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    Summary
    EudraCT Number:2020-002822-10
    Sponsor's Protocol Code Number:PL101-HD301
    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:2021-05-24
    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 number2020-002822-10
    A.3Full title of the trial
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Arm, Multicenter Study Evaluating the Efficacy and Safety of Pridopidine in Patients with Early Stage of Huntington Disease
    Studio di fase 3, randomizzato, in doppio cieco, controllato con placebo, a bracci paralleli, multicentrico, volto a valutare l'efficacia e la sicurezza della pridopidina in pazienti con malattia di Huntington in stadio precoce.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of Pridopidine in Patients with Early Stage of Huntington Disease
    Studio della pridopidina in pazienti con malattia di Huntington in stadio precoce
    A.3.2Name or abbreviated title of the trial where available
    PRidopidine Outcome on Function in Huntington Disease (PROOF-HD)
    Esito della pridopidina sulla funzione nella malattia di Huntington
    A.4.1Sponsor's protocol code numberPL101-HD301
    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 SponsorPrilenia Neurotherapeutics Ltd
    B.1.3.4CountryIsrael
    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 supportPrilenia Neurotherapeutics Ltd
    B.4.2CountryIsrael
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPrilenia Neurotherapeutics Ltd.
    B.5.2Functional name of contact pointHead of Clinical Operations
    B.5.3 Address:
    B.5.3.1Street AddressHamenofrim 10
    B.5.3.2Town/ cityHerzliya
    B.5.3.3Post code4672561
    B.5.3.4CountryIsrael
    B.5.4Telephone number00972775558482
    B.5.6E-mailyael.cohen@prilenia.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 number05-2139
    D.3 Description of the IMP
    D.3.1Product namePridopidine
    D.3.2Product code [PL101]
    D.3.4Pharmaceutical form Capsule, hard
    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 INNPRIDOPIDINE HYDROCHLORIDE
    D.3.9.1CAS number 882737-42-0
    D.3.9.2Current sponsor codePridiopidine
    D.3.9.4EV Substance CodeSUB78405
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number45
    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 placeboCapsule, hard
    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
    Huntington Disease
    Malattia di Huntington
    E.1.1.1Medical condition in easily understood language
    A hereditary disorder of the central nervous system that affects muscle
    coordination and leads to cognitive decline and psychiatric problems.
    Una malattia ereditaria del sistema nervoso centrale che colpisce la
    coordinazione muscolare e porta al declino cognitivo e problemi psichiatrici.
    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 10070668
    E.1.2Term Huntington's 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
    To assess the effect of pridopidine on functional capacity in participants with stage 1-2 HD
    Valutare l'effetto della pridopidina sulla capacità funzionale dei partecipanti con HD in stadio 1-2
    E.2.2Secondary objectives of the trial
    Key Secondary
    • TFC responder analysis following pridopidine treatment
    Secondary
    • To evaluate the effect of pridopidine on motor function
    • To evaluate the effect of pridopidine on other measures of efficacy
    Safety and Tolerability:
    • To evaluate the safety and tolerability of pridopidine
    Secondari principali
    • Analisi dei soggetti con TFC in seguito al trattamento con pridopidina
    Secondari
    • Valutare l'effetto della pridopidina sulla funzione motoria
    • Valutare gli effetti della pridopidina sulle altre misure di efficacia
    Sicurezza e Tollerabilità
    • Valutare la sicurezza e la tollerabilità della pridopidina
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Main study:
    1. Twenty-five years of age (inclusive) and older, at the time of signing
    the informed consent.
    2. Diagnosis of HD based on clinical features and the presence of =36
    CAG repeats in the HTT, confirmed by historical laboratory quantified
    results or by a diagnostic test at screening.
    3. Diagnostic confidence level (DCL) of 4 (unequivocal motor signs, =
    99% confidence) on the standardized motor exam UHDRS-TMS.
    4. Adult-onset HD with onset of signs and symptoms =18 years of age.
    5. Stage 1 or Stage 2 HD, defined as a UHDRS-TFC score of =7, at
    screening.
    6. UHDRS-Independence Scale (IS) score =90% at screening.
    7. UHDRS-TMS =20 at the Screening visit.
    8. Must meet all criteria required to move forward with the
    Randomization Authorization Flow (RAF) and be considered eligible by
    the RAF Reviewer.
    9. Male or female.
    10. Female participants of childbearing potential must have a negative ß-
    human chorionic gonadotropin (ß-HCG) test at screening and baseline,
    be sterile, or be postmenopausal.
    11. Female participants of childbearing potential whose male partners
    are potentially fertile (i.e., no vasectomy) must use highly effective birth
    control methods stable for at least 3 months prior to screening, for the
    duration of the study and for 30 days after discontinuation of the study
    drug.
    12. Male participants must be sterile, or if they are potentially
    fertile/reproductively competent (not surgically [e.g., vasectomy] or
    congenitally sterile) and their female partners are of childbearing
    potential, they must use, together with their female partners, effective
    birth control methods for the duration of the study and for 90 days after
    study drug discontinuation.
    13. For participants taking allowed antipsychotic, antidepressant, or
    other psychotropic medication, the dosing of medication as listed in
    Section 10.6, must be stable for at least 4 weeks before the Baseline
    visit and throughout the study (unless clinically necessary to change).
    14. Capable of providing giving signed informed consent which includes
    compliance with the requirements and restrictions listed in the informed
    consent form (ICF) and in this protocol.
    Open-label Extension:
    1. Completed the EoS visit of the Main study
    Studio principale:
    1. Venticinque anni di età (inclusi) al momento della firma del consenso informato.
    2. Diagnosi di HD in base alle caratteristiche cliniche e alla presenza di =36 ripetizioni di
    citosina-adenina-guanina (CAG) nel gene Huntington (HTT), confermate dai risultati
    quantificati storici di laboratorio storici o da un test diagnostico allo screening.
    3. Livello di confidenza diagnostica (DCL) di 4 (segni motori inequivocabili, = 99% di
    confidenza) all’esame motorio standardizzato UHDRS-TMS.
    4. HD con insorgenza da adulto, con manifestazioni di segni e sintomi =18 anni di età.
    5. HD in stadio 1 o 2, definita con un punteggio UHDRS-TFC =7 allo screening.
    6. Punteggio della Scala di indipendenza (IS) UHDRS =90% allo screening.
    7. Punteggio UHDRS-TMS =20 alla visita di screening.
    8. I soggetti devono soddisfare tutti i criteri necessari per proseguire nel percorso di
    autorizzazione alla randomizzazione (RAF) ed essere considerati idonei dal revisore
    RAF.
    9. Pazienti di sesso maschile o femminile.
    10. Le partecipanti in età fertile devono presentare un risultato negativo dell'esame per la
    gonadotropina corionica umana (ß-hCG) allo screening e al basale o essere sterili o in
    post-menopausa.
    11. Le partecipanti in età fertile il cui partner è potenzialmente fertile (ad es., non sottoposto
    a vasectomia) devono utilizzare stabilmente metodi contraccettivi altamente efficaci per
    almeno 3 mesi prima dello screening, per tutta la durata dello studio e per 30 giorni
    successivi all'interruzione del farmaco.
    12. I partecipanti di sesso maschile devono essere sterili o, se potenzialmente fertili o in
    grado di riprodurre (ovvero, non sterili a causa di interventi chirurgici come la
    vasectomia o per motivi congeniti) e con partner di sesso femminile in età fertile, devono
    utilizzare, insieme alla partner, metodi contraccettivi efficaci per l’intera durata dello
    studio e per 90 giorni dopo l'interruzione del farmaco dello studio.
    13. Per i partecipanti che assumono farmaci antipsicotici, antidepressivi o altri farmaci
    psicotropi consentiti, il dosaggio del farmaco come elencato nella sezione 10.6 deve essere stabile per almeno 4 settimane
    prima della visita basale e per tutta la durata dello studio (a meno che una variazione non
    sia clinicamente necessaria).
    14. Essere in grado di fornire il consenso informato firmato che include
    rispetto dei requisiti e delle restrizioni elencati nel
    modulo di consenso informato(ICF) e in questo protocollo
    Stuio in aperto di estensione:
    1 Aver completato la visita EoS dello studio Prinicipale
    E.4Principal exclusion criteria
    1. Prolonged QTcF interval (defined as a QTcF interval of >450 ms for
    male and >470 ms for female) at screening.
    2. Clinically significant heart disease within 12 weeks before
    randomization, defined as follows:
    a. Participants with clinically significant heart disease, a clinically
    significant history of arrhythmia, symptomatic or uncontrolled atrial
    fibrillation despite treatment, or confirmed ventricular tachycardia, or
    presence of left bundle
    branch block.
    b. Participants with a known history of congenital long QT syndrome or a
    first degree relative with this condition.
    c. Heart rate <50 beats per minute, sick sinus syndrome, complete
    atrioventricular block, congestive heart failure, polymorphic ventricular
    tachycardia, clinically relevant hypocalcemia, hypokalemia or
    hypomagnesemia
    3. History of epilepsy or seizures within the last 5 years.
    4. Serious medical illness (includes, but not limited to, uncontrolled
    hypertension; respiratory disease, including severe forms of asthma;
    severe hepatic disease (confirmed Hepatitis B virus [HBV], Hepatitis C
    virus [HCV];, confirmed human immunodeficiency virus [HIV]); renal
    disease; acquired immune deficiency syndrome; and unstable psychiatric
    or other neurologic disorders) and metastatic cancer. For serious kidney
    and liver and liver illnesses see also exclusion criterion 12 (laboratory
    test abnormalities)
    5. Known intracranial neoplasms, vascular malformations, history of
    cerebrovascular accident, or intracranial hemorrhage.
    6. Female participants who are pregnant, planning to become pregnant
    or breastfeeding
    7. Medications that prolong QT interval, taken within 4 weeks of the
    Baseline visit (note, Amiodarone is not allowed within 6 weeks of the
    Baseline visit) or at any timepoint during the study, including nonallowed antipsychotic medications, tricyclic antidepressants, and/or
    Class I antiarrhythmics
    8. Use of pridopidine within 12 months before the Baseline visit.
    9. Treatment with any investigational product within 6 weeks or 5 halflives (whichever is longer) before the Screening visit or a plan to
    participate in another clinical study that assesses any investigational
    product during the study.
    10. Gene therapy at any time
    11. Laboratory values that fall outside of the central laboratory's
    reference range at screening and are considered clinically significantly
    abnormal by the Investigator, and affect the participant's suitability to
    participate in the study or put the participant at risk if he/she enters the
    study in the Investigator's opinion.
    12. Have any of the following laboratory test abnormalities at screening
    a. CrCl <30 mL/min at screening, calculated using the CockcroftGault
    equation: (140–age) × mass (kg) × [0.85 if female] / 72 × serum
    creatinine (mg/dL).
    b. Aspartate aminotransferase (AST) =2.5 × upper limit of normal (ULN)
    c. Alanine aminotransferase (ALT) =2.5 × ULN
    d. Gamma- glutamyl transferase (GGT) =3.0 × ULN
    e. Total bilirubin >1,5 mg/dL
    13. Alcohol and/or substance abuse disorder within the 6 months prior
    to screening, as defined by the Diagnostic and Statistical Manual–Fifth
    Edition (DSM-5) Text Revision criteria for substance abuse.
    14. Active suicidal ideation as measured by a most severe suicide
    ideation score of 4 (Active Suicidal Ideation with Some Intent to Act,
    without Specific Plan) or 5 (Active Suicidal Ideation with Specific Plan
    and Intent) on the C-SSRS, or participants who answered "Yes" on any
    of the 5 C-SSRS Suicidal Behavior Items (actual attempt, interrupted
    attempt, aborted attempt, preparatory acts, or behavior), if the attempt
    or acts were performed within 1 year of screening, or participants who,
    in the opinion of the investigator, present a serious risk of suicide.
    15. Known allergy to any ingredient of the study drug (pridopidine,
    silicified microcrystalline cellulose, or magnesium stearate)
    16. Vulnerable participant (e.g., people kept in detention)
    17. An employee of the Sponsor,Investigator or Investigator study site
    1. Intervallo QTcF prolungato (definito come intervallo QTcF > 450 ms per i maschi e >
    470 ms per le femmine) allo screening2.
    2. Malattia cardiaca clinicamente significativa nelle 12 settimane precedenti alla
    randomizzazione, definita come segue:
    a. Partecipanti con malattie cardiache clinicamente significative, anamnesi
    clinicamente significativa di aritmia, fibrillazione atriale sintomatica o non
    controllata nonostante il trattamento, tachicardia ventricolare confermata2 o
    presenza di blocco di branca sinistro.
    b. Partecipanti con anamnesi nota di sindrome del QT lungo congenita o parente di
    primo grado affetto da questo disturbo.
    c. Frequenza cardiaca <50 battiti al minuto, sindrome del seno malato, blocco
    atrioventricolare completo, insufficienza cardiaca congestizia, tachicardia
    ventricolare polimorfica, ipocalcemia, ipopotassiemia o ipomagnesemia
    clinicamente rilevanti.
    3. Anamnesi di epilessia o crisi convulsive negli ultimi 5 anni.
    4. Una patologia medica grave include, a titolo non limitativo, ipertensione non controllata;
    malattia respiratoria, comprese forme gravi di asma; malattia epatica grave (virus
    dell’epatite B confermato [HBV], virus dell’epatite C [HCV], virus
    dell’immunodeficienza umana confermato [HIV]); malattia renale; sindrome da
    immunodeficienza acquisita; stato psichiatrico instabile o altri disturbi neurologici) e
    tumore metastatico.
    Per le malattie renali ed epatiche gravi si veda anche il criterio di esclusione 12 (valori
    anormali nei test di laboratorio).
    5. Neoplasie intracraniche note, malformazioni vascolari, anamnesi di accidente
    cerebrovascolare o emorragia intracranica.
    6. Partecipanti incinte, che pianificano di restare incinte o in allattamento.
    7. Farmaci che prolungano l'intervallo QT, assunti nelle 4 settimane precedenti alla visita
    basale (notare che Amiodarone non è consentito nelle 6 settimane precedenti alla visita
    basale) o in qualsiasi momento durante lo studio, compresi farmaci antipsicotici,
    antidepressivi triciclici e/o antiaritmici di classe I non consentiti.
    8. Uso di pridopidina nei 12 mesi precedenti alla visita basale.
    9. Trattamento con prodotto sperimentale nelle 6 settimane o 5 emivite (a seconda del
    periodo più lungo) precedenti alla visita di screening o intenzione di partecipare a un altro
    studio clinico che valuta un prodotto sperimentale durante lo studio.
    10. Terapia genica in qualsiasi momento.
    11. I valori di laboratorio che non rientrano nell’intervallo di riferimento del laboratorio
    centrale allo screening e sono considerati anomali in modo clinicamente significativo
    dallo sperimentatore e influiscono sull’idoneità alla partecipazione allo studio o, se il
    partecipante accede allo studio, lo mettono a rischio a parere dello sperimentatore3.
    12. Avere uno qualsiasi dei seguenti valori anormali nei test di laboratorio allo screening:
    a. Clearance della creatinina (CrCl)<30 ml/min/ allo screening, calcolata in base
    all'equazione di Cockcroft-Gault: (140-età) × massa ( kg) × [0.85 se femmina] / 72 ×
    creatinina sierica (mg/dl)
    b. Aspartato aminotransferasi (AST) =2,5 × limite superiore della norma (ULN)
    c. Alanina aminotransferasi (ALT) =2,5 × ULN
    d. Gamma glutamil transferasi (GGT) =3,0 × ULN
    Bilirubina totale >1,5 mg/dl
    13. Abuso di alcol e/o disturbo da abuso di sostanze stupefacenti nei 6 mesi precedenti allo
    screening, come definito dai criteri di revisione del testo del Diagnostic and Statistical
    Manual–Fifth Edition (DSM-5) in relazione all’abuso di sostanze.
    14. Ideazione suicidaria attiva secondo il punteggio di ideazione suicidaria più grave di 4
    o 5 nella scala C-SSRS opartecipanti che hanno risposto “Sì” a una delle 5 voci dei comportamenti suicidari della
    scala C-SSRS se il tentativo o gli atti sono avvenuti entro 1 anno prima dello
    screening, o partecipanti che, a giudizio dello sperimentatore, presentano un grave rischio
    di suicidio.
    15. Allergia nota a un ingrediente del farmaco dello studio
    16. Partecipante vulnerabile
    17.dipendente dello Sponsor
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline to Week 65 in the UHDRS-TFC score
    Variazione dal basale alla settimana 65 del
    punteggio UHDRS-TFC
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and week 65
    Basale e settimana 65
    E.5.2Secondary end point(s)
    Key Secondary
    1. Proportion of participants with no worsening (change = 0 point) from
    baseline to Week 65 in UHDRS-TFC
    Secondary
    2. Change from baseline to Week 65 in the UHDRS-Total Motor Score
    (TMS)
    3. Change from baseline to Week 65 in Quantitative motor (Q-Motor)
    finger tapping (Digitomotography)
    4. Change from baseline to Week 65 in Composite UDHRS (cUHDRS) total
    score
    5. Change from baseline to Week 52 in UHDRS-TFC scoreXML File Identifier: /1DEHRVl0p8ZzQ1/JCwmuJ7DLiQ=
    Page 13/25
    6. Change from baseline to Week 52 in UHDRS-TMS score
    7. Proportion of participants with no worsening from baseline in Clinical
    Global Impression of Change (CGI-C) at Week 65
    8. Change from baseline to Week 78 in UHDRS-TFC score
    Safety and Tolerability
    • Incidence (count and rate) of AEs and serious AEs (SAEs) overall, by
    severity, by relationship to study drug, and those that led to withdrawal
    from the study
    • Incidence and shifts of clinically significant abnormalities in
    electrocardiogram (ECG), laboratory tests and vital signs
    • Analysis of Columbia Suicide Severity Rating Scale (C-SSRS)
    throughout the study
    • Tolerability:
    - The number (%) of participants who complete the treatment period
    - The number (%) of participants who fail to complete the treatment
    period due to AEs
    - The number (%) of participants who fail to complete the treatment
    period due to the prolongation of the Fridericia corrected QT interval
    (QTcF) compared to baseline and QT-change stopping rules
    Secondari principali
    1.Proporzione di partecipanti con nessun
    peggioramento (variazione = 0 punti) del
    punteggio UHDRS-TFC dal basale alla
    settimana 65
    2. Variazione dal basale alla settimana 65 del
    punteggio UHDRS-TMS (Total Motor
    Score)
    3. Variazione dal basale alla settimana 65 nel
    finger tapping nella valutazione quantitativa
    dell’attività motoria (Q-Motor)
    4. Variazione dal basale alla settimana 65 del
    punteggio totale UDHRS composito
    (cUHDRS)
    5. Variazione dal basale alla settimana 52 del
    punteggio UHDRS-TFC
    6. Variazione dal basale alla settimana 52 del
    punteggio UHDRS-TMS
    7. Percentuale di partecipanti con nessun
    peggioramento nel punteggio Clinical
    Global Impression of Change (CGI-C) dal
    basale alla settimana 65
    4. Variazione dal basale alla settimana 65 del
    punteggio totale UDHRS composito
    (cUHDRS)
    5. Variazione dal basale alla settimana 52 del
    punteggio UHDRS-TFC
    6. Variazione dal basale alla settimana 52 del
    punteggio UHDRS-TMS
    7. Percentuale di partecipanti con nessun
    peggioramento nel punteggio Clinical
    Global Impression of Change (CGI-C) dal
    basale alla settimana 65
    Sicurezza e tollerabilità
    Incidenza totale (conteggio e tasso) di
    eventi avversi (AE) ed eventi avversi gravi
    (SAE) in base a gravità, correlazione al
    farmaco dello studio ed eventi che hanno
    portato al ritiro
    • Incidenza e variazioni delle anomalie
    clinicamente significative
    nell'elettrocardiogramma (ECG)1, negli
    esami di laboratorio e nei segni vitali
    • Analisi del punteggio Columbia-Suicide
    Severity Rating Scale (C-SSRS) per l'intera
    durata dello studio
    • Tollerabilità:
    - Numero (%) di partecipanti che
    completano il periodo di trattamento
    - Numero (%) di partecipanti che non
    completano il periodo di trattamento a
    causa di AE
    - Numero (%) di partecipanti che non
    completano il periodo di trattamento a
    causa dell’aumento dell’intervallo QT
    corretto secondo Fridericia (QTcF)
    rispetto al basale e alle regole di
    interruzione per variazione QT
    E.5.2.1Timepoint(s) of evaluation of this end point
    According to the protocol
    In accordo al protocollo
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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
    European Union
    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
    The Main study will be considered completed when all participants
    either complete the study through Week 65 or Week 78 (EoS visit) or
    discontinue earlier (ET visit).
    OLE: Until 6 months after the last participant completes the doubleblind treatment period. OLE duration may be extended pending
    emerging data from the double-blind portion of the study.
    Lo studio principale sarà considerato completato quando tutti i partecipanti
    completano lo studio tra la settimana 65 o la settimana 78 (EoS visita) o interrompono anticipatamente (ET visita).
    OLE: Fino a 6 mesi dopo che l'ultimo partecipante ha completato il periodo di trattamento in doppio cieco. La durata OLE può essere estesa in mancanza di
    dati emergenti dalla porzione in doppio cieco dello studio
    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 days6
    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 days6
    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: 420
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 60
    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 state40
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 240
    F.4.2.2In the whole clinical trial 480
    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
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Huntington Study Group
    G.4.3.4Network Country United States
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-11-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-11-23
    P. End of Trial
    P.End of Trial StatusOngoing
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