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    Summary
    EudraCT Number:2007-005543-22
    Sponsor's Protocol Code Number:TKT028
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2008-01-02
    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 ConcernedCzechia - SUKL
    A.2EudraCT number2007-005543-22
    A.3Full title of the trial
    A Multi-center, Open-Label, Randomized Study Evaluating the Safety and
    Efficacy of Three Dosing Regimens of Replagal Enzyme Replacement
    Therapy in Adult Patients with Fabry Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An investigation of 3 dosages of Replagal in adult patients with Fabry disease
    A.4.1Sponsor's protocol code numberTKT028
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorShire Human Genetic Therapies, Inc. (Shire HGT)
    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 supportShire Human Genetics Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire Human Genetic Therapies, Inc.
    B.5.2Functional name of contact pointClinical Project Manager
    B.5.3 Address:
    B.5.3.1Street Address300 Shire Way
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number+17814829622
    B.5.5Fax number+17814822954
    B.5.6E-mailawijatyk@shire.com
    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 Replagal
    D.2.1.1.2Name of the Marketing Authorisation holderShire Human Genetic Therapies AB
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/00/002
    D.3 Description of the IMP
    D.3.1Product nameReplagal
    D.3.4Pharmaceutical form Solution for infusion
    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.3.8INN - Proposed INNagalsidase alpha
    D.3.9.3Other descriptive nameReplagal
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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) No
    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 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 Yes
    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 typeHuman protein alpha-galactosidase A produced in a human cell line by genetic engineering technology
    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
    Fabry Disease
    E.1.1.1Medical condition in easily understood language
    deficiency in enzyme alpha galactosidase A
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10016016
    E.1.2Term Fabry's 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
    To compare the effects of 2 dosing regimens of Replagal (0.2 mg/kg
    administered intravenously (IV) every other week and 0.2 mg/kg IV every
    week) on the reduction from baseline in left ventricular mass (LVM) as
    measured by echocardiography
    E.2.2Secondary objectives of the trial
    To compare the effects of 2 dosing regimens of Replagal (0.2 mg/kg IV every
    other week and 0.2 mg/kg IV every week)
    - on exercise tolerance by maximal
    oxygen consumption (Vo2max) using the standard exponential exercise
    protocol (STEEP) and by the 6-Minute Walk Test (6MWT)
    - on the improvement from baseline
    in disease-specific quality of life (QoL) using the summary score of the
    Minnesota Living with Heart Failure questionnaire (MLHF-Q)
    - on the improvement from baseline
    in New York Heart Association (NYHA) functional class for heart failure
    symptoms
    - as assessed by adverse events
    (AEs), concomitant medications, vital signs, physical examinations,
    electrocardiograms (ECGs), anti-agalsidase alfa antibodies and standard
    clinical and laboratory (hematology, serum chemistry, and urinalysis)
    measurements
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. The patient is ≥ 18 years-old.
    2a. The patient is a hemizygous male with Fabry disease as confirmed by a deficiency of
    α−galactosidase-A activity measured in serum, leukocytes, or fibroblasts. Male
    patients who do not already have a documented deficiency of α−galactosidase-A
    activity will provide a blood sample during Screening for determination of
    α−galactosidase-A activity level in their serum.
    OR
    2b. The patient is a heterozygous female with Fabry disease as confirmed by a mutation
    of the α−agalactosidase A gene. Female patients who do not already have a
    documented mutation of the α−galactosidase A gene will provide a blood sample
    during Screening for genotyping.
    3. The patient has not received enzyme replacement therapy (ERT) for Fabry disease
    (i.e., the patient is ERT-naïve).
    4. The patient’s LVM/h is > 50 g/m2.7 for male patients and > 47 g/m2.7 for female
    patients, as measured by echocardiography, which is the upper limit of normal.
    5. 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 a negative
    pregnancy test at the time of enrollment and as required throughout their participation
    in the study.
    6. The patient has provided written informed consent that has been approved by the
    Institutional Review Board/Independent Ethics Committee (IRB/IEC).
    E.4Principal exclusion criteria
    1. The patient has documented NYHA functional class IV heart failure symptoms.
    2. The patient has clinically significant systemic hypertension defined as an untreated
    resting blood pressure (BP) > 160 / 110 mmHg or poorly controlled hypertension
    defined as a BP > 150 / 100, while receiving medication(s) for treatment of
    hypertension.
    3. The patient has hemodynamically significant valvular stenosis or regurgitation by
    Doppler echocardiography.
    4. The patient is morbidly obese, defined as having a body mass index > 39 kg/m2.
    5. The patient has a known autosomal dominant sarcoplasmic contractile protein gene
    mutation.
    6. The patient has received treatment with any investigational drug or device within the
    30 days prior to study entry.
    7. 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.
    8. The patient has a positive test for hepatitis B surface antigen, hepatitis C antibody, or
    human immunodeficiency virus (HIV) antibody.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of this study will be the change from Baseline to Week 53 in LVM normalized to height (LVM/h), as measured by echocardiography.
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 53
    E.5.2Secondary end point(s)
    1.Change from Baseline to Week 53 in exercise tolerance, as measured
    by Vo2max at peak exercise using the STEEP exercise protocol and by
    distance walked in meters (m) using the 6MWT
    2. Change from Baseline to Week 53 in NYHA Class
    3.Change from Baseline to Week 53 in QoL using the MLHF-Q summary score
    4. Safety: AEs, concomitant medications, vital signs, physical examinations,
    ECGs, anti-agalsidase alfa antibodies and standard clinical assessments
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 53
    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 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) 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) Yes
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    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 Yes
    E.8.2.3.1Comparator description
    Alternative doses of IMP (PR1)
    E.8.2.4Number of treatment arms in the trial3
    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 EEA5
    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
    Australia
    Paraguay
    United States
    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 last 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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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 No
    F.1.1Number of subjects for this age range: 0
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 39
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 43
    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)
    Subjects who had successfully completed 12 months of treatment under study protocol TKT028 would be offered the opportunity to enroll in a 12 month protocol extension study.
    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 Decision2008-02-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2008-06-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2012-07-05
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