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    Summary
    EudraCT Number:2006-006304-11
    Sponsor's Protocol Code Number:TKT034
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2010-03-04
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2006-006304-11
    A.3Full title of the trial
    A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.

    Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa
    A.3.2Name or abbreviated title of the trial where available
    TKT034
    A.4.1Sponsor's protocol code numberTKT034
    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 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.3.9.2Current sponsor codeGA-GCB
    D.3.9.3Other descriptive nameGene activated human glucocerebrosidase
    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 typeGene activated protein derived from human cell line by insertion of regulatory structural DNA sequences into the glucocerebrosidase locus.
    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
    Type I Gaucher Disease

    Enfermedad de Gaucher de Tipo I
    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
    To evaluate the safety of every other week dosing of GA-GCB in patients with type 1 Gaucher disease who were previously treated with imiglucerase.
    E.2.2Secondary objectives of the trial
    To evaluate changes from Baseline in hemoglobin concentration after every other
    week dosing of GA-GCB.

    To evaluate changes from Baseline in platelet count after every other week dosing of GA-GCB.

    To evaluate changes from Baseline in liver and spleen volumes by abdominal
    magnetic resonance imaging (MRI) after every other week dosing of GA-GCB.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    The patient has a documented diagnosis of type 1 Gaucher disease, as documented by deficient glucocerebrosidase (GCB) activity in leukocytes or by genotype analysis.

    The patient has received imiglucerase at a dose ≤ 60 U/kg and ≥ 15 U/kg every other
    week for a minimum of 30 consecutive months. Patients must have received the
    same dose and dose regimen during the 6 months prior to study enrollment. (Note:
    Patients who are anti-imiglucerase antibody positive will be allowed to enter this
    study).

    The patient is at least 2 years of age.

    Female patients of child bearing potential must agree to use a medically acceptable
    method of contraception at all times during the study and must have negative results
    to a pregnancy test performed at Screening and as required throughout their
    participation in the study.

    The patient, the patient’s parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).

    The patient must be sufficiently cooperative to participate in this clinical study as
    judged by the Investigator.
    E.4Principal exclusion criteria
    The patient has a hemoglobin concentration ≤ 10 g/dL and a platelet count ≤ 80,000 platelets/cu mm.

    The difference between the patient’s highest and lowest hemoglobin concentration
    collected over 3 consecutive evaluations is > 1 g/dL, AND/OR, the difference
    between the patient’s highest and lowest platelet count collected over 3 onsecutive evaluations is > 20%. (Note: At least 1 of these values must have been collected at
    least 6 months prior to the Screening evaluation and 1 value must be collected during Screening).

    The patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease.

    The patient has received treatment with any investigational drug or device within the
    30 days prior to study entry; such use during the study is not permitted.

    The patient is known to be positive for human immunodeficiency virus (HIV), i.e.,
    has a documented positive result. Patients who do not have a documented positive
    result will be tested for HIV during Screening.

    The patient is known to be positive for hepatitis B and/or C, i.e., has a documented
    positive result. Patients who do not have a documented positive result will be tested
    for hepatitis during Screening.

    The patient presents with sustained iron, folic acid and/or vitamin B12 deficiencyrelated anemia during Screening (i.e., non-Gaucher disease-related anemia).

    The patient, patient’s parent(s), or patient’s legal guardian(s) is/are unable to
    understand the nature, scope, and possible consequences of the study.

    The patient has a significant comorbidity that might affect study data or confound the
    study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver disease,
    etc.).

    The patient is unable to comply with the protocol, e.g., has a clinically relevant
    medical condition making implementation of the protocol difficult, has an
    uncooperative attitude, is unable to return for safety evaluations, or is otherwise
    unlikely to complete the study, as determined by the Investigator.

    The patient has had inconsistent treatment with imiglucerase in the last 6 months such as a drug holiday.

    The patient has experienced an anaphylactic reaction during treatment with
    imiglucerase.

    The patient has received miglustat during the 6 months prior to study enrollment.

    The patient is currently receiving erythropoietin or chronic systemic corticosteroids in
    the last 6 months. (NOTE: Use of intermittent corticosteroids as premedication to
    prevent infusion reactions is allowed.)

    The patient has experienced spleen infarction.

    The patient has bone necrosis.
    E.5 End points
    E.5.1Primary end point(s)
    To evaluate the safety of transitioning patients previously receiving enzyme replacement therapy with imiglucerase to GA-GCB therapy administered as the same number of units as their imiglucerase therapy. Safety assessments include adverse events (including infusion-related adverse events), concomitant medication use, vital signs, 12-lead ECG, physical examinations, and clinical laboratory tests (hematology, serum chemistry, urinalysis, and anti-GA-GCB antibody testing).
    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) 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) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 EEA8
    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
    Last patient visit.
    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 months8
    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 months10
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children over 2 years of age
    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 27
    F.4.2.2In the whole clinical trial 40
    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)
    Patients will be offered participation in an open label extension study (protocol not yet finalised).
    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 Decision2007-08-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2007-06-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2009-06-26
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