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    Summary
    EudraCT Number:2017-002147-15
    Sponsor's Protocol Code Number:200200
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-09-10
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2017-002147-15
    A.3Full title of the trial
    Intravenous immunoglobulin for Unverricht–Lundborg disease: single-patient trial.
    Immunoglobuline policlonali periodiche nella malattia di Unverricht-Lundborg: trial su singolo paziente.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Intravenous immunoglobulin for a rare genetic disease, belonging to progressive myoclonus epilepsies, called Unverricht–Lundborg disease: single-patient trial.
    Immunoglobuline per via endovenosa in una rara malattia genetica appartenente al gruppo delle epilessie miocloniche progressive denominata malattia di Unverricht–Lundborg: trial su singolo paziente.
    A.3.2Name or abbreviated title of the trial where available
    Immunoglobulin for Unverricht–Lundborg disease.
    Immunoglobuline per la malattia di Unverricht–Lundborg.
    A.4.1Sponsor's protocol code number200200
    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 SponsorAZIENDA SOCIO SANITARIA TERRITORIALE DI MANTOVA
    B.1.3.4CountryItaly
    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 supportASST di Mantova
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationASST di Mantova
    B.5.2Functional name of contact pointNeurologia
    B.5.3 Address:
    B.5.3.1Street AddressStrada Lago Paiolo 10
    B.5.3.2Town/ cityMantova
    B.5.3.3Post code46100
    B.5.3.4CountryItaly
    B.5.4Telephone number0376201686
    B.5.5Fax number0376201969
    B.5.6E-mailalfonso.ciccone@asst-mantova.it
    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 VENITAL - 50 G/L SOLUZIONE PER INFUSIONE FLACONCINO DA 100 ML + SET INFUSIONALE
    D.2.1.1.2Name of the Marketing Authorisation holderKEDRION S.P.A.
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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.3.2Product code J06BA02
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.11 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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    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 No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) Yes
    D.3.11.7Plasma derived medicinal product Yes
    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.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Unverricht-Lundborg disease
    Malattia di Unverricht-Lundborg
    E.1.1.1Medical condition in easily understood language
    Rare inherited form of drug resistant epilepsy classified as a type of progressive myoclonus epilepsy.
    Within 5 to 10 years, the myoclonic episodes may become severe enough to interfere with walking a
    Rara forma di epilessia ereditaria farmacoresistente, classificata fra le epilessie miocloniche progressive. In 5-10 anni le mioclonie sono così gravi da interferire con la deambulazione e le attività
    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 LLT
    E.1.2Classification code 10054858
    E.1.2Term Unverricht's syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10054895
    E.1.2Term Baltic myoclonic epilepsy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10054894
    E.1.2Term Unverricht-Lundborg 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
    Improvement of the action myoclonus
    Miglioramento del mioclono d'azione
    E.2.2Secondary objectives of the trial
    Improvement in the overall score and
    in individual sections of the Unified Myoclonus Rating Scale at one year; patient preferences based on results at the end of the trial.
    Miglioramento al punteggio globale e a singole sezioni della Unified Myoclonus Rating Scale ad un
    anno; preferenze del paziente sulla base dei risultati alla fine del trial.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A person of 23 years of age, suffering from Unverricht-Lundborg disease (clinical and genetic diagnosis).
    Caso di 23 anni di età, affetto da malattia di Unverricht-Lundborg (diagnosi clinica e genetica).
    E.4Principal exclusion criteria
    Contraindications to treatment with intravenous immunoglobulin.
    Controindicazioni alla terapia con immunoglobuline endovena.
    E.5 End points
    E.5.1Primary end point(s)
    Improvement of at least 20% of the action
    myoclonus measured with the Unified Myoclonus Rating Scale at one a year.
    Miglioramento di almeno il 20% del Mioclono d’azione misurato con la Unified Myoclonus Rating
    Scale ad un anno.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Once a month for a year.
    Una volta al mese per un anno.
    E.5.2Secondary end point(s)
    Unified Myoclonus Rating Scale overall score improvement.; Improvement of the score of each of the Unified Myoclonus Rating Scale sections.; Patient preference.
    Miglioramento al punteggio globale della Unified Myoclonus Rating Scale.; Miglioramento del punteggio delle singole sezioni della Unified Myoclonus Rating Scale.; Preferenza del paziente.
    E.5.2.1Timepoint(s) of evaluation of this end point
    One year.; One year.; One year.
    Un anno.; Un anno.; Un anno.
    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) 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 No
    E.8.1.6Cross over Yes
    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 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.8.5.1Number of sites anticipated in the EEA1
    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 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
    LVLS
    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 months0
    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 months0
    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.1.1.1In 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: 1
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 1
    F.4.2.2In the whole clinical trial 1
    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)
    If the outcome is in favor of the experimental treatment, the patient shlould continue the experimental treatment, otherwise he will continue the standard treatment with antiepileptic therapy.
    In caso di esito a favore del trattamento sperimentale il paziente continuerà il trattamento sperimentale altrimenti proseguirà con la sola terapia antiepilettica.
    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 Decision
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-06-23
    P. End of Trial
    P.End of Trial StatusCompleted
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