Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43857   clinical trials with a EudraCT protocol, of which   7284   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    Essai multicentrique de traitement de la maladie de Huntington par la cystéamine

    Summary
    EudraCT number
    		 2010-019444-39
    Trial protocol
    		 FR  
    Global end of trial date
    31 Aug 2017

    Results information
    Results version number
    		 v1(current)
    This version publication date
    09 Sep 2020
    First version publication date
    09 Sep 2020
    Other versions
    Summary report(s)
    		 résumé RF CYST-HD

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    PHRC2004-03bis
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03296176
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    CHU Angers
    Sponsor organisation address
    4 rue Larrey, Angers, France,
    Public contact
    Pr Dominique Bonneau, CHU Angers, dobonneau@chu-angers.fr
    Scientific contact
    Pr Dominique Bonneau, CHU Angers, dobonneau@chu-angers.fr
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    21 Jun 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    28 Feb 2017
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Aug 2017
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Evaluer l'effet de la cystéamine chez des patients symptomatiques atteints de la maladie de Huntington en comparant les deux groupes de patients (cystéamine versus placebo) sur les résultats de l'Unified Huntington Disease Rating Scale (UHDRS, Huntington Study Group 1996). Le critère principal de l'étude sera la progression du score moteur de l'UHDRS en comparant les valeurs obtenues dans le groupe traité aux valeurs obtenues dans le groupe contrôle à 18 mois.
    Protection of trial subjects
    Une comparaison de l'évolution sera effectuée sur 18 mois avant/après traitement sur l'ensemble des patients
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    21 Oct 2010
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Scientific research
    Long term follow-up duration
    		 72 Months
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 96
    Worldwide total number of subjects
    96
    EEA total number of subjects
    96
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    96
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 Il s’agit d’un essai thérapeutique de phase II-III national, multicentrique, randomisé contrôlé cystéamine versus placebo en double aveugle Les sujets sont sélectionnés par les investigateurs et parmi les patients atteints de la maladie de Huntington, volontaires, pris en charge par les services de neurologie et de génétique de chacun des centres

    Pre-assignment
    Screening details
    		 La phase d’inclusion des patients est prévue sur 2 visites à 1 mois d’intervalle : M-1 et M0. Ce n’est qu’à l’issue de cette deuxième visite que la décision d’inclusion pourra être prise après avoir vérifié les différents critères et avoir fait signer le consentement au moins en 2 exemplaires par les différentes parties.

    Period 1
    Period 1 title
    double aveugle
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 traitement
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    RP103
    Investigational medicinal product code
    cysteamine
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Le traitement à l'étude est le RP103 dosé à 75mg. Le principe actif est le bitartrate de cystéamine. Le traitement se présente sous la forme de gélules à libération retardée de cystéamine, de taille 0. Les excipients sont les suivants : cellulose microcristalline, hypromellose, sodium laurylsulfate et eau purifiée. L'enrobage gastro-résistant est constitué par l'Eudragit L30D55, du citrate triethyle et du talc. Les traitements sont administrés par voie orale deux fois par jour à 12 heures d'intervalle

    Arm title
    		 placebo
    Arm description
    		 -
    Arm type
    		 Placebo

    Investigational medicinal product name
    placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Les traitements sont administrés par voie orale deux fois par jour à 12 heures d'intervalle. Le traitement comparateur est représenté par des gélules de placebo de même taille et de même aspect. Les gélules de placebo sont des gélules composées de microsphères de sucrose et d'amidon associés à du talc comme lubrifiant.

    Number of subjects in period 1
    		traitement placebo
    Started
    51
    45
    Completed
    45
    44
    Not completed
    6
    1
         Lost to follow-up
    6
    1
    Period 2
    Period 2 title
    traitement compassionnel
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 traitement
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    RP103
    Investigational medicinal product code
    cysteamine
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Le traitement à l'étude est le RP103 dosé à 75mg. Le principe actif est le bitartrate de cystéamine. Le traitement se présente sous la forme de gélules à libération retardée de cystéamine, de taille 0. Les excipients sont les suivants : cellulose microcristalline, hypromellose, sodium laurylsulfate et eau purifiée. L'enrobage gastro-résistant est constitué par l'Eudragit L30D55, du citrate triethyle et du talc. Les traitements sont administrés par voie orale deux fois par jour à 12 heures d'intervalle

    Number of subjects in period 2 [1]
    		traitement
    Started
    86
    Completed
    66
    Not completed
    20
         Adverse event, serious fatal
    8
         Consent withdrawn by subject
    5
         Adverse event, non-fatal
    5
         Lost to follow-up
    1
         Protocol deviation
    1
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: 10 patients n'ont pas souhaité participer aux différentes phases de l'étude
    Period 3
    Period 3 title
    prolongation de l'étude
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 prolongation de l'étude
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    RP103
    Investigational medicinal product code
    cysteamine
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Le traitement à l'étude est le RP103 dosé à 75mg. Le principe actif est le bitartrate de cystéamine. Le traitement se présente sous la forme de gélules à libération retardée de cystéamine, de taille 0. Les excipients sont les suivants : cellulose microcristalline, hypromellose, sodium laurylsulfate et eau purifiée. L'enrobage gastro-résistant est constitué par l'Eudragit L30D55, du citrate triethyle et du talc. Les traitements sont administrés par voie orale deux fois par jour à 12 heures d'intervalle

    Number of subjects in period 3
    		prolongation de l'étude
    Started
    66
    Completed
    14
    Not completed
    52
         Adverse event, serious fatal
    4
         Physician decision
    2
         Consent withdrawn by subject
    2
         discontinuation of treatment by the laboratory
    37
         Adverse event, non-fatal
    1
         Pregnancy
    1
         Lack of efficacy
    1
         Protocol deviation
    4

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    traitement
    Reporting group description
    		 -

    Reporting group title
    placebo
    Reporting group description
    		 -

    Reporting group values
    		 traitement placebo Total
    Number of subjects
    		 51 45 96
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 51 45 96
    Age continuous
    Age compris entre 18 et 65 ans.
    Units: years
        median (standard deviation)
    		 45.5 ± 8.9 50.4 ± 9.7 -
    Gender categorical
    Units: Subjects
        Female
    		 21 25 46
        Male
    		 30 20 50
    Subject analysis sets

    Subject analysis set title
    Phase primaire : M0 à M18 placebo versus traitement
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    L’effet de la cystéamine sur le fonctionnement moteur, négativement impactée dans le cas de la maladie de Huntington, a été étudié en opposant une population traitée et une population témoin choisies de manière randomisée en double-aveugle parmi les 96 patients de l’étude. Trois mesures de score moteur ont été effectuées à 0, 12 et 18 mois. Après 18 mois de traitement vs placebo, nous n’avons pas mis en évidence un effet significatif du traitement sur l’évolution du score moteur (SMD = -1.5 ± 1.71, P=0.385).

    Subject analysis set title
    Etude M0 à M72 (sans placebo)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Nous avons effectué une deuxième phase d’analyse en incorporant les données de la phase de prolongation. Nous voyons qu’un nombre important de patients a choisi de poursuivre le traitement à l’issu des 36 mois de l’étude (plus que 4 visites). Les patients avec 9 visites ont complété l’intégralité de la phase de prolongation.

    Subject analysis sets values
    		 Phase primaire : M0 à M18 placebo versus traitement Etude M0 à M72 (sans placebo)
    Number of subjects
    96
    86
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    96
    86
    Age continuous
    Age compris entre 18 et 65 ans.
    Units: years
        median (standard deviation)
    46.9 ± 9.4
    46.9 ± 9.4
    Gender categorical
    Units: Subjects
        Female
    46
    41
        Male
    50
    45

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    traitement
    Reporting group description
    		 -

    Reporting group title
    placebo
    Reporting group description
    		 -
    Reporting group title
    traitement
    Reporting group description
    		 -
    Reporting group title
    prolongation de l'étude
    Reporting group description
    		 -

    Subject analysis set title
    Phase primaire : M0 à M18 placebo versus traitement
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    L’effet de la cystéamine sur le fonctionnement moteur, négativement impactée dans le cas de la maladie de Huntington, a été étudié en opposant une population traitée et une population témoin choisies de manière randomisée en double-aveugle parmi les 96 patients de l’étude. Trois mesures de score moteur ont été effectuées à 0, 12 et 18 mois. Après 18 mois de traitement vs placebo, nous n’avons pas mis en évidence un effet significatif du traitement sur l’évolution du score moteur (SMD = -1.5 ± 1.71, P=0.385).

    Subject analysis set title
    Etude M0 à M72 (sans placebo)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Nous avons effectué une deuxième phase d’analyse en incorporant les données de la phase de prolongation. Nous voyons qu’un nombre important de patients a choisi de poursuivre le traitement à l’issu des 36 mois de l’étude (plus que 4 visites). Les patients avec 9 visites ont complété l’intégralité de la phase de prolongation.

    Primary: phase primaire

    		 Close Top of page
    End point title
    		 phase primaire
    End point description
    End point type
    Primary
    End point timeframe
    Trois mesures de score moteur ont été effectuées à 0, 12 et 18 mois
    End point values
    		 traitement placebo
    Number of subjects analysed
    45
    44
    Units: SMD = -1.5 ± 1.71
    1
    1
    Statistical analysis title
    		 phase primaire
    Comparison groups
    traitement v placebo
    Number of subjects included in analysis
    89
    Analysis specification
    Post-hoc
    Analysis type
    		 superiority
    P-value
    		 = 0.385
    Method
    		 score moteur
    Confidence interval

    Primary: phase deux

    		 Close Top of page
    End point title
    		 phase deux
    End point description
    End point type
    Primary
    End point timeframe
    M0 à M72
    End point values
    		 traitement placebo traitement prolongation de l'étude
    Number of subjects analysed
    51
    45
    86
    66
    Units: score moteur
    51
    45
    86
    66
    Statistical analysis title
    		 Phase primaire : M0 à M18 placebo vs traitement
    Comparison groups
    traitement v placebo
    Number of subjects included in analysis
    96
    Analysis specification
    Post-hoc
    Analysis type
    		 other [1]
    P-value
    		 = 0.385
    Method
    		 motor score
    Parameter type
    		 Mean difference (final values)
    Point estimate
    1.5
    Confidence interval
         level
    		 90%
         sides
    1-sided
         lower limit
    		 -
         upper limit
    		 2
    Variability estimate
    Standard deviation
    Dispersion value
    1.71
    Notes
    [1] - évaluer l’effet de la cystéamine chez les patients symptomatiques

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Les événements indésirables ont été recueillis pendant toute la durée de l'étude de M0 à M72.
    Adverse event reporting additional description
    Les événements indésirables non graves sont décrits par bras pendant la phase en double aveugle de M0 à M18. Les évènements indésirables graves sont décrits sur la totalité de l'étude de M0 à M72 sans mention de bras de traitement étant donné que l'aveugle n'a été levé que pour les suspicions d'effets indésirables graves.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18
    Reporting groups
    Reporting group title
    M0-M72
    Reporting group description
    		 -

    Serious adverse events
    		 M0-M72
    Total subjects affected by serious adverse events
         subjects affected / exposed
    35 / 96 (36.46%)
         number of deaths (all causes)
    5
         number of deaths resulting from adverse events
    5
    Vascular disorders
    Haemorrhage intracranial
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Haematoma
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Surgical and medical procedures
    Pilonidal sinus repair
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Aborted pregnancy
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Oedema peripheral
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Consciousness fluctuating
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Death
    Additional description: unknown cause of death (at home)
    alternative assessment type: Non-systematic
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    2 / 2
    Respiratory, thoracic and mediastinal disorders
    Foreign body aspiration
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Psychiatric disorders
    Anxiety disorder
    alternative assessment type: Non-systematic
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Depressive symptom
    alternative assessment type: Non-systematic
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Suicide attempt
    alternative assessment type: Non-systematic
         subjects affected / exposed
    7 / 96 (7.29%)
         occurrences causally related to treatment / all
    3 / 7
         deaths causally related to treatment / all
    3 / 3
    Delusional disorder, unspecified type
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Disorientation
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hallucination
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hallucination, auditory
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Panic attack
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Suicidal ideation
    alternative assessment type: Non-systematic
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    2 / 2
    Violence-related symptom
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Weight decreased
    alternative assessment type: Non-systematic
         subjects affected / exposed
    3 / 96 (3.13%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Alcohol abuse
    alternative assessment type: Non-systematic
         subjects affected / exposed
    3 / 96 (3.13%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Fall
    alternative assessment type: Non-systematic
         subjects affected / exposed
    3 / 96 (3.13%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Fracture
    alternative assessment type: Non-systematic
         subjects affected / exposed
    3 / 96 (3.13%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Road traffic accident
    alternative assessment type: Non-systematic
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Myocardial infarction
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Tachyarrhythmia
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Tachycardia
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Hypotonia
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Confusional state
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Language disorder
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Lymphopenia
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Eye disorders
    Cataract
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Enzyme activity increased
    alternative assessment type: Non-systematic
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Peritonitis
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pleural infection bacterial
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory tract infection
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Viral infection
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Bursitis infective
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Feeding disorder
    alternative assessment type: Non-systematic
         subjects affected / exposed
    2 / 96 (2.08%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 M0-M72
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 96 (12.50%)
    Investigations
    Alanine aminotransferase increased
    Additional description: Possibly related-treatment adverse event in M0-M18 phase
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences all number
    1
    Blood creatine phosphokinase increased
    Additional description: Possibly related-treatment adverse event in M0-M18 phase
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences all number
    1
    Aspartate aminotransferase increased
    Additional description: Possibly related-treatment adverse event in M0-M18 phase
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences all number
    1
    Nervous system disorders
    Headache
    Additional description: Possibly related-treatment adverse event in M0-M18 phase
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences all number
    1
    General disorders and administration site conditions
    Fatigue
    Additional description: Possibly related-treatment adverse event in M0-M18 phase
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences all number
    1
    Gastrointestinal disorders
    Diarrhoea
    Additional description: Possibly related-treatment adverse event in M0-M18 phase
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences all number
    1
    Nausea
    Additional description: Possibly related-treatment adverse event in M0-M18 phase
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences all number
    1
    Breath odour
    Additional description: Possibly related-treatment adverse event in M0-M18 phase
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences all number
    1
    Vomiting
    Additional description: Possibly related-treatment adverse event in M0-M18 phase
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences all number
    1
    Abdominal pain
    Additional description: Possibly related-treatment adverse event in M0-M18 phase
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences all number
    1
    Infections and infestations
    Gastroenteritis
    Additional description: Possibly related-treatment adverse event in M0-M18 phase
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Decreased appetite
    Additional description: Possibly related-treatment adverse event in M0-M18 phase
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 96 (1.04%)
         occurrences all number
    1

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    17 May 2011
    Modification d’un critère de non-inclusion
    20 Sep 2011
    Prolongation de la durée de recrutement avec modification de la durée d’essai clinique
    19 Jun 2012
    Changement d’investigateur principal dans un lieu de recherche déjà déclaré
    17 Sep 2013
    changement de statisticien et Modification de la période de traitement
    10 Jul 2014
    Ajout des co-investigateurs et Modification de la période de traitement
    03 Feb 2015
    Modification de la période de traitement
    07 Jul 2015
    Ajout d’une IRM durant la troisième période de prolongation de l’étude (visite M63). Suppression du dosage du BDNF aux visites M63 et M72. Ajout d'un co-investigateur. Changement d’investigateur principal dans un lieu de recherche déjà déclaré
    26 May 2016
    Ajout d’une étude ancillaire PK-CYST-HD et Changement du nom/coordonnées du représentant légal du promoteur
    20 Sep 2016
    Modification du suivi des patients suite à l’arrêt prématuré ou non du traitement

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/28436572
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Thu Apr 25 06:52:18 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA