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    Summary
    EudraCT Number:2019-002108-41
    Sponsor's Protocol Code Number:MT-1186-A01
    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:2021-06-17
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2019-002108-41
    A.3Full title of the trial
    A Phase 3, Multi-center, Open-label, Safety Study of Oral Edaravone Administered over 48 Weeks in Subjects with Amyotrophic Lateral Sclerosis (ALS)
    Studio di Fase 3, multicentrico, in aperto, sulla sicurezza di edaravone orale somministrato per un periodo di 48 settimane in soggetti con sclerosi laterale amiotrofica (SLA)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Safety Study of Oral Edaravone in Subjects with Amyotrophic Lateral Sclerosis (ALS)
    Studio sulla sicurezza di edaravone orale in soggetti con sclerosi laterale amiotrofica (SLA)
    A.3.2Name or abbreviated title of the trial where available
    n/a
    n/a
    A.4.1Sponsor's protocol code numberMT-1186-A01
    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 SponsorMitsubishi Tanabe Development America Inc.
    B.1.3.4CountryUnited States
    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 supportMitsubishi Tanabe Pharma Development America, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMitsubishi Tanabe Pharma Development America, Inc.
    B.5.2Functional name of contact pointDaniel Selness
    B.5.3 Address:
    B.5.3.1Street Address525 Washington Boulevard, Suite 400
    B.5.3.2Town/ cityJersey City
    B.5.3.3Post codeNJ 07310
    B.5.3.4CountryUnited States
    B.5.4Telephone number7048829
    B.5.5Fax number7048829
    B.5.6E-maildaniel_selness@mt-pharma-us.com
    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 nameEdaravone
    D.3.2Product code [MT-1186]
    D.3.4Pharmaceutical form Oral suspension
    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.3.9.1CAS number 89-25-8
    D.3.9.2Current sponsor codeMT-1186
    D.3.9.4EV Substance CodeSUB06453MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number105
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Amyotrophic Lateral Sclerosis (ALS)
    Sclerosi Laterale Amiotrofica (SLA)
    E.1.1.1Medical condition in easily understood language
    A disease that affects nerve cells in the brain and the spinal cord
    Una malattia che colpisce le cellule nervose del cervello e del midollo spinale
    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 21.1
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    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 evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral Sclerosis (ALS) over 24 and 48 weeks
    Valutare la sicurezza e la tollerabilità a lungo termine di edaravone per via orale in soggetti con sclerosi laterale amiotrofica (SLA) nell’arco di 24 e 48 settimane
    E.2.2Secondary objectives of the trial
    Not applicable
    non applicabile
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects must provide signed and dated informed consent form (ICF) to participate in the study. Subjects must be able to (in the judgement of the Investigator) understand the nature of the study and all risks involved with participation in the study. Subjects must be willing to cooperate and comply with all protocol restrictions and requirements.
    2. Subjects will be male or female, = 18 to 75 years of age at the time the ICF is signed.
    3. Subjects will be diagnosed with Definite ALS, Probable ALS, Probable laboratory-supported ALS, or Possible ALS according to the El Escorial revised criteria for the diagnosis of ALS.
    4. Subjects will be living and functioning independently (eg, able to eat, excrete, ambulate independently without assistance of others). The use of supportive tools and adaptive utensil is allowed.
    5. Subjects will have a baseline forced vital capacity percentage (%FVC) = 70%.
    6. Subjects whose first symptom of ALS has occurred within 3 years at the time of providing written informed consent.
    1. Per partecipare allo studio i soggetti devono fornire un modulo di consenso informato (ICF) firmato e datato. A giudizio dello Sperimentatore, i soggetti devono essere in grado di comprendere la natura dello studio e tutti i rischi che la partecipazione allo studio comporta. I soggetti devono essere disposti a collaborare e a ottemperare a tutte le limitazioni e a tutti i requisiti del protocollo.
    2. I soggetti saranno di sesso maschile o femminile, di età compresa tra 18 (inclusi) e 75 anni al momento della firma dell'ICF.
    3. Ai soggetti sarà stata diagnosticata SLA definita, SLA probabile, SLA probabile suffragata dai dati di laboratorio o SLA possibile in base ai criteri El Escorial rivisti per la diagnosi di SLA.
    4. I soggetti vivranno in modo indipendente e saranno funzionalmente indipendenti (es. in grado di mangiare, evacuare, camminare in modo indipendente, senza assistenza da parte di terzi). È consentito l’uso di strumenti di supporto e utensili adattivi.
    5. I soggetti avranno una percentuale di capacità vitale forzata (FVC%) al basale = 70%.
    6. Soggetti i cui primi sintomi di SLA si siano manifestati nei 3 anni precedenti il momento della consegna del consenso informato scritto.
    E.4Principal exclusion criteria
    Exclusions Related to General Health or Concomitant Conditions:
    1. Subjects undergoing treatment for a malignancy or those with a pending biopsy result.
    2. Subjects who have the presence or history of any clinically significant disease (except ALS) that could interfere with the objectives of the study (the assessment of safety and efficacy) or the safety of the subject, as judged by the Investigator.
    3. Subjects of childbearing potential unwilling to use an acceptable method of contraception from the screening visit until 3 months after the last dose of study medication. Subjects who are sexually active and who do not agree to use contraception during the study period.
    4. Subjects who are female and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1).
    5. Subjects who have a significant risk of suicide. Subjects with any suicidal behavior or suicidal ideation of type 4 (active suicidal ideation with some intent to act, without a specific plan) or type 5 (active suicidal ideation with specific plan and intent) based on the Columbia–Suicide Severity Rating Scale (C-SSRS) within the 3 months before the screening visit.
    6. Subjects who have alanine aminotransferase (ALT) or aspartate aminotransferase (AST) elevations greater than 2 times the upper limit of normal (ULN) at screening.
    7. Subjects with a glomerular filtration rate (GFR) <30 mL/min per 1.73 m2 at screening.

    Exclusions Related to Medications:
    8. Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites.
    9. Subjects with hereditary fructose intolerance.
    10. Subjects who participated in another study and were administered an investigational product within 1 month or 5 half-lives of the investigational agent, whichever is longer before providing informed consent for the present study.
    11. Subjects who are unable to take their medications orally.
    Esclusioni relative alle condizioni di salute generali o a condizioni concomitanti.
    1. Soggetti sottoposti a trattamento per una neoplasia o in attesa del risultato di una biopsia.
    2. Soggetti con qualunque malattia clinicamente significativa (eccetto la SLA) in atto o nell’anamnesi, che potrebbe interferire con gli obiettivi dello studio (la valutazione di sicurezza ed efficacia) o con la sicurezza del soggetto, a giudizio dello Sperimentatore.
    3. Soggetti di sesso femminile in grado di procreare che non siano disposte a usare un metodo contraccettivo accettabile dalla visita di screening fino a 3 mesi dopo l’ultima dose del farmaco in studio. Soggetti sessualmente attivi che non acconsentano a usare un contraccettivo durante il periodo dello studio.
    4. Soggetti di sesso femminile gravide (con un test di gravidanza positivo) o che allattano alla visita di screening (Visita 1).
    5. Soggetti con un significativo rischio suicidario. Soggetti con comportamento suicidario o ideazione suicidaria di tipo 4 (ideazione suicidaria attiva con qualche intenzione di agire, senza un piano specifico) o di tipo 5 (ideazione suicidaria attiva con un piano specifico e con intenzione) sulla base della Scala della Columbia University per la valutazione della gravità del rischio di suicidio (C-SSRS, Columbia-Suicide Severity Rating Scale) nei 3 mesi precedenti la visita di screening.
    6. Soggetti con aumenti di ALT o AST >2 volte l’ULN allo screening.
    7. Soggetti con velocità di filtrazione glomerulare (GFR) <30 mL/min/1,73 m2.

    Esclusioni relative ai farmaci:
    8. Soggetti con eventi pregressi di ipersensibilità a edaravone, agli additivi o eccipienti di edaravone o ai solfiti.
    9. Soggetti con intolleranza ereditaria al fruttosio.
    10. Soggetti che abbiano partecipato a un altro studio con somministrazione di un prodotto sperimentale entro 1 mese o 5 emivite dell’agente sperimentale, a seconda di quale dei due periodi abbia maggior durata, prima di fornire il consenso informato per il presente studio.
    11. Soggetti non in grado di assumere farmaci per via orale.
    E.5.1
    E.5 End points
    E.5.1Primary end point(s)
    The primary safety endpoints are to evaluate the safety and tolerability of oral edaravone and include the following safety assessments:
    - Adverse events (AEs), adverse drug reactions (ADRs), and treatment-emergent adverse events ([TEAEs] eg, grade, incidence and severity);
    - Physical examination;
    - Body weight;
    - 12-lead electrocardiogram (ECG) parameters;
    - Vital signs (heart rate, sitting systolic and diastolic blood pressure, and axillary, oral, or tympanic body temperature);
    - Laboratory safety assessments (eg, hematology, chemistry, and urinalysis);
    - Unsteadiness and sensory evaluation (eg, assessment of unsteadiness and peripheral sensation will be evaluated by assessment of vibratory sensation with a tuning fork applied to the lateral side of the right and left ankles);
    - Columbia–Suicide Severity Rating Scale (C-SSRS);
    - Forced vital capacity percentage (%FVC).
    Gli endpoint primari di sicurezza sono la valutazione della sicurezza e della tollerabilità di edaravone per via orale e includeranno le seguenti valutazioni per la sicurezza:
    - Eventi avversi (AE), reazioni avverse da farmaco (ADR, adverse drug reaction) ed eventi avversi emergenti dal trattamento (TEAE, treatment emergent adverse event) (es. grado, incidenza, gravità).
    - Esame obiettivo.
    - Peso corporeo.
    - Parametri dell’elettrocardiogramma (ECG) a 12 derivazioni.
    - Parametri vitali (frequenza cardiaca, pressione sistolica e diastolica da seduti e temperatura corporea ascellare, orale o timpanica).
    - Valutazioni di laboratorio (es. esami ematologici, ematochimici e analisi delle urine).
    - Valutazione dell’instabilità e del sensorio (es. la valutazione dell’instabilità e della sensibilità periferica sarà condotta attraverso la valutazione della sensibilità vibratoria applicando un diapason sul lato esterno delle caviglie destra e sinistra).
    - C-SSRS.
    - FVC%.
    E.5.1.1Timepoint(s) of evaluation of this end point
    - Visit 1- 15
    - Visit 1, 2, 4, 6, 9, 12 and 15
    - Visit 1, 2, 4, 6, 9, 12 and 15
    - Visit 1, 2, 9 and 15
    - Visit 1, 2, 4, 6, 9, 12 and 15
    - Visit 1, 2, 4, 6, 9, 12 and 15
    - Visit 1, 2, 4, 6, 9, 12 and 15
    - Visit 1, 6, 9 and 15
    - Visit 1, 2, 4, 6, 9, 12 and 15
    - alla Visita 1- 15
    - alla Visita 1, 2, 4, 6, 9, 12 e 15
    - alla Visita 1, 2, 4, 6, 9, 12 e 15
    - alla Visita 1, 2, 9 e 15
    - alla Visita 1, 2, 4, 6, 9, 12 e 15
    - alla Visita 1, 2, 4, 6, 9, 12 e 15
    - alla Visita 1, 2, 4, 6, 9, 12 e 15
    - alla Visita 1, 6, 9 e 15
    - alla Visita 1, 2, 4, 6, 9, 12 e 15
    E.5.2Secondary end point(s)
    Not applicable.
    non applicabile
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable.
    non applicabile
    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 Yes
    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 No
    E.8.1.2Open Yes
    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 Yes
    E.8.2.3.1Comparator description
    n/a
    n/a
    E.8.2.4Number of treatment arms in the trial1
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    Canada
    Japan
    United States
    France
    Germany
    Italy
    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 years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    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: 75
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 75
    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 Yes
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Male subjects with partners of child-bearing potential using contraception
    Soggetti di sesso maschile con partner in grado di procreare che utilizzano metodi contraccettivi
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 150
    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)
    Subjects who complete the study and are compliant may (based upon criteria) be eligible to roll over into a long-term open-label treatment study.
    I soggetti che completano lo studio e che risultano conformi possono (in base ai criteri di cui sopra) essere idonei a proseguire con uno studio di trattamento in aperto a lungo termine.
    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 Decision2020-04-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-06
    P. End of Trial
    P.End of Trial StatusCompleted
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    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.

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