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    Summary
    EudraCT Number:2013-002986-21
    Sponsor's Protocol Code Number:MST-188-01
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-04-09
    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 number2013-002986-21
    A.3Full title of the trial
    Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC): A Phase 3, Randomized, Double-Blind, Placebo-Controlled Multicenter Clinical Trial of MST-188 (purified poloxamer 188) Injection in Subjects with Sickle Cell Disease Experiencing Vaso-Occlusive Crisis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of Purified Poloxamer 188 in Subjects with Sickle Cell Disease Experiencing a Vaso-occlusive Crisis
    A.3.2Name or abbreviated title of the trial where available
    Evaluation of Purified Poloxamer 188 in Subjects in Crisis (EPIC)
    A.4.1Sponsor's protocol code numberMST-188-01
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01737814
    A.5.4Other Identifiers
    Name:IND NumberNumber:31,246
    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 SponsorMast Therapeutics, 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 supportMast Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPharm-Olam International Belgium BVBA
    B.5.2Functional name of contact pointCountry Manager
    B.5.3 Address:
    B.5.3.1Street AddressDorp 14a
    B.5.3.2Town/ cityTestelt
    B.5.3.3Post code3272
    B.5.3.4CountryBelgium
    B.5.4Telephone number3213338071
    B.5.5Fax number3213338075
    B.5.6E-mailsara.weytjens@pharm-olam.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/3/13/1112
    D.3 Description of the IMP
    D.3.1Product nameMST-188 (purified poloxamer 188) Injection
    D.3.2Product code MST-188
    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 INNPurified poloxamer 188
    D.3.9.1CAS number 9003-11-6
    D.3.9.2Current sponsor codePurified poloxamer 188 (vepoloxamer)
    D.3.9.4EV Substance CodeSUB16038MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 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 with Vaso-Occlusive Crisis
    E.1.1.1Medical condition in easily understood language
    Sickle Cell Disease in hospitalized subjects Experiencing a Vaso-Occlusive Crisis
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level LLT
    E.1.2Classification code 10040644
    E.1.2Term Sickle cell disease
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to demonstrate the efficacy of MST-188 in reducing the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease (SCD). The duration of VOC will be measured from the time of randomization to the time at which a subject receives the last dose of parenteral opioid analgesia for the treatment of VOC prior to hospital discharge.
    E.2.2Secondary objectives of the trial
    The secondary objectives are:
    o To compare the re-hospitalization rate (for VOC) between the treatment arms.
    o To compare the occurrence of acute chest syndrome (ACS) between the treatment arms.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    The sub-studies Pharmacodynamic laboratory biomarkers will be evaluated in a sub-study to this trial.
    The effects of tissue oxygen saturation will be evaluated in a sub-study to this trial. These sub-studies will only be conducted in United States.
    E.3Principal inclusion criteria
    1. Age 4 through 65 years
    2. Subject has a confirmed diagnosis of HbSS, HbSC, HbSβ+thal, or HbSβ0thal
    3. Subject is experiencing acute pain typical of vaso-occlusive crisis requiring treatment with parenteral analgesia
    4. Subject requires hospitalization
    E.4Principal exclusion criteria
    1. Subject has acute chest syndrome
    2. Subject's laboratory results indicate inadequate organ function
    3. Subject is pregnant or nursing an infant
    4. Subject had a painful crisis requiring hospitalization within the preceding 14 days or has experienced > 5 hospitalizations for VOC in the prior 6 months
    5. Subject has been transfused within the past 14 days
    6. Subject is hospitalized for a condition other than VOC
    7. Subject has complications related to SCD
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of the study is the time from randomization to last parenteral opioid analgesic dose for the treatment of VOC prior to hospital discharge.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The time from randomization to last parenteral opioid analgesic dose for the treatment of VOC prior to hospital discharge.
    E.5.2Secondary end point(s)
    Secondary Endpoints
    1. Re-hospitalization for VOC: The number and percent of subjects who are re-hospitalized for VOC within 14 days after discharge from the hospital.
    2. Occurrence of ACS: The number and percent of subjects who meet the protocol definition of ACS within 120 hours of randomization.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Within 14 days after discharge from the hospital.
    2. Within 120 hours of randomization
    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 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 No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 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 EEA4
    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
    Belgium
    Brazil
    Dominican Republic
    Lebanon
    Netherlands
    Oman
    Panama
    Saudi Arabia
    Spain
    Turkey
    United Kingdom
    United States
    Jordan
    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: 30-day post-hospitalization contact: Last subject will be queried regarding re-hospitalization for VOC 30 days (±2 days) following discharge. If this date is within 2 days of the 30-Day Post-Infusion Follow-up visit, the visits may be combined.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months7
    E.8.9.2In all countries concerned by the trial days0
    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: 232
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 135
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 97
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 155
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Minors must provide assent to participate in this study at an age-appropriate level determined and approved by the Institutional Review Board(IRB)/Independent Ethics Committee (IEC) for the study center.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 44
    F.4.2.2In the whole clinical trial 388
    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)
    Subjects completing this study may be eligible to participate in an open-label extension study to evaluate the safety of repeat exposures of MST-188 in VOC. A separate protocol is being contemplated based on understandings gained during the course of this study, input from experts and approval of appropriate ethics committees and regulatory bodies.
    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 Decision2014-06-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-06-16
    P. End of Trial
    P.End of Trial StatusCompleted
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