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    Summary
    EudraCT Number:2005-001651-37
    Sponsor's Protocol Code Number:OGT 918-011
    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:2006-01-25
    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 number2005-001651-37
    A.3Full title of the trial
    A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease
    A.4.1Sponsor's protocol code numberOGT 918-011
    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 SponsorActelion Pharmaceuticals Ltd
    B.1.3.4CountrySwitzerland
    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 Information not present in EudraCT
    D.2.1.1.1Trade name Zavesca
    D.2.1.1.2Name of the Marketing Authorisation holderActelion Registration Ltd
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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/3/00/006
    D.3 Description of the IMP
    D.3.1Product nameMiglustat
    D.3.2Product code OGT918
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNmiglustat
    D.3.9.1CAS number 72599-27-0
    D.3.9.2Current sponsor codeOGT918
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 Yes
    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) 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 Information not present in EudraCT
    D.3.11.8Extractive medicinal product Information not present in EudraCT
    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 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
    Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol:

    1 BACKGROUND AND RATIONALE

    1.1 Disease
    MedDRA Classification
    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 long-term (i.e. over a 24 month period) effect on liver volume of miglustat (100mg TID) as maintenance therapy in adult patients with stable mild to moderate Type 1 Gaucher disease after a switch from Enzyme Replacement Therapy (ERT)
    E.2.2Secondary objectives of the trial
    To evaluate the long-term (i.e. over a 24 month period) effects of miglustat (100mg TID) on spleen volume and other markers of Type 1 Gaucher disease

    To evaluate the long-term safety and tolerability with particular reference to any changes in the neurological assessments (including cognitive function)
    E.2.3Trial contains a sub-study Information not present in EudraCT
    E.3Principal inclusion criteria
    Eligible patients must meet all of the following inclusion criteria:

    1. Patients aged 18 years or older at the study entry with a confirmed diagnosis of type 1 mild to moderate Gaucher disease at the time of enrollment. Diagnosis will be confirmed on glucocerebrosidase assay or molecular analysis of the glucocerebrosidase gene.

    2. Patients treated with ERT for at least 3 years and who are receiving their current dose for a minimum of last six months.

    3. Clinically and biologically stable for the previous 2 years, i.e. (with at least 3 timepoint assessments available for each parameters)
    a. Stable organomegaly, defined by <= 10% variation.
    b. Free of progressive symptomatic bone disease (no proven bone disease progression for the last 2 years)
    c. Controlled hemoglobin level above 11 g/dl ,
    d. Controlled platelets counts with a mean of at least 100x10 Power 9 /l
    e. Stable chitotriosidase activity, defined by <= 20% variation.

    4. Patients who provide written informed consent to participate in the study
    E.4Principal exclusion criteria
    Eligible patients must meet none of the following exclusion criteria:
    1. Patients (males and females) who do not agree to use reliable contraception throughout the study and for three months after cessation of miglustat treatment.
    2. Female patients who are pregnant or breast feeding, or will not undergo a pregnancy test prior to enrollment into the study.
    3. Patients with a history of significant lactose intolerance.
    4. Patients with a confirmed neuropathy.
    5. Patients suffering from clinically significant diarrhea (>3 liquid stools per day for >7 days) without definable cause within 6 months of Visit 1, or who have a history of significant gastrointestinal disorders (e.g., Irritable Bowel Syndrome).
    6. Patients with history of cataracts or known increased risk of cataract formation.
    7. Patients with severe renal impairment i.e., with a creatinine clearance <30 ml/min/1.73mPower2
    8. Patients who are not ambulant, or have severe bone disease according to the bone history and symptom list (see appendix 9)
    9. Patients splenectomised before 18 years old, because of massive splenomegaly or severe cytopenia.
    10. Patients with an intercurrent medical condition that would render them unsuitable for study.
    11. Patients currently undergoing therapy with other investigational product, or who have received an investigational product within 3 months prior to study start.
    12. Patients with an intercurrent active medical condition such as HIV or Hepatitis B/C that would render them unsuitable for study.
    13. Patients who for whatever reason are, in the opinion of the Investigator, thought to be unsuitable for the study.
    14. Patients who have previously received treatment with miglustat.
    15. Patients with a history of evidence of oculomotor gaze palsies, ataxia or other manifestations typically associated with type 3 (“neuronopatic”) Gaucher disease.
    16. Predicted hypersensitivity to miglustat or any excipients.
    17. Patients with current alcohol or drug abuse or dependence.
    E.5 End points
    E.5.1Primary end point(s)
    Percent change from baseline to Month 24 in liver volume

    For the primary endpoint, the clinically acceptable difference is a mean percentage increase from Baseline to Month 24 of no greater than 10% in liver volume.
    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 Information not present in EudraCT
    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 Information not present in EudraCT
    E.7.1.2Bioequivalence study Information not present in EudraCT
    E.7.1.3Other Information not present in EudraCT
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    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.8.6 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 Information not present in EudraCT
    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
    per protocol
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months30
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months30
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    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 Information not present in EudraCT
    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 2006-01-25. 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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 50
    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)
    Zavesca is a maketed drug, and if the treament was benefical to the patient while participating in the study, the physicien may prescribe the drug to the patient after study completion
    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 Decision2006-04-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2005-12-30
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2010-06-22
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