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    Clinical Trial Results:
    A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults with Phenylketonuria

    Due to the EudraCT – Results system being out of service between 31 July 2015 and 12 January 2016, these results have been published in compliance with revised timelines.
    Summary
    EudraCT number
    2010-024311-13
    Trial protocol
    DE   IT   BE   ES   NL  
    Global end of trial date
    07 Nov 2014

    Results information
    Results version number
    v1(current)
    This version publication date
    28 Jul 2016
    First version publication date
    28 Jul 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    700773-004
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01977820
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Merck Serono, a division of Merck KGaA
    Sponsor organisation address
    Frankfurter Strasse 250, Darmstadt, Germany, 64293
    Public contact
    Communication Centre merck KGaA, Merck KGaA, Merck Serono, a division of Merck KGaA, +49 6151725200, service@merckgroup.com
    Scientific contact
    Communication Centre merck KGaA, Merck KGaA, Merck Serono, a division of Merck KGaA, +49 6151725200, service@merckgroup.com
    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
    07 Nov 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Nov 2014
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The purpose of this pilot trial is to assess the effect of sapropterin on the cognitive abilities of young adults with phenylketonuria (PKU).
    Protection of trial subjects
    Subject protection was ensured by following high medical and ethical standards in accordance with the principles laid down in the Declaration of Helsinki, and that are consistent with Good Clinical Practice and applicable regulations
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    24 Feb 2014
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Italy: 1
    Country: Number of subjects enrolled
    Switzerland: 1
    Worldwide total number of subjects
    2
    EEA total number of subjects
    1
    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)
    2
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    First/Last subject (informed consent): 24 Feb 2014/12 Aug 2014. Study premature termination date: 07 Nov 2014; Subjects were randomized at 2 study centers.

    Pre-assignment
    Screening details
    A total of 10 subjects were screened for the trial. 2 subjects were randomized (1 subjects in the Sapropterin and 1 subjects in the placebo).

    Period 1
    Period 1 title
    Overall study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Double blind
    Roles blinded
    Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Experimental: Sapropterin
    Arm description
    Sapropterin tablets was administered orally once daily during both the 2-Week response test period and 24-Week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.
    Arm type
    Experimental

    Investigational medicinal product name
    Sapropterin
    Investigational medicinal product code
    Other name
    Kuvan
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received Sapropterin tablets orally once daily at a dose of 20 milligram per kilogram (mg/kg) during both the 2-Week response test period and 24-Week study period.

    Arm title
    Drug: Placebo
    Arm description
    Subjects was administered Sapropterin tablets orally once daily during the 2-Week response test period and Placebo tablets matching to Sapropterin was administered orally once daily during the 24-Week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.
    Arm type
    Active comparator

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received Sapropterin tablets orally once daily at a dose of 20 mg/kg during both the 2-Week response test period and Placebo tablets matching to Sapropterin was administered orally once daily during the 24-Week study period.

    Number of subjects in period 1
    Experimental: Sapropterin Drug: Placebo
    Started
    1
    1
    Completed
    0
    0
    Not completed
    1
    1
         Sponsor terminated the study
    1
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Experimental: Sapropterin
    Reporting group description
    Sapropterin tablets was administered orally once daily during both the 2-Week response test period and 24-Week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.

    Reporting group title
    Drug: Placebo
    Reporting group description
    Subjects was administered Sapropterin tablets orally once daily during the 2-Week response test period and Placebo tablets matching to Sapropterin was administered orally once daily during the 24-Week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.

    Reporting group values
    Experimental: Sapropterin Drug: Placebo Total
    Number of subjects
    1 1 2
    Age categorical
    Units: Subjects
        >=18 to 29 Years
    1 1 2
    Gender categorical
    Units: Subjects
        Female
    1 0 1
        Male
    0 1 1

    End points

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    End points reporting groups
    Reporting group title
    Experimental: Sapropterin
    Reporting group description
    Sapropterin tablets was administered orally once daily during both the 2-Week response test period and 24-Week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.

    Reporting group title
    Drug: Placebo
    Reporting group description
    Subjects was administered Sapropterin tablets orally once daily during the 2-Week response test period and Placebo tablets matching to Sapropterin was administered orally once daily during the 24-Week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.

    Primary: Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation

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    End point title
    Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation [1]
    End point description
    An AE was defined as any new untoward medical occurrences/worsening of pre-existing medical condition without regard to possibility of causal relationship. A SAE was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect. The safety analysis population included all the randomized subjects who received at least one dose of study treatment.
    End point type
    Primary
    End point timeframe
    Screening up to 24 weeks + 4-week follow-up
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive data was planned to be analyzed.
    End point values
    Experimental: Sapropterin Drug: Placebo
    Number of subjects analysed
    1
    1
    Units: subjects
        AEs
    1
    0
        SAEs
    0
    0
        AEs leading to death
    0
    0
        AEs leading to discontinuation
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Screening up to 24 weeks + 4-week follow-up
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    14.1
    Reporting groups
    Reporting group title
    Sapropterin
    Reporting group description
    Subject was administered with 20 mg/kg sapropterin tablets orally once daily during the 2-week response test period. The subject upon completing the 2-Week response test period was randomized to receive sapropterin during the 24-week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.

    Reporting group title
    Placebo
    Reporting group description
    Subject was administered with 20 mg/kg sapropterin tablets orally once daily during the 2-week response test period. The subject upon completing the 2-Week response test period was randomized to receive placebo tablets matching to sapropterin orally once daily during the 24-week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.

    Serious adverse events
    Sapropterin Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 1 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Sapropterin Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 1 (0.00%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    Gastrointestinal disorders
    Abdominal Pain
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0
    Aphthous stomatitis
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 1 (0.00%)
         occurrences all number
    1
    0
    Abdominal pain upper
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 1 (0.00%)
         occurrences all number
    2
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? No

    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.
    The study was prematurely discontinued by the Sponsor due to severe enrolment difficulties as a consequence of the limited availability of treatment naïve patients with a diagnosis of phenyl ketonuria.
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