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    Summary
    EudraCT Number:2008-003573-41
    Sponsor's Protocol Code Number:1101-202
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2008-07-16
    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 ConcernedUK - MHRA
    A.2EudraCT number2008-003573-41
    A.3Full title of the trial
    A randomised, double-blind, placebo-controlled study to assess the safety and activity of TRF-1101 on microvascular blood flow, vascular endothelial injury, and vasoocclusive pain in patients with sickle cell disease
    A.3.2Name or abbreviated title of the trial where available
    A Study of TRF-1101 in Sickle Cell Disease
    A.4.1Sponsor's protocol code number1101-202
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorTRF Pharma, 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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 Pentosan Polysulfate SP 54
    D.2.1.1.2Name of the Marketing Authorisation holderbene Arzneimittel GmbH
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNpentosan polysulfate sodium
    D.3.9.1CAS number 8000044895
    D.3.9.2Current sponsor codeTRF - 1101
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 placeboTablet
    D.8.4Route of administration of the placeboOral 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
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10040644
    E.1.2Term Sickle cell disease
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the safety and tolerability of pentosan polysulfate sodium administered daily for three months to patients with sickle cell disease
    E.2.2Secondary objectives of the trial
    1) To assess the pharmacodynamic activity of pentosan polysulfate sodium

    2) To assess activity of pentosan polysulfate sodium on vascular endothelial injury and vasoocclusive pain associated with sickle cell disease.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Be 18 years of age or older at the time of informed consent;

    2. Have a diagnosis of homozygous sickle cell anemia or sickle cell-beta thalassemia;

    3. Have had 2 - 10 documented pain crises in the past year (pain crises are defined as visits to a medical clinic, Emergency Department or hospital, being bedridden and requiring constant analgesia at home for at least three days, or having a three-day interruption of life’s activities [i.e., school, work, planned leisure activity] because of pain);

    4. If female and of child bearing potential, have a negative serum or urine pregnancy test and be using an effective birth-control method with a history of reliability for the individual patient (use of misepristone is not allowed);

    5. Be properly informed of the nature and risks of the clinical investigation, be willing and able to comply with all clinical investigation-related procedures and assessments, and sign an Institutional Review Board (EC) approved Informed Consent Form prior to entering the clinical investigation.
    E.4Principal exclusion criteria
    1. Have a history of abnormal bleeding, stroke, moya moya vascular malformations, or any other contraindication to anticoagulation;
    2. Be currently taking anticoagulant or thrombolytic medication;
    3. Be currently taking an endothelin receptor antagonist, e.g., bosentan (Tracleer®);
    4. Have a known sensitivity or allergy to heparin or PPS;
    5. Have a history of thrombocytopenia (platelet count < 100 x 10³/mm³) induced by heparin or PPS;
    6. Have had fewer than 2 documented pain crises in the past year;
    7. Have had a pain crisis within one month of screening or randomization;
    8. If currently on or recently discontinued hydroxyurea treatment, have initiated or discontinued treatment or changed regimen within the past 6 months;
    9. Have had a transfusion within last 120 days or have HbA% > 15% from prior transfusion;
    10. Creatinine levels > 1.53 mg/dL (135 micromol/L);
    11. ALT levels ≥ 3 times upper limit of normal;
    12. Platelet count < 100 x 10³/mm³;
    13. INR > 2.2;
    14. Be unable to tolerate oral medications;
    15. Have unreliable venous access;
    16. Be noncompliant with regular care;
    17. Have a positive pregnancy test, be currently lactating, or be trying to become pregnant;
    18. Have participation in an investigational drug or medical device study within previous 30 days;
    19. Have any other condition or circumstance that in the opinion of the Investigator makes the patient a poor candidate for participation in the study.
    E.5 End points
    E.5.1Primary end point(s)
    There are five outcomes to be measured in this study.
    1) Changes in blood flow as measured by laser Doppler technology. Blood flow in the microcirculation will be measured in three places on the subject’s forearm. It is anticipated that a combination of the four measurements will be used in the calculation of the statistics to be used for this outcome. The Statistical Analysis Plan will define this outcome in detail. It is also possible to analyse this outcome as a multi-level clustered model with clustering at the forearm level (four measurements at different locations) and over time (measurements at multiple visits) to control for the inherent correlation within the forearm and within the same subjects measured repeatedly over time.
    2) Change in circulating markers of blood vessel injury and inflammation: The change (as defined for outcome above) for each marker will be determined for use in descriptive analysis and in comparison of changes between treatment groups.
    3) Change in frequency of pain: The change in frequency of pain will be calculated as a median with a non-parametric comparison of frequency between treatment groups due to the inherently non-normal distribution of pain scores.
    4) Physician’s global assessment of response to treatment: This assessment would occur at every visit and would consist of a single reading ranging from -3 to +3. The change in this assessment would be calculated as the change from baseline represented in a multi-dimensional contingency table.
    5) Change in the level of P-selectin blocking activity: The change in the P-selectin blocking activity for each patient from the baseline measure (b) of P-selectin to the P-selectin at visits (f) conducted at weeks -4, 0, 1, 4, 8, 12, and +4 will be determined as b-f for each f and used in descriptive analyses. In addition, a comparison between treatment groups of the change in P-selectin blocking activity will be conducted.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Information not present in EudraCT
    E.6.2Prophylaxis Information not present in EudraCT
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Information not present in EudraCT
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence Information not present in EudraCT
    E.6.9Dose response Information not present in EudraCT
    E.6.10Pharmacogenetic Information not present in EudraCT
    E.6.11Pharmacogenomic Information not present in EudraCT
    E.6.12Pharmacoeconomic Information not present in EudraCT
    E.6.13Others Information not present in EudraCT
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Information not present in EudraCT
    E.7.1.1First administration to humans Information not present in EudraCT
    E.7.1.2Bioequivalence study Information not present in EudraCT
    E.7.1.3Other Information not present in EudraCT
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) Information not present in EudraCT
    E.7.4Therapeutic use (Phase IV) Information not present in EudraCT
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    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.8.3 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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 Information not present in EudraCT
    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
    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 months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    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 Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 state50
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 100
    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 Decision2008-08-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-01-15
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2009-04-03
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