Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   37236   clinical trials with a EudraCT protocol, of which   6125   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2013-005454-30
    Sponsor's Protocol Code Number:C-935788-049
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-09-05
    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 ConcernedBulgarian Drug Agency
    A.2EudraCT number2013-005454-30
    A.3Full title of the trial
    A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura
    Открито продължение на проучването от фаза 3 на Fostamatinib Disodium за лечение на персистираща/хронична имунна тромбоцитопенична пурпура
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of Fostamatinib Disodium in the Treatment of Thrombocytopenic Purpura
    A.4.1Sponsor's protocol code numberC-935788-049
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02077192
    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 SponsorRigel Pharmaceuticals, 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 supportRigel Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRigel Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointAnn M. Lowe, MD
    B.5.3 Address:
    B.5.3.1Street Address1180 Veterans Boulevard
    B.5.3.2Town/ citySouth San Francisco
    B.5.3.3Post codeCA 94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number1 415 309 7672
    B.5.5Fax number1 650 624 1282
    B.5.6E-mailalowe@rigel.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namefostamatinib disodium tablets
    D.3.2Product code R935788
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNFostamatinib disodium
    D.3.9.1CAS number 914295-16-2
    D.3.9.2Current sponsor codeR935788 disodium hexahydrate
    D.3.9.3Other descriptive nameFOSTAMATINIB DISODIUM
    D.3.9.4EV Substance CodeSUB32625
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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.IMP: 2
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namefostamatinib disodium tablets
    D.3.2Product code R935788
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNfostamatinib disodium
    D.3.9.1CAS number 914295-16-2
    D.3.9.2Current sponsor codeR935788 disodium hexahydrate
    D.3.9.3Other descriptive nameFOSTAMATINIB DISODIUM
    D.3.9.4EV Substance CodeSUB32625
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Persistent/Chronic Immune Thrombocytopenic Purpura
    E.1.1.1Medical condition in easily understood language
    Thrombocytopenic Purpura
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10067644
    E.1.2Term Immune-mediated thrombocytopenic purpura
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to assess the long term safety of fostamatinib in subjects with persistent/chronic ITP
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to establish the long-term efficacy of fostamatinib in achieving and maintaining a stable platelet count in subjects who complete the treatment phase of Study C935788-047 or Study C935788-048, and to assess the pharmacokinetic (PK) profile of fostamatinib in subjects with persistent/chronic ITP.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject must be willing and able to give written informed consent by signing an IRB-approved Informed Consent Form prior to participating in any study-specific procedures.
    2. Subjects must have completed the Week 24 evaluation of Study C-935788C-047 or Study C-935788C-048 or have discontinued early (starting at Week 12) due to lack of response. No more than 7 days may have elapsed between the last day of treatment on the Study C-935788-047 or C-935788-048 and initial dosing (Day 1) in Study C-935788-049
    3. Male or female at least 18 years of age.
    4. Females must be either post-menopausal for at least 1 year or surgically sterile; or if female of child-bearing potential, must not be pregnant or lactating and must agree to use an acceptable method of birth control throughout the duration of the trial and for 30 days following the last dose. Acceptable methods of birth control are defined as: hormonal contraception (pill, injection or implant) used consistently for at least 30 days prior toenrollment, intrauterine device (IUD), double-barrier (ie, condom and spermicide, or condom and diaphragm), or complete abstinence.
    5. In the Investigator’s opinion, the subject has the ability to understand the nature of the study and any hazards of participation and to communicate satisfactorily with the Investigator.
    E.4Principal exclusion criteria
    1. Any subject who discontinued participation in Study C-935788-047 or Study C-935788-048 prior to Week 12 or for any reason other than lack of response. Exceptions may be considered on a case-by-case basis after consultation between the Investigator and Sponsor with the specific reason for the exception noted.
    2. Subjects with poorly controlled hypertension during Study C-935788-047 or Study C-935788-048, defined as persistent or repeated systolic ≥ 140 mmHg, or diastolic ≥ 90 mmHg whether or not the subject is receiving anti-hypertensive treatment.
    3. Subjects with the following laboratory abnormalities at the time of enrollment (Day 1): a leukocyte count < 2,000/μL, a neutrophil count of < 1,000/μL, lymphocyte count < 750/μL, Hgb < 10 g/dL, or transaminase levels (ALT, AST) > 1.5x ULN, bilirubin > 1.5x ULN, or estimated glomerular filtration rate (eGFR) < 30 mL/min.
    4. Subjects who have a significant infection, an acute infection such as influenza, or known inflammatory process at the time of enrollment into this study.
    5. Subjects who have received any blood or blood products within the 2 weeks prior to enrollment (IVIg or anti-D are allowed if used for rescue therapy).
    E.5 End points
    E.5.1Primary end point(s)
    Version 1: The first version of the primary efficacy endpoint is for the purpose of assessing efficacy among all patients while they are on active treatment in one of the prior studies, in the current extension study, or in both. This version of the primary efficacy endpoint is the achievement and maintenance of a stable platelet count defined as follows:
    1) Achievement of a platelet count of at least 50,000/μL within 12 weeks of beginning active treatment.
    2) Achievement of a sustained stable platelet response; defined as no two visits, at least 4 weeks apart, with a platelet count < 50,000/μL, without an intervening visit with a platelet count of ≥ 50,000/μL unrelated to rescue therapy, within a period of 12 months following initial achievement of the target platelet count (see above).
    Version 2: The second version of the primary efficacy endpoint is for the purpose of a within-subject, between-study comparison of fostamatinib and placebo among subjects randomized to placebo in either of the prior studies, This version of the primary efficacy endpoint is the achievement and maintenance of a stable platelet count defined as follows:
    1) Achievement of a platelet count of at least 50,000/μL within 12 weeks of beginning treatment (placebo treatment in the prior study and fostamatinib treatment in the present study) and
    2) Achievement of a sustained stable platelet response; defined as no two visits, at least 4 weeks apart, with a platelet count < 50,000/ μL, without an intervening visit with a platelet count of ≥ 50,000/ μL unrelated to rescue therapy, within a period of 12 weeks following initial achievement of the target platelet count (see above).
    E.5.1.1Timepoint(s) of evaluation of this end point
    - within 12 weeks of beginning active treatment
    - within a period of 12 weeks following initial achievement of the target platelet count
    E.5.2Secondary end point(s)
    • The duration of any stable platelet response.
    • Proportion of subjects in whom a reduction in the dose of concomitant ITP therapy can be achieved while maintaining an adequate platelet count.
    E.5.2.1Timepoint(s) of evaluation of this end point
    various study visits
    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 Yes
    E.6.7Pharmacodynamic No
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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 No
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA60
    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
    Austria
    Bulgaria
    Canada
    Czech Republic
    Denmark
    France
    Germany
    Hungary
    Italy
    Netherlands
    Poland
    Spain
    United Kingdom
    United States
    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
    The study will end upon completion of all protocol procedures
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 120
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 30
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 150
    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 Decision2014-11-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-12-10
    P. End of Trial
    P.End of Trial StatusCompleted
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2020 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA