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    Summary
    EudraCT Number:2019-001100-38
    Sponsor's Protocol Code Number:BHV3241-301
    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:2021-01-26
    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 number2019-001100-38
    A.3Full title of the trial
    Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects with Multiple System Atrophy (M-STAR Study)
    Studio randomizzato, in doppio cieco, controllato con placebo, a gruppi paralleli per valutare l’efficacia e la sicurezza di BHV-3241 in soggetti con atrofia multisistemica (Studio M-STAR)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess whether a drug called BHV-3241 works and is safe to use in people with Multiple System Atrophy (M-STAR Study)
    Uno studio per valutare se un farmaco chiamato BHV-3241 funziona ed è sicuro da somministrare a persone con Atrofia multisistemica (Studio M-STAR)
    A.3.2Name or abbreviated title of the trial where available
    M-STAR Study
    M-STAR Study
    A.4.1Sponsor's protocol code numberBHV3241-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03952806
    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 SponsorBiohaven Pharmaceuticals, 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 supportBiohaven Pharmaceuticals, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBiohaven Pharmaceuticals, Inc
    B.5.2Functional name of contact pointMedical Information
    B.5.3 Address:
    B.5.3.1Street Address215 Church Street
    B.5.3.2Town/ cityNew Haven, CT
    B.5.3.3Post code06510
    B.5.3.4CountryUnited States
    B.5.4Telephone number+12034040410
    B.5.5Fax number+12032444239
    B.5.6E-mailmedinfo@biohavenpharma.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1404
    D.3 Description of the IMP
    D.3.1Product nameBHV-3241
    D.3.2Product code [/]
    D.3.4Pharmaceutical form Film-coated 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 INNVerdiperstat
    D.3.9.2Current sponsor codeBHV-3241
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    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
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    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
    Multiple System Atrophy
    Atrofia multisistemica
    E.1.1.1Medical condition in easily understood language
    A progressive neurodegenerative disorder that affects the involuntary nervous system and movement.
    Una malattia neurodegenerativa progressiva che colpisce il sistema nervoso involontario e il movimento.
    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 10064060
    E.1.2Term Multiple system atrophy
    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 efficacy of BHV-3241, compared to placebo, as
    measured by a change from baseline in a modified Unified MSA Rating
    Scale (UMSARS), consisting of a subset of items from Part I and Part II,
    at Week 48.
    • To assess the safety and tolerability of BHV-3241, relative to placebo,
    in subjects with MSA.
    - Valutare l’efficacia di BHV-3241 rispetto al placebo, misurata mediante ucambiamento dal basale in una scala unificata di valutazione della MSA
    (UMSARS) modificata, che consiste di un sottogruppo di argomenti dalla
    Parte I e Parte II, alla Settimana 48.
    - Valutare la sicurezza e tollerabilità di BHV-3241, rispetto al placebo, in
    soggetti con MSA.
    E.2.2Secondary objectives of the trial
    Key Secondary
    • To evaluate the efficacy of BHV-3241, compared to placebo, as measured by the Clinical Global Impression of Improvement (CGI-I) score at Week 48.
    •To evaluate the impact of BHV-3241 on quality of life, relative to placebo, as measured by a change from baseline in the motor subscale of the MSA-Quality of Life (MSA-QoL) scale at Week 48.
    • To evaluate the impact of BHV-3241 on quality of life, relative to placebo, as measured by a change from baseline in the non-motor subscale of the MSA- QoL scale at Week 48.
    •To evaluate the efficacy of BHV-3241, compared to placebo, as measured by a change from baseline in the UMSARS Part I and Part II total score at Week 48.

    Other Secondary
    • To assess the impact of BHV-3241, compared to placebo, as measured by a change from baseline at Week 48 in the following instruments:
    - Patient Global Impression of Severity (PGI-S),
    - Clinical Global Impression of Severity (CGI-S),
    - UMSARS Part III,
    - UMSARS Part IV.
    Obiettivi chiave secondari
    - Valutare l’efficacia di BHV-3241, rispetto al placebo, misurata secondo la scala dell’impressione clinica globale di cambiamento (CGI-I) alla Settimana 48.
    - Valutare l'impatto di BHV-3241 sulla qualità della vita, rispetto al placebo, misurata dalla variazione dal basale sulla scala di Qualità della Vita per la MSA Progressiva (MSA-QoL), sotto-scala motoria, alla Settimana 48.
    - Valutare l'impatto di BHV-3241 sulla qualità della vita, rispetto al placebo, misurata dalla variazione dal basale sulla scala MSA-QoL, sotto-scala non motoria, alla Settimana 48.
    - Valutare l’efficacia di BHV-3241 rispetto al placebo, misurata mediante un cambiamento dal basale del punteggio totale di UMSARS Parte I e Parte II, alla Settimana 48.

    Altri obiettivi secondari
    Valutare l’impatto di BHV-3241 rispetto al placebo, misurato mediante un cambiamento dal basale alla Settimana 48 dei seguenti strumenti:
    - PGI-S
    - CGI-S
    - UMSARS Parte III e Parte IV.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Other types of substudies
    Specify title, date and version of each substudy with relative objectives: Title:Optional Cerebrospinal fluid (CFS) sub-study

    Objective: To evaluate BHV-3241 PK concentrations and PD biomarkers in the CSF at Baseline and second at Week 48 or early withdrawal.

    Potential Exploratory CSF analytes may include concentrations of BHV3241 and neurofilament light chain (NfL).

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Titolo: Sottostudio facoltativo sul liquido cefalorachidiano (CSF)

    Obiettivo: Valutare le concentrazioni farmacocinetiche (PK) di BHV-3241 e i biomarcatori predittivi della progressione di malattia (PD) nel CSF al basale e successivamente alla settimana 48 o al ritiro anticipato.

    Potenziali analisi esplorative del CSF potrebbero includere concentrazioni di BHV-3241 e catene leggere del neurofilamento (NfL).
    E.3Principal inclusion criteria
    Informed Consent
    a. Subjects must provide a written signed and dated informed consent form/forms prior to the initiation of any protocol required procedures. Only patients with the capacity to understand the nature, significance, and scope of the clinical trial interventions and to express their wishes accordingly may provide consent to participate in the study.

    b. Caregivers must be willing to sign and date an IRB/EC-approved written informed consent form that outlines the caregiver expectations and responsibilities in this study in accordance with regulatory and institutional guidelines.

    Age and Sex
    a. Male and female subjects between the ages of greater than or equal to 40 to less than or equal to 80 years at time of Screening.

    Target Population
    a. Diagnosis of probable or possible MSA according to consensus clinical
    criteria including subjects with MSA of either subtype (MSA-P or MSAC).

    b. Able to ambulate without the assistance of another person, defined as the ability to take at least 10 steps. Use of assistive devices is allowed.
    c. Anticipated survival of at least 3 years at the time of Screening, as judged by the Investigator.
    d. A brain MRI scan (conducted within the 14 days prior to Baseline/Day 1, approximately) that does not rule out a diagnosis of MSA.
    e. Able to tolerate MRI.
    f. Body mass index (BMI) < 40 kg/m2 at Screening.
    g. Able to swallow tablets whole and anticipated to be able to do so throughout the duration of the study.
    h. Willing and able to adhere to the study drug regimen.
    i. Willing and able to perform all protocol-specified assessments and comply with the study visit schedule.
    j. Able to read, understand, and speak local language fluently to ensure comprehension of informed consent and protocol-specified assessments.
    k. Must have reliable caregiver to accompany subject to all study visits. With the same Caregiver giving caregiver completing assessments at Baseline, Week 24 and Week 48/ Early discontinuation, when possible.
    Caregiver must be able to read, understand, and speak local language fluently to ensure comprehension of informed consent and protocol-specified assessments. Caregiver must also have frequent contact with subject (at least 3 hours per week at one time or different times) and be willing to monitor the subject's health and concomitant medications throughout the study.
    l. If subject is receiving treatment for MSA, the doses must have been stable for at least 30 days prior to Baseline/Randomization and medication present at Baseline expected to remain relatively stable during the study period. This may include medications commonly used for Parkinson's disease or those for autonomic dysfunction.
    m. Stable on other chronic medications and supplements for at least 30 days prior to Baseline/Randomization and expected to remain relatively stable during the study period.
    n. Women of child bearing potential (WOCBP) and fertile men (including those vasectomized for less than 6 months) with female partners who are WOCBP (not surgically sterile and not post-menopausal) must agree to use highly effective birth control, including two methods og contraception, for the duration of the study (beginning 30 days prior to Baseline/Randomization and extending to 30 days for women and 90 days for men after the last dose of study drug). The two methods of contraception should include:
    - one barrier method (e.g. diaphragm with spermicide, condom with spermicidal gel, intrauterine contraceptive devices [placed at least 4 weeks prior to sexual intercourse], cervical cap);
    - and one other method that could include hormonal contraceptives (e.g. oral contraceptives, injectable contraceptives, contraceptive implant, patch) used for at least 4 weeks prior to sexual intercourse.
    o. WOCBP must have a negative serum pregnancy test at Screening and a negative urine pregnancy test within approximately 24 hours prior to dosing at Baseline/Randomization .
    Consenso informato
    a. I soggetti devono fornire il/i modulo/i di consenso informato scritto, apponendo firma e data prima di avviare qualsiasi procedura richiesta dal protocollo. Solo i pazienti con capacità di comprendere la natura, significato e scopo degli interventi della sperimentazione clinica ed esprimere i loro desideri di conseguenza possono fornire il consenso a partecipare allo studio.
    b. I caregiver devono acconsentire a firmare e datare un modulo di consenso informato scritto approvato dall’IRB/EC, che delinea le aspettative e responsabilità del caregiver in questo studio, ai sensi delle linee guida normative e istituzionali.

    Età e sesso
    a. Soggetti uomini e donne di età compresa tra maggiore o uguale 40 e minore o uguale 80 anni al momento dello screening.

    Popolazione target
    a. Diagnosi di probabile o possibile MSA in base a criteri clinici generali, inclusi i soggetti con MSA di entrambe i sottotipi (MSA-P o MSA-C).
    b. In grado di deambulare senza l’assistenza di un’altra persona, definita come la capacità di compiere almeno 10 passi. L’uso di dispositivi di assistenza è consentito.
    c. Sopravvivenza prevista di almeno 3 anni al momento dello screening, in base al parere dello Sperimentatore.
    d. Una RM dell’encefalo (eseguita approssimativamente entro i 14 giorni prima del basale/Giorno 1) che non escluda una diagnosi di MSA.
    e. In grado di tollerare la RM.
    f. Indice di massa corporea (BMI) < 40 kg/m2 allo screening.
    g. In grado di ingerire compresse intere e che si prevede sia in grado di farlo nel corso di tutto lo studio.
    h. Disposto e in grado di aderire al regime di trattamento del farmaco in studio.
    i. Disposto e in grado di eseguire tutte le valutazioni specificate da protocollo e di aderire al piano di visite dello studio.
    j. In grado di leggere, comprendere e parlare fluentemente la lingua locale per garantire la comprensione del consenso informato e delle valutazioni specificate da protocollo.
    k. Presenza di un caregiver affidabile per accompagnare il soggetto a tutte le visite dello studio. Con lo stesso caregiver che fornisce valutazioni del caregiver al
    Baseline, Settimana 24 e Settimana 48 / Interruzione anticipata, quando possibile. Il caregiver deve essere in grado di leggere, comprendere e parlare fluentemente la lingua locale per garantire la comprensione del consenso informato e delle valutazioni specificate da protocollo. Inoltre, il caregiver deve mantenere contatti frequenti con il soggetto (almeno 3 ore alla settimana in una o più sessioni) ed essere disposto a monitorare la salute del paziente e i farmaci concomitanti che assume nel corso dello studio.
    l. Se il soggetto sta ricevendo un trattamento per la MSA, deve presentare stabilità da almeno 30 giorni prima del baseline/randomizzazione e si deve prevedere che rimanga relativamente stabile durante il periodo dello studio. Tale trattamento può includere farmaci comunemente utilizzati per la malattia di Parkinson o farmaci per la disfunzione autonomica.
    m. Stabile con altri farmaci cronici e integratori per almeno 30 giorni prima del baselien/randomizzazione e che si aspetti rimanere relativamente stabile durante il periodo dello studio.

    Spazio esaurito, per gli altri criteri fare riferimento al Protocollo
    E.4Principal exclusion criteria
    Target Disease Exceptions
    a. Subjects having advanced disease as defined in the protocol.
    b. Subjects having significant cognitive impairment, defined by a score of less than or equal to 20 on the MoCA

    Medical History Exclusions
    a. Any condition that would interfere with the subject's ability to comply with study instructions, place the subject at unacceptable risk, and/or confound the interpretation of safety or efficacy data from the study, as judged by the Investigator.
    b. Diagnosis of neurological disorders, other than MSA as defined in the protocol
    c. History of or Screening brain MRI scan indicative of significant abnormality.
    d. Contraindication to MRI examination for any reason.
    e. For optional CSF sub-study: contraindication to undergoing an LP.
    f. Presence of clinically significant thyroid disease with abnormal free T4 levels and TSH >10mIU/L (despite treatment) at Screening , confirmed by repeat.
    g. Within 1 year prior to screening or between screening and Baseline (Day 1), any of the following: myocardial infarction; hospitalization for congestive heart failure; hospitalization for, or symptoms of, unstable angina; or syncope not related to MSA.
    h. Diagnosis of clinically significant psychiatric disorder as defined in the protocol.
    i. History of substance use disorder (drug or alcohol) in the last 12 months, with the exception of nicotine, as defined by DSM-V criteria.
    j. History or presence of gastrointestinal or other disease known to interfere with absorption, distribution, metabolism, or excretion of drugs, or a history of surgery known to interfere with absorption or excretion of drugs excretion of drugs (i.e. gastric bypass).
    k. History of any other clinically significant disease that, based on the judgment of the Investigator, is clinically unstable, is likely to deteriorate during the course of the study, could put the patient at risk because of participation in the study, could affect the subject's ability to complete the study, or could influence the study results.
    l. History of human immunodeficiency virus infection.
    m. Hematologic or solid malignancy diagnosis within 5 years prior to Screening.
    n. Any major surgery within 4 weeks of Screening.
    o. Blood transfusion within 4 weeks of Screening.
    p. History of brain surgery for Parkinsonism.
    q. History of stem-cell treatment.
    r. Women who are pregnant or breastfeeding.
    s. Subjects or prisoners who are involuntarily detained or incarcerated for treatment of either a psychiatric or physical illness must not be enrolled into the study.
    t. Any medical condition, based on the judgement of the Investigator, that would confound the ability to adequately assess safety and efficacy outcome measures

    Physical and Laboratory Test Findings
    Please refer to Protocol for other criteria.
    Eccezioni inerenti la malattia target
    a. Soggetti con malattia avanzata secondo la definizione del protocollo.
    b. Soggetti con deterioramento cognitivo significativo, definito da un punteggio non inferiore o uguale a 20 del MoCA

    Esclusioni inerenti l’anamnesi
    a. Qualsiasi condizione che interferirebbe con la capacità del soggetto di aderire alle istruzioni dello studio, sottoporrebbe il paziente a un rischio inaccettabile e/o confonderebbe l’interpretazione dei dati di sicurezza o efficacia dello studio, in base al parere dello Sperimentatore.
    b. Diagnosi di disturbi neurologici diversi da MSA, secondo la definizione del protocollo
    c. Anamnesi di anomalia significativa o RM dell’encefalo allo screening indicativa della stessa.
    d. Controindicazione all’esame di RM per qualsiasi motivo.
    e. Per il sottostudio facoltativo del CSF: controindicazione alla rachicentesi.
    f. Presenza di patologie tiroidee clinicamente significative con livelli di T4 liberi anormali e TSH> 10mIU / L (nonostante il trattamento) allo screening, confermata da ripetizione.
    g. Nel corso dell’anno precedente allo screening o tra lo screening e il basale (Giorno - 1) qualsiasi dei seguenti eventi: infarto del miocardio; ricovero ospedaliero per insufficienza cardiaca congestizia; ricovero ospedaliero per, o sintomi di, angina instabile; o sincope non correlata a MSA.
    h. Diagnosi di disturbi psichiatrici clinicamente significativi, secondo la definizione del protocollo.
    i. Anamnesi di disturbi dovuti ad utilizzo di sostanze (droga o alcool) negli ultimi 12 mesi, ad eccezione della nicotina, secondo la definizione dei criteri del DSM-V.
    j. Anamnesi o presenza di malattia gastrointestinale o altre malattie che interferiscono con l’assorbimento, la distribuzione, il metabolismo o l’escrezione di farmaci, o un’anamnesi di chirurgia che notoriamente interferisce con l’assorbimento o l’escrezione di farmaci (ad esempio bypass gastrico).
    k. Anamnesi di qualsiasi altra patologia clinicamente significativa che, in base al parere dello Sperimentatore, è clinicamente instabile, presenta la probabilità di deteriorare nel corso dello studio, potrebbe mettere a rischio il paziente a causa della partecipazione allo studio, potrebbe compromettere la capacità del paziente di completare lo studio, o potrebbe influenzare i risultati dello studio.
    l. Anamnesi di infezione da virus di immunodeficienza umana.
    m. Diagnosi di neoplasia maligna ematologica o solida nei 5 anni prima dello screening.
    n. Qualsiasi intervento chirurgico significativo entro 4 settimane dallo screening.
    o. Trasfusione di sangue entro 4 settimane dallo screening.
    p. Anamnesi di chirurgia del cervello per trattare il Parkinson.
    q. Anamnesi di trattamento con cellule staminali.
    r. Donne in stato di gravidanza o che allattano.
    s. Soggetti o detenuti che sono involontariamente detenuti o incarcerati per il trattamento di una malattia psichiatrica o fisica non devono essere arruolati nello studio.
    t. Qualsiasi condizione medica che, in base al parere dello Sperimentatore, potrebbe confondere la capacità di valutare adeguatamente le misure di esito di sicurezza ed efficacia

    Risultati di test fisici e di laboratorio
    Fare riferimento Protocollo per gli altri criteri.
    E.5 End points
    E.5.1Primary end point(s)
    • Change from baseline in a modified UMSARS score, based on a subset of items from Part I and II, at Week 48.
    • The frequency of unique subjects with: serious adverse events; adverse events leading to discontinuation; adverse events judged to be
    related to study medication; clinically significant ECG abnormalities and clinically significant laboratory abnormalities.
    • Variazione dal basale del punteggio modificato UMSARS, in base a un sottogruppo di elementi della Parte I e II, alla settimana 48.
    •Frequenza di soggetti unici in relazione a: eventi avversi seri; eventi avversi che portano all’interruzione; eventi avversi considerati correlati al farmaco in studio; anomalie clinicamente significative dell’ECG e anomalie clinicamente significative di laboratorio.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Efficacy:
    Unified MSA Rating Scale (UMSARS) Parts I-II - Screening, Baseline, Wks 4, 12, 24, 36, 48 or early disontinuation

    Safety:
    Physical Examination; Vital Signs; Sheehan Suicidality Tracking Scale Screening, Baseline, Wks 2, 4, 12, 24, 36, 48 or Early Discontinuation.
    Physical Measurements - Screening, Baseline, Wk48 or Early Discontinuation.
    ECG (12-lead); Laboratory Tests; Pregnancy Test - Screening, Baseline, Wk 4, 12, 24, 36, 48 or Early Discontinuation.
    SAE/AEs - throughout the study.
    Efficacia:
    Scala di valutazione unificata MSA (Unified MSA Rating Scale, UMSARS) Parte I e II - screening, basale, settimane 4, 12, 24, 36, 48 o interruzione anticipata Impressione Clinica Globale della Gravità (Clinical Global Impression of Severity, CGI-S) - basale, settimane 4, 12, 24, 36, 48
    Sicurezza:
    Esame obiettivo; parametri vitali; Sheehan Suicidality Tracking Scale - screening, basale, settimane 2, 4, 12, 24, 36, 48 o interruzione anticipata.
    Misurazioni fisiche - screening, basale, settimana 48 o interruzione anticipata.
    ECG (12 derivazioni); test di laboratorio; test di gravidanza - screening, basale, settimane 4, 12, 24, 36, 48 o interruzione anticipata.
    SAE/AE - durante tutto lo studio
    E.5.2Secondary end point(s)
    Key Secondary
    • The CGI-I score will be evaluated at Week 48.
    • Change from baseline in the MSA-QoL motor subscale at Week 48.
    • Change from baseline in the MSA-QoL non-motor subscale at Week 48.
    • Change from baseline in UMSARS Part I and II total score at Week 48.
    Other Secondary
    • Change from baseline at Week 48 on the following instruments:
    - PGI-S,
    - CGI-S,
    - UMSARS Part III,
    - UMSARS Part IV.
    Secondari chiave
    • Il punteggio CGI-I verrà valutato alla settimana 48.
    • Cambiamenti dal baseline sulla sottoscala motoria del MSA-QoL alla settimana 48.
    • Cambiamenti dal basaline sulla sottoscala non-motoria del MSA-QoL alla settimana 48.
    • Cambiamenti dal baseline sul punteggio totale UMSARS Parte I e II alla settimana 48.

    Altri secondari
    • Cambiamenti dal basale alla settimana 48 con i seguenti strumenti:
    - PGI-S,
    - CGI-S,
    - UMSARS Parte III,
    - UMSARS Parte IV.
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Clinical Global Impression of Improvement (CGI-I)- Wks 4, 12, 24, 36,
    48 or Early Discontinuation.
    • MSA QoL at Baseline and Weeks 24 and 48 or Early Discontinuation
    • UMSARS (Part I - IV) - Screening, Baseline, Wks 4, 12, 24, 36, 48 or early discontinuation
    • Patient Global Impression of Severity (PGI-S) - Baseline, Wks 4, 12, 24, 36, 48 or Early Discontinuation
    • Clinical Global Impression of Severity (CGI-S) - Baseline, Wks 4, 12, 24, 36, 48 or Early Discontinuation
    • Impressione Clinica Globale del Miglioramento (CGI-I) - settimane 4, 12, 24, 36, 48 o interruzione anticipata.
    • MSA QoL al basale e settimane 24 e 48 o interruzione anticipata
    • UMSARS (Parte I - IV) - screening, basale, settimane 4, 12, 24, 36, 48
    • Impressione Globale del Paziente della Gravità (PGI-S) - basale, settimane 4, 12, 24, 36, 48 o interruzione anticipata
    • Impressione Clinica Globale della Gravità (CGI-S) - basale, settimane 4, 12, 24, 36, 48 o interruzione anticipata
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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
    Austria
    China
    France
    Germany
    Italy
    United Kingdom
    United States
    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
    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 months10
    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 months4
    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: 126
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 126
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 119
    F.4.2.2In the whole clinical trial 252
    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)
    none
    nessuno
    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 Decision2020-02-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-12-10
    P. End of Trial
    P.End of Trial StatusOngoing
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