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    Summary
    EudraCT Number:2009-014136-37
    Sponsor's Protocol Code Number:Thal-002
    National Competent Authority:Greece - EOF
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2010-06-15
    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 ConcernedGreece - EOF
    A.2EudraCT number2009-014136-37
    A.3Full title of the trial
    A pilot study on the safety and efficacy of haemopoietic stem cell mobilization (CD34+ cells) with MOZOBIL ± G-CSF, in adult patients diagnosed with beta-thalassaemia major.
    A.4.1Sponsor's protocol code numberThal-002
    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 SponsorUniversity of Washington
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    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 Mozobil
    D.2.1.1.2Name of the Marketing Authorisation holderGenzyme Europe B.V.,Gooimeer 10, NL-1411DD, Naarden, Netherlands
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation number(EU/3/04/227 )
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INN1, 1’ [1,4-phenylenebis (methylene)]-bis-1,4,8,11-tetraazacyclotetradecane
    D.3.9.1CAS number 110078-46-1
    D.3.9.3Other descriptive namePLERIXAFOR
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.2 ml in 20mg/ml
    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 Information not present in EudraCT
    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 nameGranulokine G-CSF
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNrecombinant methionylated human granulocyte-colony stimulating factor (G-CSF)
    D.3.9.3Other descriptive nameFilgrastim, rhGCSF recombinant human granulocyte-colony stimulating factor
    D.3.10 Strength
    D.3.10.1Concentration unit µg/ml microgram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300μg/1ml to 480μg/1.6ml
    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) 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 Yes
    D.3.11.13.1Other medicinal product typePharmaceutical product prepared via recombinant DNA technology. Produced by E.coli bacteria in which the human gene of granulocyte growth factor has been inserted.
    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
    Beta-thalassaemia major
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 12.0
    E.1.2Level LLT
    E.1.2Classification code 10004505
    E.1.2Term Beta thalassaemia
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 12.0
    E.1.2Level LLT
    E.1.2Classification code 10004514
    E.1.2Term Beta-thalassaemia
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 12.0
    E.1.2Level LLT
    E.1.2Classification code 10043391
    E.1.2Term Thalassaemia beta
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    i) To determine the safety of peripheral blood stem cell (PBSC) mobilization with Mozobil alone or with G-CSF +Mozobil in adults with b thalassemia major
    ii) To collect with Mozobil or Mozobil+G-CSF a total of at least 6X106CD34+ cells/kg for subsequent clinical scale transduction with a beta-globin lentiviral vector, in a clinical beta-globin gene transfer trial.
    E.2.2Secondary objectives of the trial
    i) To determine the clonogenic capacity of cells mobilized by Mozobil alone- or by G-CSF + Mozobil,
    ii) To determine the cells’ ability to be transduced with a recombinant lentivirus vector for b-globin
    iii) To determine the transduced cells’ potential to engraft in a xenograft model.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    a) Βeta- thalassemia major
    b) Age >18<50
    c) Karnofsky performance status ³80%
    d) Splenectomized patients or patients with spleen volume <800cm3 (only for the non splenectomized patients
    who will receive Mozobil + G-CSF) [V=0.523 x length x thickness x width (De Odorico I et al, 1999)]
    e) Compliant with regular transfusions and regular chelation
    f) Liver iron by MRI <280μmol/gr or 1.7msec by T2*MRI
    g) Heart iron by MRI >2.8 (SI/SD) or 9msec by T2*MRI
    h) Hepatitis B or C virus load negative by PCR
    i) Left ventricular ejection fraction (LVEF) >45% by echocardiogram
    j) Adequate respiratory function with DLCO >50%
    k) Negative pregnancy test, if female
    l) Ability to give informed consent and willingness to meet all the expected requirements of the protocol for the
    duration of the study
    E.4Principal exclusion criteria
    a) History of thrombosis or known thrombophilia
    b) Symptomatic viral, bacterial or fungal infection within 6 weeks prior eligibility evaluation
    c) Pregnancy or lactation
    d) HIV positivity
    e) History of malignancy, other than local skin cancer
    f) Other systematic disease non thalassemia-associated
    g) Splenectomized patients with platelet count >900,000 (only for the splenectomized patients who will receive
    low dose G-CSF+ Mozobil)
    h) Additional risk factors for thrombosis, including Factor V Leiden; antiphospholipid antibodies and less than
    50% of the lowest normal value for the following procoagulants: antithrombin 3, protein C, or protein S.
    E.5 End points
    E.5.1Primary end point(s)
    - the safety of Mozobil or Mozobil+G-CSF mobilization in splenectomized and non-splenectomized patients with b thalassemia major
    - the proportion of patients who will yield greater than or equal to 6X106 CD34+cells/kg by Mozobil alone
    - the proportion of patients who will yield cumulatively greater than or equal to 6X106 CD34+cells/kg by the combination of Mozobil+G-CSF and who had previously failed to reach the cell dose target either with G-CSF alone or Mozobil-alone
    - the number of apheresis days required to reach more than or equal to 6X106 CD34+cells/kg by Mozobil alone
    - the number of apheresis days required to cumulatively reach more than or equal to 6X106 CD34+cells/kg by the combination of Mozobil+G-CSF in patients who had previously failed to reach this cell dose target either by G-CSF or Mozobil.

    Importantly, in patients previously mobilized with G-CSF alone (Thal-001 study) or with Mozobil alone (Thal-002 study) the comparison of paired samples from each patient will eliminate inter-patient variability in the analysis, as each patient will serve as his or her own control. This will allow conclusions to be drawn on the relative efficacy of the two mobilization schemes that would otherwise be difficult to draw from this number of patients.
    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 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 Yes
    E.7.1.3.1Other trial type description
    Administration of approved products (Mozobil , Mozobil + GCSF) under a new indication
    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 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) Yes
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    combination Mozobil+G-CSF
    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 No
    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 months11
    E.8.9.1In the Member State concerned days15
    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) Information not present in EudraCT
    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 state20
    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 Decision2010-06-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-07-13
    P. End of Trial
    P.End of Trial StatusOngoing
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