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    Summary
    EudraCT Number:2007-003086-40
    Sponsor's Protocol Code Number:PIX001
    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:2007-11-21
    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 number2007-003086-40
    A.3Full title of the trial
    A Phase I/II study of Pixantrone in Patients with an Aggressive Relapsing Remitting (RR) or Secondary Progressive (SP) Multiple Sclerosis (PIXAMS)
    A.4.1Sponsor's protocol code numberPIX001
    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 SponsorFondation Charcot Stichting
    B.1.3.4CountryBelgium
    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 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 namePixantrone
    D.3.2Product code BBR 2778
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNPixantrone
    D.3.9.1CAS number 144675-97-8
    D.3.9.2Current sponsor codeBBR 2778
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    aggressive relapsing remitting (RR) or secondary progressive (SP) multiple sclerosis
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1. To determine if four consecutive administrations of 120 mg/m² each of PIX (equivalent to 12 mg/m² mitoxantrone) will provide an effective immunosuppression as reflected by a lymphopenia ≤ 1000/mm³ after completion of PIX administration.
    2. To evaluate the effects of PIX on brain inflammatory processes as demonstrated by MRI: accumulation of new lesions (gadolinium enhancing and/or T2 lesions).
    E.2.2Secondary objectives of the trial
    1. cardiac tolerance monitored by echocardiography and cardiotroponin T
    2. immune changes as demonstrated by lymphocyte subset typing (CD3, CD4, CD8, B cells) and other immunological parameters
    3. vital signs
    4. side effects and adverse reactions
    5. effects of treatment on relapse rate and disability progression (EDSS, 9HP test, 25 FWT).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. patients with aggressive relapsing-remitting MS who failed to respond to approved immunomodulatory drugs (interferons beta, glatiramer acetate) and who cannot or do not wish to receive mitoxantrone or tysabri or patients with aggressive relapsing-progressive or secondary progressive MS who cannot or do not wish to receive mitoxantrone
    2. patients within ≥ 18 and ≤ 55 years of age
    3. clinically definite RR or SP MS, according to Lublin and Reingold definition and the McDonald criteria
    4. Expanded Disability Status Scale Score (EDSS) ≥ 2.5 and ≤ 5.5
    5. patients in the RR phase with a poor prognosis because of frequent (≥ 2/year) and severe relapses (transient EDSS ≥3) or patients in the SP phase with clinical signs or symptoms of rapid progression over the last year equivalent to an increase in EDSS of >/ 1 point
    6. ≥ 1 gadolinium enhancing lesion(s) within 3 months prior to screening or welldocumented new T2 lesion within 3 months prior to screening
    7. left ventricular ejection fraction ≥ 50%
    8. standard laboratory parameters within the normal range, including hematology (lymphocytes > 1000/mm³, ANC (absolute neutrophil count) ≥2000/ml, thrombocytes ≥100.000/ml)
    9. male or female patients:
    NB: female patients of child-bearing potential should have a negative pregnancy test at the time of inclusion
    male and female patients within reproductive ages should agree to practice effective contraception during the trial and for 6 months following the last administration of PIX.
    10. have given written informed consent prior to treatment, with the understanding that consent may be withdrawn at any time without prejudice
    E.4Principal exclusion criteria
    1. previous treatment with immunosuppressants (Mitoxantrone, Cyclophosphamide, Methotrexate, Azathioprine, rituximab, alemtuzumab …) in the past year
    2. in case of a recent relapse, the time from start of relapse to study entry should be at least one month
    3. any high dose (500 mg or more) corticosteroids or ACTH in the month preceding study entry
    4. patients with cardiac risk factors: prior history of congestive heart failure, myocardial infarction, uncontrolled ischemic heart disease or hypertension
    5. patients currently treated with potential cardiotoxic drugs
    6. patients who are at medical risks because of systemic disease which might interfere with patient safety (e.g.: impairment of renal or hepatic function, severe infection, alcohol abuse or drug addiction)
    7. patients with impaired intellectual functions or psychiatric disorders preventing normal cooperation during the trial
    8. participation in any investigational study within 4 weeks prior to treatment start; patient must have recovered from all side effects of other investigational therapy
    9. history of, or clinical signs or symptoms suggestive of HIV, HBV, HCV
    10. any circumstance at the time of study entry that would preclude completion of the study or the required follow-up.
    E.5 End points
    E.5.1Primary end point(s)
    1. Proportion of patients with a lymphopenia ≤ 1000/mm³ at month 3.
    2. Decrease in brain inflammatory processes as demonstrated by MRI: accumulation of new lesions (gadolinium enhancing and/or T2 lesions):
    number of patients with new Gd+/T2 lesions
    mean total number of Gd+/T2 lesions
    mean total number of Gd+/T2 lesions/scan
    mean number of new Gd+/T2 lesions
    mean number of new Gd+/T2 lesions/scan
    MRI scans will be performed within 3 months before screening, and at months 6 and 12.
    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 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) Yes
    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 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 Yes
    E.8.1.7.1Other trial design description
    open, label, multicentre, non-comparative
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 concerned1
    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 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 No
    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 years
    E.8.9.1In the Member State concerned months21
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months21
    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.1.3Elderly (>=65 years) No
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 20
    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)
    NA
    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 Decision2008-02-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-02-25
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2012-05-07
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