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    Clinical Trial Results:
    Pilot study of the effects of desipramine on neurovegetative parameters in children with Rett's syndrome

    Summary
    EudraCT number
    		 2007-006739-30
    Trial protocol
    		 FR  
    Global end of trial date
    11 Aug 2014

    Results information
    Results version number
    		 v1(current)
    This version publication date
    11 Aug 2018
    First version publication date
    11 Aug 2018
    Other versions
    Summary report(s)
    		 Summary

    Trial information

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    Trial identification
    Sponsor protocol code
    2007-37
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT00990691
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    ASSISTANCE PUBLIQUE DES HOPITAUX DE MARSEILLE
    Sponsor organisation address
    DRCI, 80 RUE BROCHIER, MARSEILLE, France, 13354
    Public contact
    Me GARRIDO PRADALIE, DIRECTION DE LA RECHERCHE CLINIQUE ET DE L'INNOVATION, +33 491382870, drci@ap-hm.fr
    Scientific contact
    Pr Josette MANCINI, Service de Santé Publique Information médicale, Hôpital de la Timone, 147 Bd baille, 13005 M, +33 491382870, alexandra.giuliani@ap-hm.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 Aug 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    11 Aug 2014
    Global end of trial reached?
    Yes
    Global end of trial date
    11 Aug 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of the study is to study the efficacy of desipramine on cardiorespiratory variability and tolerance of desipramine in 36 patients with Rett Syndrome aged 4 to 18 years weighing up to 60 kg and presenting respiratory rate disorders
    Protection of trial subjects
    no protection was needed
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    17 Feb 2009
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy, Scientific research
    Long term follow-up duration
    		 7 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 36
    Worldwide total number of subjects
    36
    EEA total number of subjects
    36
    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)
    24
    Adolescents (12-17 years)
    12
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The patients are recruited in 5 French centers . It is a randomized, double-blind, placebo-controlled study in 3 parallel groups in subjects with Rett Syndrome (high dose of Desipramine, low dose and placebo). Patient with Rett Syndrome diagnosed clinically and genotyped MECP2 (Xq28), aged 6 to 17 and weighing up to 60 kg

    Pre-assignment
    Screening details
    		 The screening visit verifies that the patient meets the inclusion criteria. the subject and the holders of parental authority or guardianship give their consent for written participation after presentation by the investigator of the study, reading of the informed consent form and response of the investigator to his questions.

    Period 1
    Period 1 title
    baseline
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Carer, Assessor
    Blinding implementation details
    The randomization will be balanced by block of 12 patients and stratified per center in order to maintain sufficient power when a center effect is highlighted. The sealed decoding envelopes will be kept at the pharmacy of each site and in the investigative file of each site. The randomization list will be kept in a sealed envelope in the UPCET biometrics department. The code will only be opened after a process of blind review of the data and freezing of the database.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 High desipramine
    Arm description
    		 Daily single oral dose of "strong" dose desipramine. The dose delivered to each patient will depend on their initial weight (15-25kg: 50mg, 26-35kg: 75mg, 36-45kg: 100mg,> 46kg: 150mg)
    Arm type
    		 Experimental

    Investigational medicinal product name
    DESIPRAMINE
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Cachet
    Routes of administration
    Oral use
    Dosage and administration details
    Weight of patient 15-25 kg 26-35 kg 36-45 kg >46kg Groupe 1 50 mg 75 mg 100 mg 150 mg Groupe 2 25 mg 50 mg 75 mg 100 mg Groupe 3 placebo placebo placebo placebo

    Arm title
    		 Low desipramine
    Arm description
    		 Daily single oral intake of low dose desipramine. The dose delivered to each patient will depend on their initial weight (15-25kg: 25mg, 26-35kg: 50mg, 36-45kg: 75mg,> 46kg: 100mg)
    Arm type
    		 Experimental

    Investigational medicinal product name
    DESIPRAMINE
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Cachet
    Routes of administration
    Oral use
    Dosage and administration details
    Weight of patient 15-25 kg 26-35 kg 36-45 kg >46kg 25 mg 50 mg 75 mg 100 mg

    Arm title
    		 placebo
    Arm description
    		 Daily single oral intake of placebo.
    Arm type
    		 Placebo

    Investigational medicinal product name
    lactose
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Pillules
    Routes of administration
    Oral use
    Dosage and administration details
    25 mg

    Number of subjects in period 1
    		High desipramine Low desipramine placebo
    Started
    12
    12
    12
    Completed
    11
    12
    11
    Not completed
    1
    0
    1
         Consent withdrawn by subject
    1
    -
    1
    Period 2
    Period 2 title
    TREATMENT PERIOD
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Carer, Assessor
    Blinding implementation details
    The randomization will be balanced by block of 12 patients and stratified per center in order to maintain sufficient power when a center effect is highlighted. The sealed decoding envelopes will be kept at the pharmacy of each site and in the investigative file of each site. The randomization list will be kept in a sealed envelope in the UPCET biometrics department. The code will only be opened after a process of blind review of the data and freezing of the database.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 High desipramine
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    DESIPRAMINE
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Cachet
    Routes of administration
    Oral use
    Dosage and administration details
    Weight of patient 15-25 kg 26-35 kg 36-45 kg >46kg Groupe 1 50 mg 75 mg 100 mg 150 mg Groupe 2 25 mg 50 mg 75 mg 100 mg Groupe 3 placebo placebo placebo placebo

    Arm title
    		 Low desipramine
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    DESIPRAMINE
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Pillules
    Routes of administration
    Oral use
    Dosage and administration details
    Weight of patient 15-25 kg 26-35 kg 36-45 kg >46kg 25 mg 50 mg 75 mg 100 mg

    Arm title
    		 placebo
    Arm description
    		 -
    Arm type
    		 Placebo

    Investigational medicinal product name
    lactose
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Cachet
    Routes of administration
    Oral use
    Dosage and administration details
    Weight of patient 15-25 kg 26-35 kg 36-45 kg >46kg Groupe 1 50 mg 75 mg 100 mg 150 mg Groupe 2 25 mg 50 mg 75 mg 100 mg Groupe 3 placebo placebo placebo placebo

    Number of subjects in period 2
    		High desipramine Low desipramine placebo
    Started
    11
    12
    11
    Completed
    7
    8
    11
    Not completed
    4
    4
    0
         Adverse event, non-fatal
    3
    4
    -
         Lost to follow-up
    1
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    baseline
    Reporting group description
    		 -

    Reporting group values
    		 baseline Total
    Number of subjects
    		 36 36
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 24 24
        Adolescents (12-17 years)
    		 12 12
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 10 ( 1 ) -
    Gender categorical
    Units: Subjects
        Female
    		 36 36
        Male
    		 0 0

    End points

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    End points reporting groups
    Reporting group title
    High desipramine
    Reporting group description
    		 Daily single oral dose of "strong" dose desipramine. The dose delivered to each patient will depend on their initial weight (15-25kg: 50mg, 26-35kg: 75mg, 36-45kg: 100mg,> 46kg: 150mg)

    Reporting group title
    Low desipramine
    Reporting group description
    		 Daily single oral intake of low dose desipramine. The dose delivered to each patient will depend on their initial weight (15-25kg: 25mg, 26-35kg: 50mg, 36-45kg: 75mg,> 46kg: 100mg)

    Reporting group title
    placebo
    Reporting group description
    		 Daily single oral intake of placebo.
    Reporting group title
    High desipramine
    Reporting group description
    		 -

    Reporting group title
    Low desipramine
    Reporting group description
    		 -

    Reporting group title
    placebo
    Reporting group description
    		 -

    Primary: Apnea-hypopnea index

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    End point title
    		 Apnea-hypopnea index
    End point description
    End point type
    Primary
    End point timeframe
    1h of monitoring
    End point values
    		 High desipramine Low desipramine placebo
    Number of subjects analysed
    7
    8
    11
    Units: number by hour
    7
    25
    11
    Statistical analysis title
    		 respiratory assessment
    Comparison groups
    High desipramine v Low desipramine v placebo
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 [1]
    P-value
    		 < 0.05
    Method
    		 Kruskal-wallis
    Parameter type
    		 Mean difference (net)
    Confidence interval
    Notes
    [1] - Comparison between Desipramine groups and placebo group

    Secondary: plasma concentration of DMI

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    End point title
    		 plasma concentration of DMI
    End point description
    End point type
    Secondary
    End point timeframe
    6 months of treatment
    End point values
    Number of subjects analysed
    Units: ng/mL
        arithmetic mean (standard deviation)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    6 months
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    2
    Reporting groups
    Reporting group title
    low desipramine
    Reporting group description
    		 -

    Reporting group title
    high despiramine
    Reporting group description
    		 -

    Reporting group title
    placebo
    Reporting group description
    		 -

    Serious adverse events
    		 low desipramine high despiramine placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 12 (8.33%)
    1 / 11 (9.09%)
    1 / 11 (9.09%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Investigations
    Electrocardiogram QT prolonged
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Status epilepticus
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Motor dysfunction
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    INSOMNIA
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 3.9%
    Non-serious adverse events
    		 low desipramine high despiramine placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 12 (58.33%)
    9 / 11 (81.82%)
    11 / 11 (100.00%)
    Vascular disorders
    Peripheral coldness
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Cardiac disorders
    ECG QTc interval prolonged
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Nervous system disorders
    EPILEPSY
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    5
    0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Pyrexia
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    0
    dry mouth
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    rectal hemorrhage
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Stomatitis
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Vomiting
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Obstructive airways disorder
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Dermatitis
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Urticaria
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0
    Psychiatric disorders
    ABNORMAL BEHAVIOR
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Affective disorder
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Agitation
         subjects affected / exposed
    0 / 12 (0.00%)
    2 / 11 (18.18%)
    1 / 11 (9.09%)
         occurrences all number
    0
    2
    3
    Mood altered
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 11 (0.00%)
    0 / 11 (0.00%)
         occurrences all number
    1
    0
    0
    Sleep disorder
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    0
    1
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    0 / 12 (0.00%)
    1 / 11 (9.09%)
    0 / 11 (0.00%)
         occurrences all number
    0
    1
    0

    More information

    		 Close Top of page

    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.
    None reported

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/29468173
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