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    Summary
    EudraCT Number:2007-002840-21
    Sponsor's Protocol Code Number:HGT-GCB-039
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2008-04-15
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2007-002840-21
    A.3Full title of the trial
    A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease
    A.3.2Name or abbreviated title of the trial where available
    GCB039
    A.4.1Sponsor's protocol code numberHGT-GCB-039
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberND
    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 SponsorSHIRE HUMAN GENETIC THERAPIES, 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 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 nameGene Activated Human Glucocerebrosidase
    D.3.2Product code GA-GCB
    D.3.4Pharmaceutical form Powder 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.3.9.2Current sponsor codeGA-GCB
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 typeNon Applicabile
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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/i/97/053/001
    D.3 Description of the IMP
    D.3.1Product nameimiglucerasi
    D.3.4Pharmaceutical form Powder 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 INNImiglucerase
    D.3.9.1CAS number 154248972
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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 typeNon Applicabile
    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
    Patients with Gaucher disease of type 1
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10018048
    E.1.2Term Gaucher's disease
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to compare the effects of GA-GCB and imiglucerase on the hemoglobin concentration in patients with Type 1 Gaucher disease.
    E.2.2Secondary objectives of the trial
    To compare the effects of GA-GCB and imiglucerase on the platelet count.To compare the effects of GA-GCB and imiglucerase on liver and spleen volumes (using magnetic resonance.To compare the effects of GA-GCB and imiglucerase on biological markers specific to Gaucher disease (plasma chitotriosidase level and chemokine (C-C motif) ligand 18 (CCL18levels.To evaluate the safety of GA-GCB and imiglucerase in patients with Type 1 Gaucher disease, on the basis of evaluations of standard clinical laboratory investigations (including rates of antibody formation and enzyme-neutralizing antibody activity) and of safety evaluations (including the incidence of infusion-related adverse events and the proportion of patients who needed pre-medication to keep infusion-related adverse events under control) for each treatment group. To compare the effects of GA-GCB and imiglucerase on the minimum time to response for hemoglobin (defined as an improvement of > or = 1 g/dl in hemoglobin levels
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patient with a documented diagnosis of Type 1 Gaucher disease, established on the basis of a deficit in glucocerebrosidase (GCB) activity, in relation to the norm, evaluated at leukocyte level, or with genotype analysis. 2. Patient no less than 2 years old. 3a. Patient with anemia related to Gaucher disease, defined as a hemoglobin concentration at least 0.5 g/dL below the lower limit of the normal value for age and sex (on the basis of results obtained during screening and on admission). AND ONE OR MORE OF THE FOLLOWING THREE CRITERIA: 3b. Patient with at least moderate splenomegaly (2 to 3 cm below the left costal margin) on palpation. (In order to be suitable for the study, patients who have undergone splenectomy must satisfy inclusion criterion 3c or inclusion criterion 3d.). OR 3c. Patient with thrombocytopenia related to Gaucher disease (defined as a platelet count of < or= 120 x 103/mm3). OR 3d. Patient with readily-palpable liver enlargement related to Gaucher disease. 4. Patient not treated for Gaucher disease (with investigational products, miglustat or imiglucerase) during the past 12 months prior to admission to the study, as documented in the history. 5. Patients of the female sex of child-bearing age must agree to use a form of contraception at all times during the study that is acceptable from a medical point of view and pregnancy tests carried out at the time of enrollment and at the required intervals during their participation in the study must be negative. 6. Patient who has, personally or through his/her parents or legal guardians, supplied written informed consent approved by the Institutional Review Board (IRB) or by the Independent Ethics Committee (IEC). 7. Patient sufficiently able to cooperate in participation in the clinical study, in the opinion of the investigator.
    E.4Principal exclusion criteria
    Patient with Type 2 or 3 Gaucher disease or with suspected Type 3 Gaucher disease. 2. Patient positive for antibodies to imiglucerase or GA-GCB during screening or having previously exhibited an anaphylactic reaction to imiglucerase or to GA-GCB or having needed pre-medication to keep reactions to imiglucerase or GA-GCB under control. 3. Patient treated with an investigational drug or device not related to Gaucher disease during the 30 days prior to admission to the study; such treatment is not permitted during the study. 4. Patient currently receiving erythrocyte growth factor or chronic systemic corticosteroids, or who has been treated with such drugs within the last 6 months. 5. Patient known to be positive for acquired immunodeficiency virus (HIV), i.e. with a documented positive result. Patients without a documented positive result will be tested for HIV during screening. 6. Patient known to have tested positive for hepatitis B and/or C, i.e. with a documented positive result. Patients without a documented positive result will be tested for hepatitis during screening. 7. Patient presents with exacerbation of anemia during screening (e.g. due to folic acid and/or vitamin B12 deficiency). 8. Patient with serum transferrin saturation < 20 microg/dL and with < 50 microg/dL serum ferritin. 9. Patient (or his/her parents or legal guardians) not in a position to understand the nature, scope and possible consequences of the study. 10. Patient with one or more significant co-morbidities that might affect study data or confound study results (e.g. malignant neoplasias, primary biliary cirrhosis, autoimmune liver disease etc.). 11. Patient not in a position to adhere to the protocol, for example, in health conditions that make implementation of the protocol difficult, with an uncooperative attitude, not in a position to attend for the safety evaluations or for whom completion of the study is otherwise unlikely, in the opinion of the investigator.
    E.5 End points
    E.5.1Primary end point(s)
    The principal end-point of this study is to compare the mean change, in relation to the baseline, in the hemoglobin concentration between the two treatment groups.
    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 No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 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 Yes
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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 years1
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months2
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) Yes
    F.1.1.6Adolescents (12-17 years) Yes
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2008-04-15. Yes
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 9
    F.4.2.2In the whole clinical trial 32
    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)
    Ai pazienti sara' offerto di partecipare ad uno studio di estensione in aperto (protocollo non ancora finalizzato)
    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 Decision2008-02-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2008-02-25
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2009-07-28
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