Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    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).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2017-001481-23
    Sponsor's Protocol Code Number:P160927J
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-10-30
    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 number2017-001481-23
    A.3Full title of the trial
    MULTICENTER, RANDOMIZED, DOUBLE BLIND, PLACEBO CONTROLLED CLINICAL TRIAL WITH RILUZOLE IN SPINOCEREBELLAR ATAXIA TYPE 2
    Etude multicentrique, randomisée en double aveugle, contrôlée versus placebo évaluant l'efficacité du RILuzole dans l' ATaxie spinocerebelleuse de type 2 SCA2.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    NA
    NA
    A.3.2Name or abbreviated title of the trial where available
    ATRIL
    A.4.1Sponsor's protocol code numberP160927J
    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 SponsorASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    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 supportMinistère des Solidarités et de la Santé
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    B.5.2Functional name of contact pointDRCI Hôpital St Louis
    B.5.3 Address:
    B.5.3.1Street Address 1 av. Claude Vellefaux
    B.5.3.2Town/ cityPARIS
    B.5.3.3Post code75010
    B.5.3.4CountryFrance
    B.5.6E-mailyannick.vacher@aphp.fr
    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 Riluzole PMCS
    D.2.1.1.2Name of the Marketing Authorisation holderPRO.MED.CS PRAHA A.S.
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameRiluzole PMCS
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    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 INNRiluzole
    D.3.9.3Other descriptive nameRiluzole
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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) 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 No
    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 placeboTablet
    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
    SCA2 patients, both gender, at least 18 years of age
    Patients SCA2, homme et femme, âgé d'au moins 18 ans
    E.1.1.1Medical condition in easily understood language
    NA
    Ataxie
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10057660
    E.1.2Term Spinocerebellar ataxia
    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
    Measuring the efficacy of treatment with riluzole in SCA2 patients during 12 months.
    Mesurer l'efficacité du traitement par riluzole chez des patients SCA2 durant 12 mois
    E.2.2Secondary objectives of the trial
    - To evaluate the quantitative evolution of cerebellar symptoms by showing decrease in the SARA score
    - Quantitative evolution of the SARA score by showing decrease in the SARA score as compared to their SARA score's natural evolution
    - Quantitative evolution of the CCFS score by showing decrease in the CCFS score
    - Evolution of extracerebellar symptoms by showing decrease in the INAS count
    - Evolution of motoneuronal symptoms
    - Volumetric 3T MRI evolutions of brainstem and cerebellar atrophy by showing decrease or stabilization in the rate of cerebellar and brainstem atrophy
    - To assess the effect on quality of life
    - To confirm long-term clinical and biological tolerance of riluzole in SCA2 patients by clinical exam and by blood analysis
    - To compare disease evolution for patients with and without motoneuronal symptoms
    - To compare survival of the patients between the two treatment groups
    - To look for factors influencing treatment efficacy for the previous criteria
    - Évaluer l'évolution quantitative des symptômes cérébelleux en montrant une diminution du score SARA
    - Evolution quantitative du score SARA en montrant une diminution du score SARA par rapport à l'évolution naturelle du score SARA
    - Evolution quantitative du score CCFS en montrant une diminution du score CCFS
    - Evolution des symptômes extracérébelleux (INAS) en montrant une diminution du taux d'INAS
    - Evolution des signes motoneuronaux (fasciculations, amyotrophie, faiblesse des membres)
    - Evolution volumétrique de l'IRM 3T du tronc cérébral et du cervelet en montrant une diminution ou une stabilisation du taux d'atrophie cérébelleuse et du tronc cérébral, mesurée par VBM et QSM,
    - Évaluer l'effet sur la qualité de vie (SF-36)
    - Confirmer la tolérance clinique et biologique à long terme du riluzole chez les patients SCA2 par examen clinique lors des visites de l'étude et par analyse de sang tous les 3 mois dans un laboratoire proche de leur domicile
    - Comparer l'évolution
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Genetically diagnosed SCA2 (CAG triplet in ATXN2 > or = 33)
    - At least 18 years of age
    - Signature of informed consent
    - Covered by social security
    - SARA score > or = 5 and < or = 26
    - Age at onset < or = 50 years old
    - SCA2 diagnostiqué génétiquement (triplet CAG dans ATXN2 > ou = 33)
    - Age égal ou supérieur à 18 ans
    - Signature du consentement éclairé
    - Affilié à la sécurité sociale
    - score SARA > ou = 5 et < ou = 26
    - Age de début < ou = 50 ans
    E.4Principal exclusion criteria
    - Treated with riluzole prior to the study
    - Hepatotoxic medication
    - Hypersensitivity to the active substance or to any of the excipients (Dibasic calcium phosphate anhydrous, Micro crystalline cellulose, Croscarmellose sodium, Colloidal silica anhydrous, Magnesium stearate, Hypromellose, Titanium dioxide (E171) Macrogol 400)
    - Hypersensitivity to any of ingredients of placebo (Lactose monohydrate, Micro crystalline cellulose, Colloidal silica, anhydrous, Magnesium stearate, Opadry II HP85F18422 White)
    - Serious systemic illnesses or conditions known for enhancing the side-effects of riluzole (i.e., severe cardiac or renal insufficiency (grade 3), hematological (grade 3) and hepatic diseases with serum values of alanine amino transferase levels higher than or equal to twice the normal higher limit or abnormal values of several other hepatic markers (gamma-GT, bilirubine, aspartate aminotransferase levels higher than twice the normal higher limit ) (grade 1),
    - Contraindications for MRI examination (metallic implant, pacemaker, artificial heart valve, brain vascular malformation, aneurysm clips, exposed by metallic fragments, artificial implants, neuronal stimulator, insulin pump, intravenous catheter, epilepsy, can be subject to form of seizures, metallic contraceptive device, ...)
    - Participation in another therapeutic trial (3 months exclusion period)
    - Pregnancy or breastfeeding
    - Non abstinence or absence of effective contraception for women
    - Inability to understand information about the protocol
    - Persons deprived of their liberty by judicial or administrative decision
    - Adult subject under legal protection or unable to consent
    - Other ataxic syndromes than SCA2
    - Traité avec riluzole avant l'étude
    - Prise de médicament hépatotoxique
    - Hypersensibilité à la substance active ou à l'un des excipients (Hydrogénophosphate de calcium anhydre, cellulose microcristalline, croscarmellose sodique, silice colloïdale anhydre, stéarate de magnésium, Hypromellose, dioxyde de titane (E171), macrogol 400)
    - Hypersensibilitéà un des composants du placebo (Lactose monohydraté, Cellulose microcristalline, Silice colloïdale anhydre, Stéarate de magnésium, Opadry II HP85F18422 Blanc)
    - Maladies systémiques graves ou conditions connues pour accentuer les effets secondaires du riluzole (par ex : une insuffisance cardiaque ou rénale grave (grade 3), des maladies hématologiques (grade 3) et hépatiques avec des valeurs sériques de l'alanine amino transferase supérieures ou égales au double de la limite supérieure normale ou des valeurs anormales de plusieurs autres marqueurs hépatiques (gamma-GT, bilirubine, aspartate aminotransférase supérieurs à deux fois la limite supérieure normale) (grade 1),
    - Contre-indications pour l'examen IRM (implant métallique, stimulateur cardiaque, valve cardiaque artificielle, malformation vasculaire cérébrale, clips sur un anévrisme, exposé à des fragments métalliques, implants artificiels, stimulateur neuronal, pompe à insuline, cathéter intraveineux, épilepsie, pouvant être soumis à une forme de crise convulsive, dispositif contraceptif non compatible, ...)
    - Participation à un autre essai thérapeutique (période d'exclusion de 3 mois)
    - Grossesse ou allaitement
    - Non-abstinence ou absence de contraception efficace pour les femmes
    - Incapacité à comprendre les informations données à propos de l'étude
    - Personnes privées de liberté par décision judiciaire ou administrative
    - Personne adulte sous protection juridique ou incapable de donner son consentement
    - Autres syndromes ataxiques que SCA2
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of patients with SARA score improved by at least one point at 12 months
    Proportion de patients avec un score SARA amélioré d'au moins un point au bout de 12 mois
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 months
    12 mois
    E.5.2Secondary end point(s)
    a. Decrease in the SARA score during one year
    b. Decrease in the SARA score as compared to the natural evolution. SARA scores will be measured at M0, M6 and M12. Pre-trial scores history within the previous years will be recorded when available.
    c. Decrease in the CCFS score during one year of treatment. CCFS scores will be measured at M0, M6 and M12. Pre-trial scores history within the two previous years will be recorded when available.
    d. Change in the INAS score during one year of treatment. INAS score will be measured at M0, M6 and M12. A specific focus on the INAS items fasciculations, amyotrophy and paresis:
    - Fasciculation: at least one of the 3 body part with presence of fasciculations
    - Amyotrophy: Upper limb distal and/or Tongue
    - Paresis: Upper limb proximal and/or distal
    e Decrease or stabilization of the volume of cerebellar and brainstem during one year of treatment with riluzole in SCA2 patients using volumetric 3T MRI at M0 and M12.
    f. The benefit of riluzole use on patient quality of life will be evaluated using a standardized quality of life questionnaire (SF-36) at M0 and M12.
    g. Long-term tolerance will be confirmed by clinical examination at study visits and by blood analysis (hepatic measurements, blood cell count) every month during the first 3 months of treatment, then every 3 months, at their home laboratory.
    h. Survival will be analyzed depending of the number of death observed during the study.
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months
    12 mois
    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 No
    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) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned9
    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 No
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    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 No
    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: 40
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 state50
    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)
    At the end of his participation, the patient will resume his usual medical follow-up.
    Treatment at the end of the patient's participation will be left to the discretion of the investigator.
    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 Decision2017-08-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-09-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-06-19
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA