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    Summary
    EudraCT Number:2014-005367-32
    Sponsor's Protocol Code Number:PROMISE
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-03-17
    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 number2014-005367-32
    A.3Full title of the trial
    PROTEIN MISFOLDING, AMYOTROPHIC LATERAL SCLEROSIS AND GUANABENZ: A PHASE II RCT WITH FUTILITY DESIGN
    MISFOLDING PROTEICO, SCLEROSI LATERALE AMIOTROFICA E GUANABENZ: STUDIO CLINICO RANDOMIZZATO DI FASE II CON DISEGNO DI FUTILITA'
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical study on the efficacy and tolerabilty of Guabenz in Amiotropic Lateral Sclerosis
    STUDIO CLINICO SULL'EFFICACIA E TOLLERABILITA' DEL GUANABENZ COME TRATTAMENTO DELLA SCLEROSI LATERALE AMIOTROFICA
    A.3.2Name or abbreviated title of the trial where available
    PROMISE
    PROMISE
    A.4.1Sponsor's protocol code numberPROMISE
    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 SponsorFONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA
    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 supportARISLA
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA
    B.5.2Functional name of contact pointSERVIZIO RICERCA E SVILUPPO CLINICO
    B.5.3 Address:
    B.5.3.1Street AddressVIA CELORIA 11
    B.5.3.2Town/ cityMILANO
    B.5.3.3Post code20133
    B.5.3.4CountryItaly
    B.5.4Telephone number+390223942321
    B.5.5Fax number+390223943569
    B.5.6E-mailcrc@istituto-besta.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 No
    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 nameGUANABENZ ACETATO
    D.3.2Product code GUANA2015
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNGUANABENZ
    D.3.9.1CAS number 23256-50-0
    D.3.9.2Current sponsor codeGUANA2015
    D.3.9.3Other descriptive nameGUANABENZ
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number8
    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 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) 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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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
    D.2.1.1.2Name of the Marketing Authorisation holder
    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.3.1Product nameRILUZOLO
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRILUZOLO
    D.3.9.1CAS number 1744-22-5
    D.3.9.2Current sponsor codeRILU2015
    D.3.9.3Other descriptive nameRILUZOLO
    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 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) 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Amiotrophic Lateral Sclerosis
    Sclerosi Laterale Amiotrofica
    E.1.1.1Medical condition in easily understood language
    Amiotrophic Lateral Sclerosis
    Sclerosi Laterale Amiotrofica
    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 19.1
    E.1.2Level PT
    E.1.2Classification code 10028003
    E.1.2Term Motor neurone disease
    E.1.2System Organ Class 10029205 - Nervous system 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 investigate the effect of guanabenz as add-on treatment compared to riluzole alone in reducing the proportion of patients progressed to higher stages of disease over 6 months in sporadic (SALS) or familial ALS (FALS) patients.
    Investigare l'effetto di guanabenz come terapia aggiuntiva al riluzolo verso placebo, per 6 mesi di trattamento, nella riduzione della proporzione di pazienti progrediti a uno stadio più grave di patologia, sia per pazienti SLA sporadici che familiari.
    E.2.2Secondary objectives of the trial
    To assess safety and tolerability related to alpha2agonist activity of guanabenz. To explore serum biomarkers of ALS progression (creatinine, albumin tau, pNfH, TDP43, cystatin C, fetuin A, transthyretin, and CD14/S100ß and pNfH/C3) in guanabenz and riluzole alone arms at baseline and study end. In patients giving consent the biomarkers will be explored also in the cerebrospinal fluid.
    Dimostrare la sicurezza e la tollerabilità dell'attività alpha2agonista di guanabenz.
    Studiare i biomarkes sierici di progressione(creatinina, albumina, tau, pNfH, TDP43, cistatina C, fetuina A, transtiretina, e CD14/S100ß e pNfH/C3) nei bracci dello studio al basale e alla fine dello studio. Nei pazienti che forniranno il consenso i biomarkers saranno esplorati anche nel liquor.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Diagnosis of, probable, probable laboratory supported or definite sporadic (SALS) or familiar (FALS) ALS according to the revised El Escorial criteria; 2) age >18 yrs; 3) onset ≤18 months before randomization; 4) sVC ≥70% in spinal onset; 5) riluzole 100 mg/day or no riluzole; 6) written informed consent
    1) diagnosi probabile, probabile con supporto di laboratorio o definita di SLA sporadica o familiare in accordo ai criteri di El Escorial rivisti;
    2) età >18 anni;
    3) esordio di malattia ≤18 mesi prima della randomizzazione;
    4) capacità vitale lenta ≥70% nelle forme spinali;
    5) trattamento con riluzolo alla dose di 100mg/giorno oppure non assunzione riluzolo;
    6) cpnsenso informato scritto.
    E.4Principal exclusion criteria
    1) PEG, NIV or tracheotomy; 3) known heart, renal or liver failure; 3) known intolerance to alfa2agonists; 4) known conditions at risk for cardiovascular disorders or symptomatic hypotension; 5) participation in a clinical trial within 3 months prior to the screening; 6) cognitive impairment defined by the Edinburg Cognitive and Behavior ALS Screen (ECAS) scale

    1) PEG, Ventilazione non invasiva, tracheostomia;
    2) nota insufficienza cardiaca, renale ed epatica;
    3) nota intolleranza agli alpha2agonisti;
    4) nota condizione di rischio cardiovascolare o ipotensione sintomatica;
    5) partecipazione ad altri studi clinci nei 3 mesi precedenti lo screening;
    6) deficit cognitivo definto attraverso la scala ECAS ( Edinburg Cognitive and Behavior ALS Screen);
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of patients progressed to higher stages of disease at 6 months (measured by the ALS-MITOS system) by at least 35% in the guanabenz arm compared to their baseline stage and to the placebo arm (null hypothesis in the futility design).
    Proporzione di pazienti progrediti ad un più grave stadio di malattia a 6 mesi ( misurato attraverso la scala MITO) di almento 35% nel braccio con guanabenz rispetto al loro basale e al gruppo placebo (ipotesi nulla del disegno di futilità).
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 months
    6 mesi
    E.5.2Secondary end point(s)
    3) Difference in at least one serum and/or CSF biomarker of neurodegeneration (creatinine, albumin, tau, pNfH, TDP43, cystatin C, fetuin A, transthyretin, and CD14/S100ß and pNfH/C3 assayed by ELISA and Western blot) comparing baseline and study end between guanabenz and placebo arm.
    3) Differenza in almento uno dei biomarkes di neurodegenerazione sierici e/o liquorali (creatinina, albumina, tau, pNfH, TDP43, cistatina C, fetuina A, transtiretina, e CD14/S100ß e pNfH/C3 effettuati con ELISA e Western blot) nei bracci di trattamento sia al basale che a fine studio.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At baseline and at the end of the study
    Basale e fine studio
    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 Yes
    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 No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Studio di futilità
    Futility study
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial4
    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 concerned25
    E.8.5The trial involves multiple Member States No
    E.8.5.1Number of sites anticipated in the EEA23
    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 years0
    E.8.9.1In the Member State concerned months36
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months36
    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: 1
    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.1.2.1Number of subjects for this age range: 166
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 42
    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 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 2017-03-17. Yes
    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 state208
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 208
    F.4.2.2In the whole clinical trial 208
    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)
    Normal clinical practice
    I pazienti proseguiranno secondo normale pratica clinica
    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 Decision2016-02-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-10-28
    P. End of Trial
    P.End of Trial StatusOngoing
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