ Class 10022891 - Investigations
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Will GM-CSF (a drug used to stimulate white blood cells) be able to restore the ingestion of germs by neutrophils (the most important white blood cells in fighting bacterial and fungal infection) to fight off infection in critically ill patients on intensive care?
    E.2.2Secondary objectives of the trial
    Will GM-CSF ( a drug used to stimulate white blood cells) have any effect on
    1)the function of monocytes (another type of white blood cell involved in fighting infection)
    2)the incidence of patients developing secondary infection while in intensive care
    3)death rates of patients requiring intensive care
    4)length of stay on the intensive care unit
    5)the function of patients organs while in intensive care
    6)the length of mechanical ventilation of patients on intensive care
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients admitted to intensive care unit within the last 72 hours
    Fulfill criteria for systemic inflammatory response syndrome (SIRS)
    Have required exogenous support of one or more organ systems (invasive ventilation, inotropes or haemofiltration) during current ICU stay
    Survival over next 48 hours considered to be the most likely outcome over this time by the attending clinician at the time of enrollment
    Neutrophil phagocytic capacity is low (i.e. < 50% neutrophil phagocytic capacity)
    E.4Principal exclusion criteria
    Absence/refusal of informed consent
    Current prescription of a colony stimulating factor
    Any history of adverse reaction/allergy to GM-CSF
    Total white cell count > 30x10^9/litre at the time of screening
    Haemoglobin <7.5g/dl at the time of screening
    Age < 18 years
    Pregnancy or lactation
    Known in-born errors of neutrophil metabolism
    Known haematological malignancy and/or known to have > 10% peripheral blood blast cells
    Known aplastic anaemia or pancytopaenia
    Platelet count <50x10^9/litre
    Chemotherapy or radiotherapy within the last 24 hours
    Solid organ or bone marrow transplantation
    Use of maintenance immunosuppressive drugs other than maintenance corticosteroids (allowed up to prednisolone 10mg/day or equivalent)
    Known human immunodeficiency virus (HIV) infection
    Active connective tissue disease (eg rheumatoid disease, systemic lupus erythematosus) requiring active pharmacological treatment
    ST segment elevation myocardial infarction, acute pericarditis (by ECG criteria) or pulmonary embolism (radiologically confirmed) in the previous week
    Involvement in any study involving an investigational medicinal product in the previous 30 days.
    E.5 End points
    E.5.1Primary end point(s)
    The primary outcome is neutrophil phagocytic capacity 2 days after administration of GM-CSF or placebo (as measured by percentage of neutrophils ingesting greater than or equal to 2 zymosan particles ex vivo).
    E.5.1.1Timepoint(s) of evaluation of this end point
    This endpoint will be evaluated after 2 days of commencing GM-CSF or placebo. Several secondary endpoints will also be evaluated.
    (Where a trained researcher is not available blood may be taken the following day)
    E.5.2Secondary end point(s)
    1.Neutrophil phagocytic capacity measured as 'area under the curve' over the study period
    2.Sequential neutrophil phagocytic capacity on alternate study days (and days 9 and 10)- to determine sustainability of any observed effects in the primary endpoint.
    3.Sequential organ failure score (SOFA)
    4.Length of stay in ICU
    5.The incidence of ICUAIs (as defined by Hospitals in Europe Link for Infection Control Surveillance (HELICS) criteria)
    6.30 day mortality
    7.Other assessments of neutrophil function which may include but not be limited to ROS generation, migratory capacity, and apoptotic rate
    8.Monocyte HLA-DR expression on alternate study days (and days 8 & 9).
    9.Total white cell count
    10.Neutrophil, monocyte, eosinophil and lymphocyte counts
    11.Safety including full blood count, urea and electrolytes, liver function tests, neutralising antibodies to GM-CSF, recording and reporting of serious adverse events
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Day 9
    2. **Days 0,2,4,6,8 & 9
    3. Days 0-10,20,30
    4. Days 0-10,20,30
    5. Days 0-10,20,30
    6. Day 30
    7. **Days 0,2,4,6,8 & 9
    8. **Days 0,2,4,6,8 & 9
    9. Days 0-9
    10.Days 0-9
    11.Days 0-9

    ** Where a trained researcher is not available blood may be taken the following day
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Dose finding study
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS. Data collection for secondary endpoints including 30 day mortality and ICU length of stay will continue for 30 days after entry of the last patient into the study.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days29
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months7
    E.8.9.2In all countries concerned by the trial days26
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 31
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 31
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Entry criteria require patients to be admitted to intensive care and in need of organ support. As such, the majority if not all of the patients will be sedated for mechanical ventilation and will therefore be unable to give prospective consent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state92
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    No additional treatment available after the trial. Usual care will resume. This treatment is being investigated as a short term intervention only.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2012-05-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-02-14
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-02-12
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