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    The EU Clinical Trials Register currently displays   44154   clinical trials with a EudraCT protocol, of which   7326   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2006-004661-34
    Sponsor's Protocol Code Number:2006.426/19
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2006-10-18
    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 ConcernedFrance - ANSM
    A.2EudraCT number2006-004661-34
    A.3Full title of the trial
    Evaluation d’un traitement par miglustat (Zavesca®) chez les patients atteints de mucopolysaccharidose de type III (maladie de Sanfilippo).
    Essai thérapeutique de phase IIb randomisé en aveugle contre placebo.
    A.3.2Name or abbreviated title of the trial where available
    ZAV-MPSIII 2006
    A.4.1Sponsor's protocol code number2006.426/19
    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 SponsorHOSPICES CIVILS DE LYON
    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 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 Yes
    D.2.1.1.1Trade name ZAVESCA
    D.2.1.1.2Name of the Marketing Authorisation holderACTELION
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 nameZAVESCA
    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.3.9.3Other descriptive nameMIGLUSTAT
    D.3.10 Strength
    D.3.10.1Concentration unit mg/g milligram(s)/gram
    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 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 No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Mucopolysaccharidose de type III
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 8.1
    E.1.2Level LLT
    E.1.2Classification code 10056890
    E.1.2Term Mucopolysaccharidosis III
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    L’objectif principal d’efficacité est l’évaluation de la capacité du miglustat (Zavesca®), par rapport à un placebo, à montrer une amélioration ou une stabilisation à 6 mois des troubles du comportement adaptatif et de socialisation chez des enfants atteints de mucopolysaccharidose de type III (maladie de Sanfilippo).
    E.2.2Secondary objectives of the trial
    - l’efficacité à 12 mois du miglustat sur les troubles du comportement adaptatif en utilisant le test de Vineland qui explore 4 domaines : communication, socialisation, vie quotidienne et motricité globale et fine.
    - la capacité du miglustat, par rapport à un placebo, à montrer une amélioration ou une stabilisation des troubles de sommeil et des troubles neuro-cognitifs (quotient de développement, hyperactivité) chez des enfants atteints de mucopolysaccharidose de type III, MPSIII (maladie de Sanfilippo)
    - la capacité du miglustat, par rapport à un placebo, à passer la barrière hémato-méningée et à montrer une diminution du taux de Gangliosides GM2 dans le liquide céphalo-rachidien.
    - la tolérance et les effets indésirables du miglustat par rapport à un placebo pendant toute la durée de l’étude chez des enfants atteints de mucopolysaccharidose de type III
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Les parents des patients ou le tuteur légal du patient doivent signer un formulaire de consentement éclairé avant toutes procédures en relation avec le protocole. Les enfants pouvant comprendre le déroulement de l’étude seront informés oralement et leur accord de participation à l’étude sera sollicité.
    -Affiliation à un régime de sécurité sociale
    - Les patients devront être âgés de 2 à 12 ans
    - Les patients doivent avoir un diagnostic de MPSIII A, B, C ou D
    - Les patients ne devront pas avoir perdu la capacité à marcher sans aide
    - Les patients et les parents devront accepter les contraintes liées à l’étude
    E.4Principal exclusion criteria
    - Les patients ou les parents refusant de participer aux évaluations demandées par le protocole avant le début du traitement
    - Les patients ayant reçu un médicament à l’essai moins de 30 jours avant l’inclusion dans l’étude
    - Les patients souffrant d’un problème médical, d’une maladie grave intercurrente ou de toute autre source de fatigue pouvant, de l’avis de l’investigateur, interférer de façon significative avec l’observance à l’étude, y compris toutes les évaluations et les suivis prescrits.
    - Les patients présentant une hypersensibilité connue au miglustat
    - Les patients ayant déjà reçu du miglustat
    - les patients sous Cérézyme®
    E.5 End points
    E.5.1Primary end point(s)
    - Stabilisation ou une amélioration à 6 mois du comportement adaptatif mesuré par le score obtenu au test de Vineland Survey form.

    - Stabilisation : le score obtenu lors du test de Vineland « Survey form » est identique entre le début de la Recherche (avant le démarrage du traitement) et 6 mois après la prise du traitement pour au moins 2 domaines parmi les 4 domaines évalués : communication, vie quotidienne, socialisation, motricité globale et fine.

    - Amélioration : le score obtenu lors du test de Vineland « Survey form » est augmenté d’au moins 1 point entre le début de la recherche (avant le démarrage du traitement à l’étude) et à 6 mois pour au moins 2 domaines (communication, vie quotidienne, socialisation, motricité globale et fine).
    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 Yes
    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) No
    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) No
    E.8.2.2Placebo Yes
    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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    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
    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 months1
    E.8.9.1In the Member State concerned days0
    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) No
    F.1.3Elderly (>=65 years) No
    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 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 state24
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 24
    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 Decision2006-12-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2006-11-22
    P. End of Trial
    P.End of Trial StatusOngoing
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