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    Summary
    EudraCT Number:2012-000242-35
    Sponsor's Protocol Code Number:G1XCGD.01
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2013-04-29
    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 ConcernedGermany - PEI
    A.2EudraCT number2012-000242-35
    A.3Full title of the trial
    A phase I/II, non randomized, multicenter, open-label study of autologous CD34+ cells transduced with the G1XCGD Lentiviral vector in patients with X-Linked Chronic Granulomatous Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Gene therapy with autologous genetically-modified CD34+ cells for X-linked Chronic Granulomatous Disease (X-CGD)
    A.3.2Name or abbreviated title of the trial where available
    Phase I/II G1XCGD.01 study : an ex-vivo gene therapy for X-CGD patients
    A.4.1Sponsor's protocol code numberG1XCGD.01
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01855685
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    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.4.1Name of organisation providing supportNet4CGD
    B.4.2CountryEuropean Union
    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 pointMalika SOUQUIERES
    B.5.3 Address:
    B.5.3.1Street Address1 bis rue de l'internationale - BP 60
    B.5.3.2Town/ cityEVRY Cedex
    B.5.3.3Post code91002
    B.5.3.4CountryFrance
    B.5.4Telephone number+33 1 69 47 25 74
    B.5.5Fax number+33 1 69 47 19 46
    B.5.6E-mailmsouquieres@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/3/12/957
    D.3 Description of the IMP
    D.3.1Product nameSuspension of autologous CD34+cells transduced with the G1XCGD viral vector
    D.3.2Product code G1XCGD transduced CD34+ cells
    D.3.4Pharmaceutical form Suspension 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 EX-VIVO WITH THE PCCLCHIMGP91/VSVG LENTIVIRAL VECTOR
    D.3.9.4EV Substance CodeSUB122488
    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
    X-linked Chronic Granulomatous Disease
    E.1.1.1Medical condition in easily understood language
    Chronic granulomatous disease is an inherited disorder in which immune system cells called phagocytes do not function properly. This results in ongoing and severe infections.
    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 14.1
    E.1.2Level PT
    E.1.2Classification code 10008906
    E.1.2Term Chronic granulomatous disease
    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
    -Evaluation of safety
    -Evaluation of efficacy by biochemical and functional reconstitution in progeny of engrafted cells and stability at 12 months)
    E.2.2Secondary objectives of the trial
    -Clinical efficacy and longitudinal evaluation of clinical effect in terms of augmented immunity against bacterial and fungal infection
    -Transduction of CD34+ haematopoietic cells from X-CGD patients by ex vivo lentivirus-mediated gene transfer
    -Evaluation of engraftment kinetics and stability
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Male X-CGD patients > 23 months of age
    -Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or reduction > 70% of the biochemical activity of the NAHPD-oxidase
    -At least one ongoing or resistant severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy
    -No HLA-matched donor available after 3 months search
    -No co-infection with Human Immunodeficiency Virus (HIV) or hepatitis B virus (HBsAg positive) or hepatitis C virus (HCV RNA positive)
    -Written informed consent for adult patient
    -Parental/guardian and where appropriate child’s signed consent/assent
    E.4Principal exclusion criteria
    -10/10 HLA identical (A,B,C,DR,DQ) family or unrelated or cord blood donor unless there is deemed to be an unacceptable risk associated with an allogeneic procedure
    -Contraindication for leukapheresis (anaemia Hb <8g/dl, cardiovascular instability, severe coagulopathy)
    -Contraindication for administration of conditioning medication
    -Administration of gammainterferon within 30 days before the infusion of transduced autologous CD34+ cells
    -Participation in another experimental therapeutic protocol within 6 months prior to baseline and during the study period
    -Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study
    -Patient/Parent/Guardian unable or unwilling to comply with the protocol requirements
    E.5 End points
    E.5.1Primary end point(s)
    -Safety of the procedure as measured by the incidence of adverse events
    -Restoration and stability over time of the NADPH functioning granulocytes assessed by a DHR test (≥ 5% of expressing cells at 12 months)
    E.5.1.1Timepoint(s) of evaluation of this end point
    - The assessment of the safety of G1XCGD by the measurement of the safety parameters, e.g. adverse events. This will include clinical adverse events, as well as any clinically significant laboratory abnormality which will have to be reported as an AE. Overall incidence of adverse events will be evaluated for the study as a whole. Similarly the incidence of serious adverse events will be monitored.
    - The determination of the efficacy of G1XCGD. It will be evaluated by the measurementof the percentage of expression in granulocytes at month 12 by DHR test.
    E.5.2Secondary end point(s)
    As a secondary efficacy endpoint, the evolution of the nutritional status, the growth and the development will be evaluated by the regular clinical examination during the study visit.
    Similarly, the evolution of the severe infection and inflammation will be evaluated by the clinical exam but also by the imaging and the laboratory results.
    For the immmunological reconstitution, the restoration of the neutrophil functionality and immunity will be measured by the NADPH oxidase activity (NBC and DHR) and the percentage of transduced CD34+ haematopoietic cells infused and of blood cells at month 1, 2, 3, 6, 9, 12, 18 and 24).
    E.5.2.1Timepoint(s) of evaluation of this end point
    As a secondary efficacy endpoint, the evolution of the nutritional status, the growth and the development will be evaluated by the regular clinical examination during the study visit.
    Similarly, the evolution of the severe infection and inflammation will be evaluated by the clinical exam but also by the imaging and the laboratory results.
    For the immmunological reconstitution, the restoration of the neutrophil functionality and immunity will be measured by the NADPH oxidase activity (NBC and DHR) and the percentage of transduced CD34+ haematopoietic cells infused and of blood cells at month 1, 2, 3, 6, 9, 12, 18 and 24 to be validated with the Investigators).
    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 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 No
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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
    France
    Germany
    Switzerland
    United Kingdom
    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
    Last visit, last subject
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months9
    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 Yes
    F.1.1Number of subjects for this age range: 15
    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) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 5
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    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)
    The IMP tested in this clinical trial is intended to be a single dose treatment, due to the expected persistence of its therapeutic effect. Nevertheless, patients will continue to receive other appropriate treatments as judge necessary by their physician. In addition, due to the nature of the IMP i.e. Gene Therapy Medicinal Product, an at least 10 years follow-up is planned after the last study visit, in order to monitor the long term safety and efficacy of the 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 Decision2013-11-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-12-23
    P. End of Trial
    P.End of Trial StatusOngoing
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