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    Clinical Trial Results:
    Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne Muscular Dystrophy.

    Summary
    EudraCT number
    2015-003195-68
    Trial protocol
    GB  
    Global end of trial date
    04 Mar 2019

    Results information
    Results version number
    v1(current)
    This version publication date
    05 Dec 2021
    First version publication date
    05 Dec 2021
    Other versions
    Summary report(s)
    EJE Testosterone study paper

    Trial information

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    Trial identification
    Sponsor protocol code
    2015-003195-68
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02571205
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Newcastle Upon Tyne Hospitals Foundation Trust
    Sponsor organisation address
    Queen Victoria Road, Newcastle Upon Tyne, United Kingdom,
    Public contact
    Prof Volker Straub, Newcastle University, +44 01912418663, volker.straub@ncl.ac.uk
    Scientific contact
    Prof Volker Straub, Newcastle University, +44 01912418663, volker.straub@ncl.ac.uk
    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
    30 Sep 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    04 Mar 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Mar 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Evaluate patient satisfaction in response to testosterone replacement therapy in DMD patients with pubertal delay.
    Protection of trial subjects
    All data was reviewed regularly by the data monitoring committee as well as the trial steering group. The CTU also performed regular audits.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    01 Oct 2015
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 15
    Worldwide total number of subjects
    15
    EEA total number of subjects
    15
    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)
    15
    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
    15 patients recruited, aged between 12 and 16.9 years of age

    Pre-assignment
    Screening details
    16 patients screened.

    Period 1
    Period 1 title
    overall trial (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Arm title
    Single arm study
    Arm description
    -
    Arm type
    Experimental

    Investigational medicinal product name
    Sustanon
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Emulsion for injection
    Routes of administration
    Injection
    Dosage and administration details
    incremental regimen, 4-weekly injection for 2 years

    Number of subjects in period 1
    Single arm study
    Started
    15
    Completed
    15

    Baseline characteristics

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    End points

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    End points reporting groups
    Reporting group title
    Single arm study
    Reporting group description
    -

    Primary: Score on TSQM at end of study

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    End point title
    Score on TSQM at end of study [1]
    End point description
    End point type
    Primary
    End point timeframe
    2 years
    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 statistical analysis performed- descriptive only
    End point values
    Single arm study
    Number of subjects analysed
    15
    Units: integer
    15
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    study period
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    118.1
    Reporting groups
    Reporting group title
    Main study group
    Reporting group description
    -

    Serious adverse events
    Main study group
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 15 (33.33%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Diarrhoea infectious
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    lower limb fracture
         subjects affected / exposed
    2 / 15 (13.33%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    planned Achilles tendon release
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 1%
    Non-serious adverse events
    Main study group
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 15 (100.00%)
    Injury, poisoning and procedural complications
    Choking sensation
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 15 (13.33%)
         occurrences all number
    2
    Blood and lymphatic system disorders
    Epistaxis
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Immune system disorders
    Rhinitis allergic
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Dehydration
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Constipation
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    gallstones
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Abdominal pain
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 15 (26.67%)
         occurrences all number
    7
    Lower respiratory tract infection
         subjects affected / exposed
    3 / 15 (20.00%)
         occurrences all number
    4
    Skin and subcutaneous tissue disorders
    Injection related reaction
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Acne
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Chillblains
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Pilonidal cyst
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Psoriasis
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Rash
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Haemorrhoids
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Dry skin
         subjects affected / exposed
    2 / 15 (13.33%)
         occurrences all number
    2
    Fungal infection
         subjects affected / exposed
    2 / 15 (13.33%)
         occurrences all number
    2
    portacath fitting
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Renal and urinary disorders
    Bladder dysfunction
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Acute kidney injury
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Urinary tract infection
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    vertebral fracture
         subjects affected / exposed
    5 / 15 (33.33%)
         occurrences all number
    6
    Lower limb fracture
         subjects affected / exposed
    3 / 15 (20.00%)
         occurrences all number
    6
    reposition femoral nail
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Back pain
         subjects affected / exposed
    4 / 15 (26.67%)
         occurrences all number
    4
    Muscle spasms
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Upper limb fracture
         subjects affected / exposed
    2 / 15 (13.33%)
         occurrences all number
    2
    Fall
         subjects affected / exposed
    2 / 15 (13.33%)
         occurrences all number
    6
    ankle injury
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    foot injury
         subjects affected / exposed
    3 / 15 (20.00%)
         occurrences all number
    3
    bilateral achilles tendon release
         subjects affected / exposed
    2 / 15 (13.33%)
         occurrences all number
    2
    Infections and infestations
    Pyrexia
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    2
    Tonsillitis streptococcal
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Otitis media
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1
    Vomiting
         subjects affected / exposed
    1 / 15 (6.67%)
         occurrences all number
    1

    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.
    None reported
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    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.

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