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    Summary
    EudraCT Number:2015-000904-24
    Sponsor's Protocol Code Number:TV7820-CNS-20016
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-02-23
    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 number2015-000904-24
    A.3Full title of the trial
    A Multi-Center, Open-Label Study Evaluating the Safety, Tolerability, and Efficacy of Pridopidine in Patients with Huntington's Disease
    Studio multicentrico in aperto finalizzato alla valutazione della sicurezza, della tollerabilità e dell’efficacia di pridopidina in pazienti affetti da malattia di Huntington
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study testing if pridopidine is safe and efficacious in patients with Huntington's Disease
    Studio per testare se pridopidina è sicura ed efficace in pazienti affetti da malattia di Huntington
    A.3.2Name or abbreviated title of the trial where available
    Open PRIDE-HD
    Open PRIDE-HD
    A.4.1Sponsor's protocol code numberTV7820-CNS-20016
    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 SponsorTEVA BRANDED PHARMACEUTICAL PRODUCTS R&D, 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 supportTeva Branded Pharmaceutical Products R&D, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTeva Pharma GmbH
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressWaldecker Str. 7
    B.5.3.2Town/ cityMoerfelden-Walldorf
    B.5.3.3Post code64546
    B.5.3.4CountryGermany
    B.5.4Telephone number00 00 00 00
    B.5.5Fax number00 00 00 00
    B.5.6E-mailInfo.era-clinical@teva.de
    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/3/05/288
    D.3 Description of the IMP
    D.3.1Product namePridopidina
    D.3.2Product code TV-7820
    D.3.4Pharmaceutical form Capsule, hard
    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 INNPridopidina
    D.3.9.1CAS number 346688-38-8
    D.3.9.2Current sponsor codeTV-7820
    D.3.9.3Other descriptive nameACR16 hydrochloride, ASP2314 hydrochloride, FR310826
    D.3.9.4EV Substance CodeSUB47886
    D.3.10 Strength
    D.3.10.1Concentration unit mg/g milligram(s)/gram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number22.5
    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 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/3/05/288
    D.3 Description of the IMP
    D.3.1Product namePridopidina
    D.3.2Product code TV-7820
    D.3.4Pharmaceutical form Capsule, hard
    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 INNPridopidina
    D.3.9.1CAS number 346688-38-8
    D.3.9.2Current sponsor codeTV-7820
    D.3.9.3Other descriptive nameACR16 hydrochloride, ASP2314 hydrochloride, FR310826
    D.3.9.4EV Substance CodeSUB47886
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Huntington's disease
    Malattia di Huntington
    E.1.1.1Medical condition in easily understood language
    An hereditary disorder of the central nervous system that affects muscle
    coordination and leads to cognitive decline and psychiatric problems.
    Disordine
    ereditario del sistema nervoso centrale che colpisce il coordinamento
    muscolare e porta ad un declino cognitivo ed a 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
    The primary objective of this study is to evaluate safety and tolerability
    of pridopidine in patients with HD
    L’obiettivo primario del presente studio è valutare la sicurezza e la tollerabilità di pridopidina in pazienti affetti da HD
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are to assess the effects of longterm, open-label dosing with pridopidine on motor symptom severity,
    overall patient function, physical performance, and healthrelated quality of life.
    Gli obiettivi secondari dello studio prevedono la valutazione degli effetti del dosaggio in aperto, a lungo termine, di pridopidina sulla gravità dei sintomi motori, le funzionalità complessive del paziente, le prestazioni fisiche e la qualità della vita correlata allo stato di salute
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    a. Patient has completed the PRIDE-HD ( TV7820-CNS-20002) trial within the last 6 months, including the follow-up period. For patients that have failed to complete PRIDE-HD or those who completed the PRIDE-HD study over 6 months prior to the screening visit, and meet all other inclusion criteria, eligibility should be discussed with the Open-PRIDE medical monitor and clinician on a case-by-case basis.
    b. For patients that experienced a drug-related SAE or had a major compliance violation during PRIDE-HD, eligibility approval should be
    discussed with PRIDE-HD's medical monitor and clinician.
    c. Able and willing to provide written informed consent prior to any study related procedure being performed. Patients with a legal guardian should be consented according to local requirements.
    d. Females of child bearing potential have to be compliant in using adequate birth control throughout the duration of the study. Adequate
    birth control is defined as consistent practice of an effective and accepted method of contraception according to local regulations (hormone-based, intrauterine device, or double barrier contraception,
    i.e., condom and diaphragm). Abstinence is an acceptable method of contraception only when this is the preferred and usual lifestyle of the subject. Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), and lactational amenorrhoea
    method (LAM) are not acceptable methods of contraception. Male study participants have to be compliant in using adequate birth control with
    their partners (as defined above) throughout the duration of the study.
    e. Body weight ≥50 kg. Patients that meet this criterion at screening but fail to meet it at a subsequent visit may be discontinued based on
    Investigator or Sponsor discretion. It is allowed to repeat the weight measurement once, if clinically appropriate.
    f. Willing and able to take oral medication and able to comply with the study specific procedures.
    g. Ambulatory, being able to travel to the study center, and judged by the investigator as likely to be able to continue to travel for the duration of the study.
    h. The patient is in good health as determined by medical examination, ECG, serum chemistry, hematology, and urinalysis.
    i. The patient must be willing and able to comply with study restrictions and to remain at the clinic for the required duration during the study period, and willing to return to the clinic for evaluation as specified in this protocol.
    a. Il paziente ha completato la sperimentazione PRIDE-HD ( TV7820-CNS-20002) negli ultimi 6 mesi, incluso il periodo di follow-up. Per i pazienti che non sono riusciti a completare PRIDE-HD o per coloro che hanno completato lo studio PRIDE-HD nell'arco dei 6 mesi precedenti la visita di screening e soddisfano tutti gli altri criteri di inclusione, è necessario discutere dell’idoneità con il medico e il responsabile del monitoraggio medico di Open-PRIDE, caso per caso.
    b. Per i pazienti che hanno riscontrato un evento avverso grave (Serious Adverse Event, SAE) correlato al farmaco o una violazione maggiore della conformità durante PRIDE-HD, è necessario discutere dell’approvazione dell’idoneità con il medico e il responsabile del monitoraggio medico di PRIDE-HD.
    c. Capacità e volontà di fornire il proprio consenso informato scritto, prima dell’avvio di qualsiasi procedura correlata allo studio. I pazienti assistiti da un tutore legale dovranno fornire il consenso in base alle disposizioni di legge locali.
    d. Le donne potenzialmente fertili devono adottare un valido metodo contraccettivo per l’intera durata dello studio. Per valido metodo contraccettivo si definisce l’adozione costante di un metodo anticoncezionale efficace e accettato in base alle normative locali (metodo ormonale, dispositivo intrauterino o metodo della doppia barriera, ovvero, preservativo e diaframma). L’astinenza è un metodo contraccettivo accettabile solo se rispecchia lo stile di vita di preferenza e abituale del soggetto. L’astinenza periodica (metodo del calendario, sintotermico, post-ovulazione), il coito interrotto e il metodo basato sull’amenorrea da allattamento (Lactational Amenorrhea Method, LAM) non sono metodi contraccettivi accettabili. I partecipanti allo studio che siano di sesso maschile devono adottare un valido metodo di contraccezione con la propria partner (come sopra definito) per l’intera durata dello studio.
    e. Peso corporeo ≥50 kg. Per i pazienti che soddisfano questo criterio allo screening ma non a una visita successiva potrebbe essere necessario interrompere lo studio, a discrezione dello sperimentatore o dello sponsor. È possibile ripetere una volta la misurazione del peso, se clinicamente appropriato.
    f. Capacità e volontà di assumere il farmaco orale e capacità di seguire le procedure specifiche dello studio.
    g. Capacità deambulatoria, di recarsi al centro di sperimentazione e, secondo l’opinione dello sperimentatore, probabile capacità di recarsi al centro per gli appuntamenti fissati nell’arco dell’intera durata dello studio
    h. Il paziente è in buona salute, come confermato dall’esame medico, dall’ECG, dall’ematochimica, dall’ematologia e dall’analisi delle urine.
    i. Il paziente deve accettare ed essere in grado di rispettare le restrizioni dello studio e rimanere in clinica per la durata richiesta durante il periodo dello studio. Inoltre, deve essere in grado di ripresentarsi in clinica per una valutazione, come specificato nel presente protocollo.
    E.4Principal exclusion criteria
    a. A prolonged QTcF interval (defined as a QTcF interval of >450 msec) at the screening visit. ECGs will be performed in triplicate during the screening visit, and the mean of the 3 QTcF measurements will be used to determine whether or not the patient is suitable for inclusion in the study.
    b. Patients with clinically significant heart disease at the screening visit, defined asfollows: (i) significant cardiac event (eg, myocardial infarction), angina pectoris or episode of congestive heart failure with symptoms >Grade 2 New York Heart
    Association classification within 12 weeks before randomization, or presence of cardiac disease that in the opinion of the investigator increased the risk
    of ventricular arrhythmia, (ii) history of arrhythmia (multifocal premature ventricular contractions, bigeminy, trigeminy, ventricular tachycardia) that was symptomatic or required treatment (Common
    Terminology Criteria for Adverse Events Grade 3), symptomatic or uncontrolled atrial fibrillation despite treatment, or asymptomatic sustained ventricular tachycardia, (iii) presence of left bundle branch block.
    c. Patients with serum potassium, magnesium, and/or calcium levels outside of the central laboratory's reference range at the screening visit and considered clinically significantly abnormal by the investigator. Repeat testing is allowed (up
    to a maximum of 3 tests) if required to establish whether values are within normal range or clinically significantly abnormal.
    d. Patients receiving medications (within the last 6 weeks prior to baseline) other than pridopidine that have been proven to prolong QT interval or who may require such medications during the course of the study such as but not limited to non-allowed anti-psychotic medications, tricyclic antidepressants, and/or Class I antiarrhythmics.
    e. Patients receiving medications (within the last 6 weeks prior to baseline) other than pridopidine that are metabolized by CYP2D6 and have the potential of reducing seizure threshold.
    f. Patients with a history of epilepsy or of seizures within the last 5 years.
    g. Creatinine clearance <60 mL/min at screening, calculated using the Cockcroft-Gault equation: (140 - age) × mass (kg) × [0.85 if female] /
    72 × serum creatinine (mg/ dL). It is allowed to repeat the test once, if clinically appropriate.
    h. Any clinically significant, abnormal, screening laboratory result which in the opinion of the investigator, affects the patients' suitability for the
    study or puts the patient at risk if he/she enters the study.
    i. Patients with 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 patients who answer "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 patients who, in the opinion of the investigator, present a serious risk of suicide.
    j. Females who are pregnant or breastfeeding.
    k. Treatment with any investigational product other than pridopidine within 6 weeks of screening or patients planning to participate in another clinical study assessing any investigational product during the study.
    saranno esclusi dallo studio i pazienti che soddisfino uno qualsiasi dei criteri indicati di seguito:
    a. Intervallo QT prolungato corretto con formula di Fridericia (Fridericia-corrected QT, QTcF) (definito come intervallo QTcF di >450 ms) nell’ambito della visita di screening. Gli ECG saranno eseguiti in triplicato durante la visita di screening e il mezzo delle 3 misurazioni QTcF sarà utilizzato per determinare se il paziente sia idoneo all’inclusione nello studio.
    b. Pazienti con malattie cardiache clinicamente significative nell’ambito della visita di screening, definite come indicato di seguito: (i) evento cardiaco significativo (ad es., infarto del miocardio), angina pectoris o episodio di insufficienza cardiaca congestizia con sintomi >grado 2 della New York Heart Association Classification (Classificazione funzionale dell’associazione cardiaca di New York) entro 12 settimane dalla
    randomizzazione o presenza di patologia cardiaca che, a giudizio dello sperimentatore, ha aumentato il rischio di aritmia ventricolare, (ii) anamnesi di aritmia (contrazioni ventricolari premature multifocali, bigeminia, trigeminia, tachicardia ventricolare) sintomatica o per la quale è stato necessario un trattamento (Criteri comuni di terminologia per eventi avversi di grado 3), fibrillazione atriale sintomatica o non controllata nonostante il trattamento o tachicardia ventricolare sostenuta asintomatica, (iii) presenza di blocco di branca sinistra.
    c. Pazienti con livelli sierici di potassio, magnesio e/o calcio, nell’ambito della visita di screening, al di fuori dell'intervallo di riferimento indicato dal laboratorio centrale e considerati anomali in modo clinicamente significativo dallo sperimentatore. La ripetizione del test è consentita (fino a un massimo di 3 test) se necessaria per stabilire se i valori rientrino nell'intervallo normale o siano anomali in modo clinicamente significativo.
    d. Pazienti in trattamento con farmaci (nelle ultime 6 settimane precedenti il basale) diversi da pridopidina che hanno dimostrato di prolungare l’intervallo QT o che potrebbero richiedere l’assunzione di tali farmaci nel corso dello studio, come, in via esemplificativa ma non esaustiva, farmaci antipsicotici non consentiti, antidepressivi triciclici e/o antiaritmici di classe I.
    e. Pazienti in trattamento con farmaci (nelle ultime 6 settimane precedenti il basale) diversi da pridopidina e metabolizzati dal citocromo P450 (CYP) 2D6 e con potenziale effetto riduttivo della soglia convulsiva.
    f. Pazienti con anamnesi di epilessia o crisi epilettiche negli ultimi 5 anni.
    g. Clearance della creatinina <60 ml/min nell’ambito della visita di screening, calcolata in base all’equazione di Cockcroft-Gault:
    (140 - età) × massa (kg) × [0,85 se donna]/72 × creatinina sierica (mg/dl). È consentito ripetere il test una volta, se clinicamente appropriato.
    h. Qualsiasi risultato di laboratorio, nell’ambito dello screening, che sia anomalo, clinicamente significativo, che, secondo l’opinione dello sperimentatore, comprometta l’idoneità del paziente allo studio o ponga il paziente a rischio qualora vi prenda parte.
    i. Pazienti con ideazione suicidaria attiva secondo un punteggio di ideazione suicidaria più grave di 4 (ideazione suicidaria attiva con parziale intenzione ad agire, senza piano specifico) o 5 (ideazione suicidaria attiva, con piano specifico e intenzione) in base alla scala di valutazione della gravità del suicidio della Columbia (Columbia-Suicide Severity Rating Scale, C-SSRS) oppure soggetti che rispondono “Sì” a una qualsiasi delle 5 voci del comportamento suicidario della C-SSRS (tentativo reale, tentativo interrotto, tentativo abortito, atti preparatori oppure comportamento) se il tentativo o gli atti sono stati compiuti entro 1 anno dallo screening, oppure soggetti che, a giudizio dello sperimentatore, presentano un grave rischio di suicidio.
    j. Donne in gravidanza o in allattamento.
    k. Trattamento con qualsiasi prodotto sperimentale diverso da pridopidina nelle 6 settimane precedenti lo screening o pazienti che abbiano intenzione di partecipare a un altro studio clinico volto a valutare qualsiasi prodotto sperimentale durante lo studio.
    E.5 End points
    E.5.1Primary end point(s)
    The safety endpoints for this study are as follows:
    - adverse events (AEs) throughout the study
    - vital signs assessments
    - concomitant medication usage throughout the study
    - physical examination findings
    - clinical laboratory evaluations (hematology, clinical chemistry, and urinalysis)
    - ECG findings
    - Suicidality [using the Columbia-Suicide Severity Rating Scale (C-SSRS)]
    The tolerability endpoints for this study are as follows:
    - Proportion of subjects (%) who prematurely discontinued from the study
    - Proportion of subjects (%) who prematurely discontinued from the study due to AEs
    Endpoint di sicurezza:
    Le misure di sicurezza includeranno quanto indicato di seguito:
    -Eventi avversi (EA)
    -Valutazioni dei segni vitali
    -Utilizzo di farmaci concomitanti
    -Risultati derivanti dall’esame obiettivo
    -Valutazioni cliniche di laboratorio (ematologia, chimica clinica e analisi delle urine)
    -Risultati dell’elettrocardiogramma (ECG)
    -Tendenze suicide [utilizzo della Scala di valutazione della gravità del suicidio della Columbia (Columbia-Suicide Severity Rating Scale, C-SSRS)]
    Endpoint di tollerabilità:
    -Percentuale di soggetti (%) che si sono ritirati precocemente dallo studio
    -Percentuale di soggetti (%) che si sono ritirati precocemente dallo studio a causa di EA
    E.5.1.1Timepoint(s) of evaluation of this end point
    The following safety and tolerability measures will be assessed during the study:
    - AEs – monitored at every study visit.
    - Vital signs assessments – monitored at every study visit.
    - Concomitant medication usage - monitored at every study visit.
    - Physical examination findings – brief physical examination will be performed at every study visit.
    - Clinical laboratory evaluations (hematology, clin. chemistry, urinalysis)
    - at each study visit
    - ECG findings – ECG will be performed at every study visit
    - Suicidality (C-SSRS) will be assessed throughout the study.
    Tolerability endpoints:
    - Proportion of subjects (%) who prematurely discontinued from the study
    - Proportion of subjects (%) who prematurely discontinued from the study due to AEs
    Le seguenti misure di sicurezza e tollerabilità verrano rilevate durante lo studio: -Eventi avversi (EA)-monitorato ad ogni visita -Valutazioni dei segni vitali-monitorato ad ogni visita -Utilizzo di farmaci concomitanti_monitorato ad ogni visita -Risultati derivanti dall’esame obiettivo_un breve esame fisico verrà effettuato ad ogni visita. -Valutazioni cliniche di laboratorio (ematologia, chimica clinica e analisi delle urine)-ad ogni visita -Risultati dell’ECG - ECG verrà effettuato ad ogni visita -Tendenze suicide [Columbia-Suicide Severity Rating Scale, C-SSRS]-verrà determinato durante lo studio Endpoint di tollerabilità: -Percentuale di soggetti che si sono ritirati precocemente dallo studio • Percentuale di soggetti che si sono ritirati precocemente dallo studio a causa di EA
    E.5.2Secondary end point(s)
    Long-term efficacy evaluations are the secondary objective of this study. Efficacy endpoints include the following: - Unified Huntington's Disease Rating Scale – Total Motor Score (UHDRSTMS) - Modified Physical Performance Test (mPPT) -Unified Huntington's Disease Rating Scale - Total Functional Capacity (UHDRSTFC) - Short Form 12 (SF-12) questionnaire (acute version) - Problem Behaviors Assessment-Short form (PBA-s)
    Le valutazioni dell’efficacia a lungo termine rappresentano l’obiettivo secondario del presente studio. Gli endpoint di efficacia includono quanto indicato di seguito -Scala di valutazione della malattia di Huntington unificata – Punteggio totale motorio (Unified Huntington’s Disease Rating Scale – Total Motor Score, UHDRS-TMS) -Test delle prestazioni fisiche modificate (Modified Physical Performance Test, mPPT) -Scala di valutazione della malattia di Huntington unificata - Capacità funzionale totale (Unified Huntington’s Disease Rating Scale - Total Functional Capacity, UHDRS-TFC) -Questionario Short Form 12 (SF-12) (versione abbreviata) -Modulo breve della valutazione dei comportamenti problematici (Problem Behaviors Assessment-Short form, PBA-s)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy analyses will be considered exploratory. Comparisons will generally be made to baseline, as appropriate. Efficacy endpoints include the following: - UHDRS-TMS - will be performed at all visits during the study. - mPPT - will be performed at baseline, week 12, week 26, week 52 and week 54. UHDRS-TFC - will be performed at baseline week 12, week 26, week 52 and week 54.
    SF-12 evaluation will be performed at baseline week 12, week 26, week 52 and week 54.
    Le analisi dell’efficacia saranno considerate esplorative.I confronti saranno effettuati generalmente al basale, a seconda delle necessità. Gli endpoints di efficacia includono i seguenti: - UHDRS-TMS saranno effettuate in occasione di tutte le visite. -mPPT- sarà eseguito al basale, nonché durante le settimane 12, 26, 52 e 54. -- UHDRS-TFC -sarà eseguita al basale, nonché durante le settimane 12, 26, 52 e 54.
    SF-12 sarà eseguita al basale, nonché durante le settimane 12, 26, 52 e 54.
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    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 Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    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 No
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA33
    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
    Australia
    Austria
    Canada
    Denmark
    France
    Germany
    Italy
    Netherlands
    Poland
    Russian Federation
    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 months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months1
    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: 368
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 32
    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 No
    F.4 Planned number of subjects to be included
    F.4.1In the member state45
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 280
    F.4.2.2In the whole clinical trial 400
    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 Decision2015-08-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-09-15
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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