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    Summary
    EudraCT Number:2013-001600-11
    Sponsor's Protocol Code Number:MP4CO-13-SCD-206
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2013-09-02
    Trial 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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2013-001600-11
    A.3Full title of the trial
    A Phase 2 Multi-center, Randomized, Double-blind, Comparator-Controlled Dose Finding Study to Evaluate MP4CO for the Acute Treatment of Vaso-occlusive Crises in Subjects with Sickle Cell Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate MP4CO for the Acute Treatment of Vaso-occlusive Crises in Subjects with Sickle Cell Disease
    A.4.1Sponsor's protocol code numberMP4CO-13-SCD-206
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01925001
    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 SponsorSangart, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSangart, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSangart Inc.
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street Address6175 Lusk Boulevard
    B.5.3.2Town/ citySan Diego
    B.5.3.3Post codeCA 92121
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1858-458-2388
    B.5.5Fax number+1858-4502498
    B.5.6E-mailMail_reg@sangart.com
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/09/698
    D.3 Description of the IMP
    D.3.1Product nameMP4CO
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNN/A
    D.3.9.2Current sponsor codeMP4CO
    D.3.9.3Other descriptive nameMP4CO
    D.3.9.4EV Substance CodeSUB32211
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number43
    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 Yes
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    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 infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Sickle cell disease (SCD) is an autosomal recessive disorder of the β globin gene of the hemoglobin molecule. One single point mutation results in a lifetime of chronic morbidity and mortality. Vaso-occlusive crises (VOCs), also known as pain crises, remain the hallmark of this disease and are the leading cause of hospitalization in SCD.
    E.1.1.1Medical condition in easily understood language
    Sickle cell disease is a genetic disorder that affects red blood cell circulation
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1. To determine the most effective dose of MP4CO at reducing the time to VOC resolution (and thereby decreasing the duration of hospitalization) in subjects with Sickle Cell Disease (SCD) admitted for vaso-occlusive crisis (VOC) management.

    2. To evaluate the safety and tolerability of MP4CO treatment administered during a VOC in subjects with SCD.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Enrollment Eligibility - Inclusion Criteria
    •Signed Informed Consent (and assent as required for minors)
    •Diagnosis of SCD (known HbSS or HbSß0)
    •Sixteen years of age or older
    •Prior history of at least one VOC requiring hospitalization within the last 24 months

    Randomization Eligibility - Inclusion Criteria
    •Subject has consented for participation in the MP4CO-13-SCD-206 trial
    • Diagnosis of an uncomplicated VOC consisting of all of the following:
    - Pain or tenderness affecting at least one part of the body
    - VAS pain level ≥ 6 cm at any point in time from start of VOC to randomization
    - Requires parenteral analgesia (including intravenous or subcutaneous routes)
    - Pain from VOC is the primary symptom at presentation
    •Requires admission to the hospital for VOC management
    •Pain is primarily due to VOC and not attributed to any other cause
    •Able to receive first dose of study drug within 12 hours of admission to the hospital for VOC management

    If a patient, not enrolled in the trial, presents with a VOC they may be enrolled into the trial at the time of VOC admission and proceed immediately to randomization if they meet all Enrollment Criteria and complete the Baseline Assessments prior to randomization.
    E.4Principal exclusion criteria
    Enrollment Eligibility - Exclusion Criteria
    •≥ 5 VOCs within the preceding 6 months requiring Emergency Room (ER) visits or hospital admissions
    •History of overt stroke or cerebral vascular accident within the previous 12 months
    •Remained in the hospital for ≥2 weeks (14 days) for VOC management within the previous 6 months
    •Known pulmonary hypertension based on an estimated tricuspid regurgitant jet velocity (TRJV) >2.90 m/s or definitive diagnosis by prior right heart catheterization
    •Baseline SaO2 level by pulse oximetry <92% on room air
    •Systemic hypertension (baseline systolic pressure ≥ 160 or diastolic pressure ≥ 90)
    •History of myocardial infarction, myocardial ischemia, or angina
    •On a chronic red blood cell transfusion therapy program (simple or exchange)
    •Renal dysfunction presenting with a GFR<60 mL/min/1.73m
    •Any diagnosis of a concurrent chronic debilitating disease that may affect the completion of the study or results of the study as determined by the investigator
    •Currently enrolled in any other investigational treatment study
    •Significant substance abuse.
    •Known to have HIV, active hepatitis B, or C infection, or active tuberculosis

    Randomization Eligibility - Exclusion Criteria:
    •Suspected diagnosis of Acute Chest Syndrome (ACS) as evidenced by one or more of the following at presentation to the medical facility:
    a.Fever >38.5°C with new hypoxia on pulse oximetry
    b.Chest radiographic findings of a new infiltrate
    c.Suspicious clinical exam for ACS (including but not limited to increased work of breathing, dyspnea, wheezing, hypoxia or rales)
    •Diagnosed or suspected systemic infection as evidenced by one or more of the following at presentation to the medical facility:
    a.Fever ≥39°C
    b.Fever >38.5°C accompanied by corrected White Blood Cell (WBC) count of ≥ 30 × 109/L or less than 2 × 109/L
    c.Positive findings suspicious for infections based on physical exam or/and diagnostic tests including but not limited to radiographic tests, laboratory tests, cerebral spinal fluid evaluation
    d.Complicated urinary tract infection (infection eg. involving the upper urinary tract i.e. pyelonephritis or with systemic symptomatology)
    •Hemoglobin level <5 g/dL (<50 g/L) at time of randomization
    •Priapism ongoing for >2 hours prior to randomization
    •Transient ischemic attack or seizure within the last 60 days
    •Admission for VOC within the last 60 days
    •Admission for ACS, significant bleeding, splenic sequestration, hepatic sequestration within the last 30 days.
    •≥5 VOCs requiring Emergency Room visits or hospital admissions within the preceding 6 months
    •Exchange red blood cell transfusion or simple packed red blood cell transfusion within the previous 30 days
    •SaO2 level by pulse oximetry <92% on room air
    •Hypertension (systolic pressure ≥160 mmHg or diastolic pressure ≥90) (within 1 hour prior to randomization)
    •Bradycardia (HR <60beats/min) (within 1 hour prior to randomization)
    •Renal dysfunction defined by a GFR <60 ml/min/1.73m2
    •Hepatic dysfunction (AST or GGT >3X ULN, or ALT >2XULN)
    •Pregnant or breastfeeding
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of this study is time to VOC resolution (TTVOCR) as assessed by the investigator. It is defined as the time from randomization to VOC resolution which is determined by achieving 2 out 3 of the following:
    • Cessation of intravenous and or subcutaneous opioid analgesia
    • Recovery of ambulation by at least 20% unless the subject was unable to ambulate for reasons other than a VOC crisis
    • Ready for discharge as defined by treating physician and subject
    E.5.1.1Timepoint(s) of evaluation of this end point
    Ongoing
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints are as follows:
    • Proportion of subjects with a reduction in VAS pain levels by 2 cm maintained ≥ 4 hours compared to worst score on day of admission.
    • Proportion of subjects with at least one return visit to ER within 7 days of discharge.
    • Proportion of subjects with at least one re-admission to hospital for treatment of VOC within 7 days of discharge.
    • Proportion of subjects with ACS complications during hospitalization for treated VOC.

    Safety endpoints will include the following:
    • Incidence of AEs and SAEs
    • Physical examination findings
    • Vital sign measurements
    • Urinalysis parameters
    • Hematology parameters
    • Blood chemistry parameters
    E.5.2.1Timepoint(s) of evaluation of this end point
    Ongoing
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    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) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    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 No
    E.8.1.6Cross over Yes
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Bahrain
    Belgium
    Brazil
    France
    Lebanon
    Netherlands
    Oman
    Qatar
    Saudi Arabia
    Turkey
    United Kingdom
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months18
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months18
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 200
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 40
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 150
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 Yes
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 200
    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)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-10-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-10-09
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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