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    Summary
    EudraCT Number:2007-004308-11
    Sponsor's Protocol Code Number:GTG002.07
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2009-07-06
    Trial results View 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 number2007-004308-11
    A.3Full title of the trial
    PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY
    FOR THE WISKOTT-ALDRICH SYNDROME
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Gene therapy for WAS
    A.3.2Name or abbreviated title of the trial where available
    Gene Therapy for WAS , version 5.0
    A.4.1Sponsor's protocol code numberGTG002.07
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01347242
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/266/2013
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGenethon
    B.1.3.4CountryFrance
    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 supportGenethon
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGenethon
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address1 bis rue de l'international
    B.5.3.2Town/ cityEvry
    B.5.3.3Post code91002
    B.5.3.4CountryFrance
    B.5.4Telephone number33169472900
    B.5.5Fax number33169471946
    B.5.6E-mailclinical_development@genethon.fr
    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/3/13/1196
    D.3 Description of the IMP
    D.3.1Product nameAutologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector
    D.3.4Pharmaceutical form Solution for injection
    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.3.9.3Other descriptive nameAUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number0.5E06 cells /Kg
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    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
    Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopenia, recurrent infections,eczema and associated with a high incidence of auto-immunity and of lymphoid malignancies. Over 150 unique mutations in the WAS gene have been identified.Loss-of-function mutations in this gene have widespread consequences on hematopoietic lineages.
    E.1.1.1Medical condition in easily understood language
    The Wiskott-Aldrich Sydrome (WAS) is a genetic disorder that affects boys. It is caused by errors in a gene that makes a part of the blood and immune system.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.1
    E.1.2Level PT
    E.1.2Classification code 10047992
    E.1.2Term Wiskott-Aldrich syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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 efficacy of heamatopoietic stem cell gene therapy in WAS patients based on the clinical improvement in at least one of the following clinical parameters, depending on the patient’s symptomatology at study entry: eczema status, frequency and severity of infections, of bruising and bleeding episodes and of autoimmune disorders and consequently to assess the number of disease-related days of hospitalisation
    E.2.2Secondary objectives of the trial
    - To assess the safety of heamatopoietic stem cell gene therapy in WAS patients
    - To asses the efficacy of heamatopoietic stem cell gene therapy on the evolution of microthrombocytopenia and its treatment
    - To assess the efficacy of heamatopoietic stem cell gene therapy on the evolution of the other haematological parameters including WASP expression
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.
    a. Males of all ages
    b. Severe WAS (clinical score 3 – 5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
    c. Molecular confirmation by WAS gene DNA sequencing

    2. Lack of HLA-genotypically identical bone marrow OR of a 10/10 antigen HLA-matched unrelated donor or cord blood after 3 month search

    3. Parental, guardian, patient signed informed consent/assessment

    4. Willing to return for follow-up during the 2 year study

    5. Only for patients who have received previous allogenic haematopoietic stem cell transplant:
    a. Failed allogenic haematopoietic stem cell transplant
    b. Contraindication to repeat allogeneic transplantation for example severe graft versus host disease
    E.4Principal exclusion criteria
    1.
    a. Patient with HLA-genotypically identical bone marrow
    b. Patient with 10/10 antigen HLA-matched unrelated donor or cord blood

    2.
    a. Contraindication to leukapheresis
    i. anaemia (Hb < 8g/dl)
    ii. cardiovascular instability
    iii. severe coagulopathy
    b. Contraindication to bone marrow harvest
    c. Contraindication to administration of conditioning medication

    3. HIV positive patient
    E.5 End points
    E.5.1Primary end point(s)
    -Improvement at 24 months in at least one of the following clinical conditions depending on the patient’s clinical symptomatology at study entry:

    ­- Improvement in the eczema status at 24 months as compared with the baseline status at study entry

    ­- Reduction in the frequency and severity of infection episodes at 24 months as compared with the baseline status and the patient’s historical data collected over the 24 months prior to study entry

    ­- Reduction in the frequency and severity of bruising and bleeding episodes at 24 months evaluated by clinical examination as compared with the baseline status at study entry and the patient’s historical data collected over the 24 months prior to study entry

    ­- Reduction in the frequency and severity of autoimmune disorders at 24 months as compared with the baseline status at study entry

    ­- Reduction in the number of disease-related days of hospitalisation as compared with the patient’s historical data collected over the 24 months prior to study entry
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 months
    E.5.2Secondary end point(s)
    1. Safety

    ­ Safety of gene therapy will be assessed through the occurrence of adverse events reported during the course of the study.

    ­ In addition, any change in medical conditions including weight, vital signs (Blood pressure, pulse rate), ECG and laboratory exams will also be assessed during the course of the study.

    ­ Safety of gene therapy will be assessed by lack of the detection of replication competent lentivirus (RCL) at 3, 6, 12 and 24 months post gene therapy.

    ­ Safety of gene therapy will be assessed by the analysis of the lentivirus integration sites performed in different cell subpopulations to investigate specific clonal expansions at 6, 12, 18 and 24 months post gene therapy and quantification of transgene copy numbers determined on sorted cell populations by real-time PCR methodology at 6 weeks and 1, 3, 6, 9, 12, 18 and 24 months post gene therapy.

    2- Efficacy

    ­ Improvement of microthrombocytopenia at 3, 6, 12 and 24 months as compared with the baseline evaluation at study entry

    ­ Decrease in the number and volume of platelet transfusions at 24 months as compared with the patient’s historical data collected over the 24 months prior to study entry

    ­ Evidence of sustained engraftment of WASP-expressing tranduced cells at 6 weeks and 1, 3, 6, 9, 12, 18 and 24 months post gene therapy

    ­ Reconstitution of humoral and cell mediated immunity at 9, 12, 18 and 24 months as compared with baseline evaluation at study entry
    E.5.2.1Timepoint(s) of evaluation of this end point
    1, 3, 6, 9, 12, 18 and 24 months depending on the endpoint.
    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 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) Yes
    E.7.1.1First administration to humans Yes
    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 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.8.3 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 concerned2
    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 No
    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
    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 months7
    E.8.9.1In the Member State concerned days31
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months7
    E.8.9.2In all countries concerned by the trial days31
    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: 5
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state5
    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)
    The study is a single dose gene therapy medicinal product.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2010-01-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2008-02-26
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-11-13
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