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    Clinical Trial Results:
    Single Center, Therapeutic Exploratory Clinical Trial to Evaluate the Safety of Sodium Oxybate (Xyrem) 500 mg/mL Oral Solution on Potential Endocrine Changes at Currently Labeled Therapeutic Dose Regimens (4.5 - 9 g/Day Divided Into Two Equal Doses) During 12 Weeks of Treatment of Cataplexy in Adult Patients With Narcolepsy

    Summary
    EudraCT number
    		 2005-004417-15
    Trial protocol
    		 BE  
    Global end of trial date
    22 Jan 2008

    Results information
    Results version number
    		 v1(current)
    This version publication date
    30 Jun 2016
    First version publication date
    14 Jun 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    C00301
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT00345800
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    UCB Pharma SA
    Sponsor organisation address
    Chemin du Foriest, Braine-l’Alleud, Belgium, B-1420
    Public contact
    Clinical Trial Registries and Results Disclosure, UCB BIOSCIENCES GmbH, +49 2173 4815 15, clinicaltrials@ucb.com
    Scientific contact
    Clinical Trial Registries and Results Disclosure, UCB BIOSCIENCES GmbH, +49 +49 2173 48 15 15, clinicaltrials@ucb.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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    29 Apr 2008
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    22 Jan 2008
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    • To monitor for endocrine changes in response to treatment of cataplexy with Xyrem • To focus on the hypothalamic pituitary axis
    Protection of trial subjects
    Not applicable
    Background therapy
    		 Not applicable
    Evidence for comparator
    		 Not applicable
    Actual start date of recruitment
    10 Apr 2006
    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
    Belgium: 25
    Worldwide total number of subjects
    25
    EEA total number of subjects
    25
    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)
    1
    Adults (18-64 years)
    23
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This single-Center, therapeutic, exploratory study started to enroll subjects in April 2006.

    Pre-assignment
    Screening details
    		 Participant Flow refers to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Sodium Oxybate
    Arm description
    		 Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sodium Oxybate
    Investigational medicinal product code
    Sodium Oxybate
    Other name
    Xyrem
    Pharmaceutical forms
    Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    Active Substance: Sodium Oxybate Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks

    Number of subjects in period 1
    		Sodium Oxybate
    Started
    25
    Completed
    25

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Sodium Oxybate
    Reporting group description
    		 Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral

    Reporting group values
    		 Sodium Oxybate Total
    Number of subjects
    		 25 25
    Age Categorical
    Units: Subjects
        Adolescents (12-17 years)
    		 1 1
        Adults (18-64 years)
    		 23 23
        From 65-84 years
    		 1 1
    Age Continuous
    Units:
        arithmetic mean (standard deviation)
    		 38.99 ± 13.94 -
    Gender Categorical
    Units: Subjects
        Male
    		 13 13
        Female
    		 12 12

    End points

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    End points reporting groups
    Reporting group title
    Sodium Oxybate
    Reporting group description
    		 Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral

    Primary: The insulin-like growth factor 1 (IGF-1) measured in fasting conditions at Baseline (Visit 2)

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    End point title
    		 The insulin-like growth factor 1 (IGF-1) measured in fasting conditions at Baseline (Visit 2) [1]
    End point description
    An assay of IGF-1 was done from blood sampled about 10 hours after bedtime on Visit 2.
    End point type
    Primary
    End point timeframe
    Baseline (Visit 2) - approximately 1 day
    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: No confirmatory statistical hypothesis testing was planned for this study.
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: ng/mL
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    207.4 ± 86.5
    No statistical analyses for this end point

    Primary: The insulin-like growth factor 1 (IGF-1) measured in fasting conditions after 1 month of treatment (Visit 3)

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    End point title
    		 The insulin-like growth factor 1 (IGF-1) measured in fasting conditions after 1 month of treatment (Visit 3) [2]
    End point description
    An assay of IGF-1 was done from blood sampled about 10 hours postdose on Visit 3.
    End point type
    Primary
    End point timeframe
    After 1 month of treatment (Visit 3)
    Notes
    [2] - 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: No confirmatory statistical hypothesis testing was planned for this study.
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: ng/mL
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    200.4 ± 77.7
    No statistical analyses for this end point

    Primary: The insulin-like growth factor 1 (IGF-1) measured in fasting conditions after 3 months of treatment (Visit 4)

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    End point title
    		 The insulin-like growth factor 1 (IGF-1) measured in fasting conditions after 3 months of treatment (Visit 4) [3]
    End point description
    An assay of IGF-1 was done from blood sampled about 10 hours postdose on Visit 4.
    End point type
    Primary
    End point timeframe
    After 3 months of treatment (Visit 4)
    Notes
    [3] - 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: No confirmatory statistical hypothesis testing was planned for this study.
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: ng/mL
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    210.2 ± 90.3
    No statistical analyses for this end point

    Secondary: The circadian rhythm of the growth hormone (GH) measured at Baseline (Visit 2)

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    End point title
    		 The circadian rhythm of the growth hormone (GH) measured at Baseline (Visit 2)
    End point description
    Blood was sampled at Baseline (Visit 2) at bedtime 10:00 pm and 1, 2, 4, 8, 12, 16, and 20 hours after bedtime for assaying GH.
    End point type
    Secondary
    End point timeframe
    Baseline (Visit 2) - approximately 1 day
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: ng/mL
    arithmetic mean (standard deviation)
        Bed time
    1.821 ± 4.844
        1h
    1.225 ± 2.895
        2h
    1.408 ± 1.656
        4h
    1.236 ± 1.904
        8h
    0.576 ± 0.632
        12h
    0.293 ± 0.31
        16h
    0.309 ± 0.282
        20h
    0.313 ± 0.362
    No statistical analyses for this end point

    Secondary: The circadian rhythm of the growth hormone (GH) measured at Visit 3

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    End point title
    		 The circadian rhythm of the growth hormone (GH) measured at Visit 3
    End point description
    Blood was sampled predose and 1, 2, 4, 8, 12, 16, and 20 hours postdose at Visit 3 for assaying GH.
    End point type
    Secondary
    End point timeframe
    Visit 3 (approximately 1 month)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: ng/mL
    arithmetic mean (standard deviation)
        Pre-dose
    2.432 ± 7.118
        1h
    5.914 ± 9.743
        2h
    5.238 ± 8.557
        4h
    3.018 ± 4.332
        8h
    0.256 ± 0.326
        12h
    0.308 ± 0.664
        16h
    0.46 ± 0.767
        20h
    0.306 ± 0.342
    No statistical analyses for this end point

    Secondary: The circadian rhythm of the growth hormone (GH) measured at Visit 4

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    End point title
    		 The circadian rhythm of the growth hormone (GH) measured at Visit 4
    End point description
    Blood was sampled predose and 1, 2, 4, 8, 12, 16, and 20 hours postdose at Visit 4 for assaying GH.
    End point type
    Secondary
    End point timeframe
    Visit 4 (approximately 3 months)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: ng/mL
    arithmetic mean (standard deviation)
        Pre-dose
    0.816 ± 1.372
        1h
    3.804 ± 3.173
        2h
    3.864 ± 5.731
        4h
    2.712 ± 3.178
        8h
    0.224 ± 0.292
        12h
    0.42 ± 1.355
        16h
    0.296 ± 0.491
        20h
    0.244 ± 0.39
    No statistical analyses for this end point

    Secondary: Cortisol measured at Baseline (Visit 2)

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    End point title
    		 Cortisol measured at Baseline (Visit 2)
    End point description
    Blood was sampled at Baseline (Visit 2) at bedtime 10:00 pm and 1, 2, 4, 8, 12, 16, and 20 hours after bedtime for assaying cortisol.
    End point type
    Secondary
    End point timeframe
    Baseline (Visit 2) - approximately 1 day
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: ug/L
    arithmetic mean (standard deviation)
        Bed time
    42.21 ± 28.53
        1h
    27.91 ± 17.09
        2h
    22.8 ± 14.54
        4h
    36.06 ± 28.46
        8h
    115.79 ± 52.33
        12h
    143.39 ± 70.67
        16h
    90.84 ± 34.83
        20h
    68.48 ± 40.77
    No statistical analyses for this end point

    Secondary: Cortisol measured at Visit 3

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    End point title
    		 Cortisol measured at Visit 3
    End point description
    Blood was sampled predose and and 1, 2, 4, 8, 12, 16, and 20 hours postdose at Visit 3 for assaying cortisol.
    End point type
    Secondary
    End point timeframe
    Visit 3 (approximately 1 month)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: ug/L
    arithmetic mean (standard deviation)
        Pre-dose
    35.84 ± 27.83
        1h
    24.75 ± 18.2
        2h
    34.62 ± 31.93
        4h
    44.28 ± 36.64
        8h
    95.61 ± 57.14
        12h
    130.21 ± 61.28
        16h
    77.33 ± 32.93
        20h
    70.49 ± 33.71
    No statistical analyses for this end point

    Secondary: Cortisol measured at Visit 4

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    End point title
    		 Cortisol measured at Visit 4
    End point description
    Blood was sampled predose and 1, 2, 4, 8, 12, 16, and 20 hours postdose at Visit 4 for assaying cortisol.
    End point type
    Secondary
    End point timeframe
    Visit 4 (approximately 3 months)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: ug/L
    arithmetic mean (standard deviation)
        Pre-dose
    34.46 ± 24.85
        1h
    25.61 ± 19.06
        2h
    30.06 ± 24.4
        4h
    45.27 ± 35.65
        8h
    113.84 ± 71.18
        12h
    146.33 ± 64.42
        16h
    92.48 ± 30.94
        20h
    75.55 ± 38.01
    No statistical analyses for this end point

    Secondary: The adrenocorticotropic hormone (ACTH) measured in fasting conditions at Baseline (Visit 2)

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    End point title
    		 The adrenocorticotropic hormone (ACTH) measured in fasting conditions at Baseline (Visit 2)
    End point description
    An assay of ACTH was done from blood sampled about 10 hours after bedtime on Visit 2.
    End point type
    Secondary
    End point timeframe
    Baseline (Visit 2) - approximately 1 day
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: pg/mL
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    65.6 ± 46.7
    No statistical analyses for this end point

    Secondary: The adrenocorticotropic hormone (ACTH) measured in fasting conditions at Visit 3

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    End point title
    		 The adrenocorticotropic hormone (ACTH) measured in fasting conditions at Visit 3
    End point description
    An assay of ACTH was done from blood sampled about 10 hours postdose on Visit 3.
    End point type
    Secondary
    End point timeframe
    Visit 3 (approximately 1 month)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: pg/mL
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    62.8 ± 37.9
    No statistical analyses for this end point

    Secondary: The adrenocorticotropic hormone (ACTH) measured in fasting conditions at Visit 4

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    End point title
    		 The adrenocorticotropic hormone (ACTH) measured in fasting conditions at Visit 4
    End point description
    An assay of ACTH was done from blood sampled about 10 hours postdose on Visit 4.
    End point type
    Secondary
    End point timeframe
    Visit 4 (approximately 3 months)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: pg/mL
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    54.8 ± 27.3
    No statistical analyses for this end point

    Secondary: The dehydroepiandrosterone sulfate (DHEA-S) measured in fasting conditions at Baseline (Visit 2)

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    End point title
    		 The dehydroepiandrosterone sulfate (DHEA-S) measured in fasting conditions at Baseline (Visit 2)
    End point description
    An assay of DHEA-S was done from blood sampled about 10 hours after bedtime on Visit 2.
    End point type
    Secondary
    End point timeframe
    Baseline (Visit 2) - approximately 1 day
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: ug/L
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    1988.2 ± 1283.3
    No statistical analyses for this end point

    Secondary: The dehydroepiandrosterone sulfate (DHEA-S) measured in fasting conditions at Visit 3

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    End point title
    		 The dehydroepiandrosterone sulfate (DHEA-S) measured in fasting conditions at Visit 3
    End point description
    An assay of DHEA-S was done from blood sampled about 10 hours postdose on Visit 3.
    End point type
    Secondary
    End point timeframe
    Visit 3 (approximately 1 month)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: ug/L
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    2200.9 ± 1517.5
    No statistical analyses for this end point

    Secondary: The dehydroepiandrosterone sulfate (DHEA-S) measured in fasting conditions at Visit 4

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    End point title
    		 The dehydroepiandrosterone sulfate (DHEA-S) measured in fasting conditions at Visit 4
    End point description
    An assay of DHEA-S was done from blood sampled about 10 hours postdose on Visit 4.
    End point type
    Secondary
    End point timeframe
    Visit 4 (approximately 3 months)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: ug/L
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    2130.7 ± 1450.4
    No statistical analyses for this end point

    Secondary: The prolactin measured in fasting conditions at Baseline (Visit 2)

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    End point title
    		 The prolactin measured in fasting conditions at Baseline (Visit 2)
    End point description
    An assay of prolactin was done from blood sampled about 10 hours after bedtime on Visit 2.
    End point type
    Secondary
    End point timeframe
    Baseline (Visit 2) - approximately 1 day
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: mUI/L
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    356.8 ± 185.9
    No statistical analyses for this end point

    Secondary: The prolactin measured in fasting conditions at Visit 3

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    End point title
    		 The prolactin measured in fasting conditions at Visit 3
    End point description
    An assay of prolactin was done from blood sampled about 10 hours postdose on Visit 3.
    End point type
    Secondary
    End point timeframe
    Visit 3 (approximately 1 month)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: mUI/L
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    280 ± 162.8
    No statistical analyses for this end point

    Secondary: The prolactin measured in fasting conditions at Visit 4

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    End point title
    		 The prolactin measured in fasting conditions at Visit 4
    End point description
    An assay of prolactin was done from blood sampled about 10 hours postdose on Visit 4.
    End point type
    Secondary
    End point timeframe
    Visit 4 (approximately 3 months)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: mUI/L
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    258.4 ± 113.6
    No statistical analyses for this end point

    Secondary: The thyroid stimulating hormone (TSH) measured in fasting conditions at Baseline (Visit 2)

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    End point title
    		 The thyroid stimulating hormone (TSH) measured in fasting conditions at Baseline (Visit 2)
    End point description
    An assay of TSH was done from blood sampled about 10 hours after bedtime on Visit 2.
    End point type
    Secondary
    End point timeframe
    Baseline (Visit 2) - approximately 1 day
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: uU/mL
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    1.366 ± 0.622
    No statistical analyses for this end point

    Secondary: The thyroid stimulating hormone (TSH) measured in fasting conditions at Visit 3

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    End point title
    		 The thyroid stimulating hormone (TSH) measured in fasting conditions at Visit 3
    End point description
    An assay of TSH was done from blood sampled about 10 hours postdose on Visit 3.
    End point type
    Secondary
    End point timeframe
    Visit 3 (approximately 1 month)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: uU/mL
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    1.292 ± 0.599
    No statistical analyses for this end point

    Secondary: The thyroid stimulating hormone (TSH) measured in fasting conditions at Visit 4

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    End point title
    		 The thyroid stimulating hormone (TSH) measured in fasting conditions at Visit 4
    End point description
    An assay of TSH was done from blood sampled about 10 hours postdose on Visit 4.
    End point type
    Secondary
    End point timeframe
    Visit 4 (approximately 3 months)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: uU/mL
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    1.425 ± 0.818
    No statistical analyses for this end point

    Secondary: The total thyroxin (T4) measured in fasting conditions at Baseline (Visit 2)

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    End point title
    		 The total thyroxin (T4) measured in fasting conditions at Baseline (Visit 2)
    End point description
    An assay of T4 was done from blood sampled about 10 hours after bedtime on Visit 2.
    End point type
    Secondary
    End point timeframe
    Baseline (Visit 2) - approximately 1 day
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: pg/mL
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    11.34 ± 1.68
    No statistical analyses for this end point

    Secondary: The total thyroxin (T4) measured in fasting conditions at Visit 3

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    End point title
    		 The total thyroxin (T4) measured in fasting conditions at Visit 3
    End point description
    An assay of T4 was done from blood sampled about 10 hours postdose on Visit 3.
    End point type
    Secondary
    End point timeframe
    Visit 3 (approximately 1 month)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: pg/mL
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    11.47 ± 2.1
    No statistical analyses for this end point

    Secondary: The total thyroxin (T4) measured in fasting conditions at Visit 4

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    End point title
    		 The total thyroxin (T4) measured in fasting conditions at Visit 4
    End point description
    An assay of T4 was done from blood sampled about 10 hours postdose on Visit 4.
    End point type
    Secondary
    End point timeframe
    Visit 4 (approximately 3 months)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: pg/mL
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    11.65 ± 1.78
    No statistical analyses for this end point

    Secondary: The osmolality measured in fasting conditions at Baseline (Visit 2)

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    End point title
    		 The osmolality measured in fasting conditions at Baseline (Visit 2)
    End point description
    An assay of osmolality was done from blood sampled about 10 hours after bedtime on Visit 2.
    End point type
    Secondary
    End point timeframe
    Baseline (Visit 2) - approximately 1 day
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: mosm/kg
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    291.6 ± 4.5
    No statistical analyses for this end point

    Secondary: The osmolality measured in fasting conditions at Visit 3

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    End point title
    		 The osmolality measured in fasting conditions at Visit 3
    End point description
    An assay of osmolality was done from blood sampled about 10 hours postdose on Visit 3.
    End point type
    Secondary
    End point timeframe
    Visit 3 (approximately 1 month)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: mosm/kg
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    292.2 ± 4.9
    No statistical analyses for this end point

    Secondary: The osmolality measured in fasting conditions at Visit 4

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    End point title
    		 The osmolality measured in fasting conditions at Visit 4
    End point description
    An assay of osmolality was done from blood sampled about 10 hours postdose on Visit 4.
    End point type
    Secondary
    End point timeframe
    Visit 4 (approximately 3 months)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: mosm/kg
    arithmetic mean (standard deviation)
        arithmetic mean (standard deviation)
    292.1 ± 5.5
    No statistical analyses for this end point

    Secondary: Electrolytes (Na, K, Ca, P) measured in fasting conditions at Baseline (Visit 2)

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    End point title
    		 Electrolytes (Na, K, Ca, P) measured in fasting conditions at Baseline (Visit 2)
    End point description
    An assay of electrolytes (Na, K, Ca, P) was done from blood sampled about 10 hours after bedtime on Visit 2.
    End point type
    Secondary
    End point timeframe
    Baseline (Visit 2) - approximately 1 day
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: mmol/L and mg/L
    arithmetic mean (standard deviation)
        Na (Sodium in mmol/L)
    141.4 ± 1.9
        K (Potassium in mmol/L)
    3.88 ± 0.21
        Ca (Calcium in mmol/L)
    2.257 ± 0.084
        P (Phosphate in mg/L)
    34.4 ± 6
    No statistical analyses for this end point

    Secondary: Electrolytes (Na, K, Ca, P) measured in fasting conditions at Visit 3

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    End point title
    		 Electrolytes (Na, K, Ca, P) measured in fasting conditions at Visit 3
    End point description
    An assay of electrolytes (Na, K, Ca, P) was done from blood sampled about 10 hours postdose on Visit 3.
    End point type
    Secondary
    End point timeframe
    Visit 3 (approximately 1 month)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: mmol/L and mg/L
    arithmetic mean (standard deviation)
        Na (Sodium in mmol/L)
    141.9 ± 2.1
        K (Potassium in mmol/L)
    3.9 ± 0.25
        Ca (Calcium in mmol/L)
    2.334 ± 0.071
        P (Phosphate in mg/L)
    34.2 ± 4.5
    No statistical analyses for this end point

    Secondary: Electrolytes (Na, K, Ca, P) measured in fasting conditions at Visit 4

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    End point title
    		 Electrolytes (Na, K, Ca, P) measured in fasting conditions at Visit 4
    End point description
    An assay of electrolytes (Na, K, Ca, P) was done from blood sampled about 10 hours postdose on Visit 4.
    End point type
    Secondary
    End point timeframe
    Visit 4 (approximately 3 months)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: mmol/L and mg/L
    arithmetic mean (standard deviation)
        Na (Sodium in mmol/L)
    141.2 ± 1.9
        K (Potassium in mmol/L)
    3.8 ± 0.27
        Ca (Calcium in mmol/L)
    2.29 ± 0.063
        P (Phosphate in mg/L)
    35 ± 3.5
    No statistical analyses for this end point

    Secondary: The number of patients reporting at least one Adverse Event (AE) during the course of the study

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    End point title
    		 The number of patients reporting at least one Adverse Event (AE) during the course of the study
    End point description
    An AE was classified as a treatment-emergent AE (TEAE) if its onset date and time was on or after the first study drug administration. Number of subjects with at least one TEAE is reported below.
    End point type
    Secondary
    End point timeframe
    Visit 1 through the end of the study (approximately 4 months)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: Participants
        Number of subjects
    20
    No statistical analyses for this end point

    Secondary: The number of patient withdrawal due to Adverse Events (AEs) during the course of the study

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    End point title
    		 The number of patient withdrawal due to Adverse Events (AEs) during the course of the study
    End point description
    An AE was classified as a treatment-emergent AE (TEAE) if its onset date and time was on or after the first study drug administration. Number of subjects with TEAE that led to temporarily discontinuation of study drug is reported below.
    End point type
    Secondary
    End point timeframe
    Visit 1 through the end of the study (approximately 4 months)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: Participants
        Number of subjects
    1
    No statistical analyses for this end point

    Secondary: The number of patients reporting at least one Serious Adverse Event (SAE) during the course of the study

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    End point title
    		 The number of patients reporting at least one Serious Adverse Event (SAE) during the course of the study
    End point description
    A Serious Adverse Event is any untoward medical occurrence that at any dose • results in death, • is life threatening, • requires in-patient hospitalization or prolongation of existing hospitalization, • results in persistent or significant disability/incapacity • is a congenital anomaly/birth defect
    End point type
    Secondary
    End point timeframe
    Visit 1 through the end of the study (approximately 4 months)
    End point values
    		 Sodium Oxybate
    Number of subjects analysed
    25
    Units: Participants
        Number of subjects
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Events were collected from Visit 1 through the end of the study (approximately 4 months).
    Adverse event reporting additional description
    Adverse Events refer to the Safety Population which is identical to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    9.0
    Reporting groups
    Reporting group title
    Sodium Oxybate
    Reporting group description
    		 Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral

    Serious adverse events
    		 Sodium Oxybate
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 25 (4.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Gastrointestinal disorders
    Rectal haemorrhage
         subjects affected / exposed
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Sodium Oxybate
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 25 (60.00%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    6 / 25 (24.00%)
         occurrences all number
    8
    Dizziness
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    2
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    5 / 25 (20.00%)
         occurrences all number
    7
    Abdominal pain upper
         subjects affected / exposed
    3 / 25 (12.00%)
         occurrences all number
    7
    Diarrhoea
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    3
    Skin and subcutaneous tissue disorders
    Hyperhidrosis
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    2
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    6
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    2
    Osteoarthritis
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    4

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    24 Jul 2007
    The study protocol was amended on 24-Jul-2007 because of supply chain issues with the medications of the named patient program. The Discharge Visit, normally foreseen at the end of the last confinement period (Visit 4), was postponed for the subjects having benefited from the Xyrem treatment, to allow them to remain on medication. According to protocol, assays of IGF-1, ACTH, DHEA-S, prolactin, TSH, T4, and electrolytes at Baseline, Month 1 and Month 3 Visits were to be performed from blood sampled about 8 hours after bedtime dose (around 6:00 am). In the study, blood was sampled around 8:00 am (about 10 hours after bedtime dose).

    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
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