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    Clinical Trial Results:
    Phase II, Randomised, Double-blind, Dose-ranging Study in Children and Young People to determine the Optimal Dose of Botulinum Toxin Type-A (Dysport®) in Managing the Symptoms of Hip Muscle Spasticity due to Cerebral Palsy.

    Summary
    EudraCT number
    2005-001794-10
    Trial protocol
    GB  
    Global end of trial date
    17 Mar 2008

    Results information
    Results version number
    v1(current)
    This version publication date
    22 Apr 2017
    First version publication date
    22 Apr 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    Y-97-52120-727
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT00455637
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Ipsen Ltd.
    Sponsor organisation address
    Bath Road, Slough, United Kingdom,
    Public contact
    Medical Director, Ipsen Ltd., clinical.trials@ipsen.com
    Scientific contact
    Medical Director, Ipsen Ltd., clinical.trials@ipsen.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
    17 Mar 2008
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    17 Mar 2008
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Mar 2008
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To compare the effectiveness of 3 doses of Dysport® (5, 10 or 15 units per kilogram [kg] per hip) in the management of chronic bilateral hip pain due to Cerebral Palsy in children/young people.
    Protection of trial subjects
    The clinical study was conducted in accordance with the International Conference on Harmonisation Consolidated Guideline on Good Clinical Practice, under the ethical principles laid down in the Declaration of Helsinki. In addition, this clinical study adhered to all local regulatory requirements.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    16 Apr 2007
    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: 6
    Worldwide total number of subjects
    6
    EEA total number of subjects
    6
    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)
    2
    Adolescents (12-17 years)
    4
    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 trial was conducted at 1 centre in the United Kingdom. A recommendation from the ethics committee to allow only patients with bilateral hip pain to be included significantly reduced the original patient pool. A total of 6 eligible children/young people were enrolled. The study was prematurely terminated with only 6 patients recruited.

    Pre-assignment
    Screening details
    A screening visit was performed 4 weeks before study entry. All 6 screened subjects were enrolled in the study.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Dysport® 5 units/kg/hip
    Arm description
    Dysport® was administered at 5 units/kg/hip up to a maximum total dose of 1000 units in a single treatment. Only patients with bilateral hip pain were entered onto the study, therefore the total dosage was divided between both hips. Patients had follow-up visits at 4, 12 and 16 (and if applicable 20) weeks post injection. Patients completed the study at Week 16 if they had clinically significant (CS) pain. If the patient did not have CS pain at Week 16, the patient continued in the study for a further 4 weeks and completed the study at Week 20.
    Arm type
    Experimental

    Investigational medicinal product name
    Dysport®
    Investigational medicinal product code
    Other name
    Clostridium botulinum type A toxin haemagglutinin complex, Abobotulinum toxin A
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Intramuscular use
    Dosage and administration details
    Dysport® was presented as a white lyophilised powder for reconstitution, containing 500 units (nominal) Clostridium botulinum type A toxin haemagglutinin complex together with 125 micrograms (mcg) of human serum albumin and 2.5 milligrams (mg) of lactose in a glass vial. Once reconstituted, the study medication was administered by intramuscular injection into the Adductor Magnus, Iliopsoas and the Medial Hamstring or Rectus Femorus group.

    Arm title
    Dysport® 10 units/kg/hip
    Arm description
    Dysport® was administered at 10 units/kg/hip, up to a maximum total dose of 1000 units in a single treatment. Only patients with bilateral hip pain were entered onto the study, therefore the total dosage was divided between both hips. Patients had follow-up visits at 4, 12 and 16 (and if applicable 20) weeks post injection. Patients completed the study at Week 16 if they had CS pain. If the patient did not have CS pain at Week 16, the patient continued in the study for a further 4 weeks and completed the study at Week 20.
    Arm type
    Experimental

    Investigational medicinal product name
    Dysport®
    Investigational medicinal product code
    Other name
    Clostridium botulinum type A toxin haemagglutinin complex, Abobotulinum toxin A
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Intramuscular use
    Dosage and administration details
    Dysport® was presented as a white lyophilised powder for reconstitution, containing 500 units (nominal) Clostridium botulinum type A toxin haemagglutinin complex together with 125 mcg of human serum albumin and 2.5 mg of lactose in a glass vial. Once reconstituted, the study medication was administered by intramuscular injection into the Adductor Magnus, Iliopsoas and the Medial Hamstring or Rectus Femorus group.

    Arm title
    Dysport® 15 units/kg/hip
    Arm description
    Dysport® was administered at 15 units/kg/hip, up to a maximum total dose of 1000 units in a single treatment. Only patients with bilateral hip pain were entered onto the study, therefore the total dosage was divided between both hips. Patients had follow-up visits at 4, 12 and 16 (and if applicable 20) weeks post injection. Patients completed the study at Week 16 if they had CS pain. If the patient did not have CS pain at Week 16, the patient continued in the study for a further 4 weeks and completed the study at Week 20.
    Arm type
    Experimental

    Investigational medicinal product name
    Dysport®
    Investigational medicinal product code
    Other name
    Clostridium botulinum type A toxin haemagglutinin complex, Abobotulinum toxin A
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Intramuscular use
    Dosage and administration details
    Dysport® was presented as a white lyophilised powder for reconstitution, containing 500 units (nominal) Clostridium botulinum type A toxin haemagglutinin complex together with 125 mcg of human serum albumin and 2.5 mg of lactose in a glass vial. Once reconstituted, the study medication was administered by intramuscular injection into the Adductor Magnus, Iliopsoas and the Medial Hamstring or Rectus Femorus group.

    Number of subjects in period 1
    Dysport® 5 units/kg/hip Dysport® 10 units/kg/hip Dysport® 15 units/kg/hip
    Started
    2
    2
    2
    Completed
    2
    2
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Dysport® 5 units/kg/hip
    Reporting group description
    Dysport® was administered at 5 units/kg/hip up to a maximum total dose of 1000 units in a single treatment. Only patients with bilateral hip pain were entered onto the study, therefore the total dosage was divided between both hips. Patients had follow-up visits at 4, 12 and 16 (and if applicable 20) weeks post injection. Patients completed the study at Week 16 if they had clinically significant (CS) pain. If the patient did not have CS pain at Week 16, the patient continued in the study for a further 4 weeks and completed the study at Week 20.

    Reporting group title
    Dysport® 10 units/kg/hip
    Reporting group description
    Dysport® was administered at 10 units/kg/hip, up to a maximum total dose of 1000 units in a single treatment. Only patients with bilateral hip pain were entered onto the study, therefore the total dosage was divided between both hips. Patients had follow-up visits at 4, 12 and 16 (and if applicable 20) weeks post injection. Patients completed the study at Week 16 if they had CS pain. If the patient did not have CS pain at Week 16, the patient continued in the study for a further 4 weeks and completed the study at Week 20.

    Reporting group title
    Dysport® 15 units/kg/hip
    Reporting group description
    Dysport® was administered at 15 units/kg/hip, up to a maximum total dose of 1000 units in a single treatment. Only patients with bilateral hip pain were entered onto the study, therefore the total dosage was divided between both hips. Patients had follow-up visits at 4, 12 and 16 (and if applicable 20) weeks post injection. Patients completed the study at Week 16 if they had CS pain. If the patient did not have CS pain at Week 16, the patient continued in the study for a further 4 weeks and completed the study at Week 20.

    Reporting group values
    Dysport® 5 units/kg/hip Dysport® 10 units/kg/hip Dysport® 15 units/kg/hip Total
    Number of subjects
    2 2 2 6
    Age Categorical
    Units: Subjects
        In utero
    0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0 0
        Newborns (0-27 days)
    0 0 0 0
        Infants and toddlers (28 days-23 months)
    0 0 0 0
        Children (2-11 years)
    0 1 1 2
        Adolescents (12-17 years)
    2 1 1 4
        Adults (18-64 years)
    0 0 0 0
        From 65-84 years
    0 0 0 0
        85 years and over
    0 0 0 0
    Gender Categorical
    Units: Subjects
        Female
    0 2 0 2
        Male
    2 0 2 4

    End points

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    End points reporting groups
    Reporting group title
    Dysport® 5 units/kg/hip
    Reporting group description
    Dysport® was administered at 5 units/kg/hip up to a maximum total dose of 1000 units in a single treatment. Only patients with bilateral hip pain were entered onto the study, therefore the total dosage was divided between both hips. Patients had follow-up visits at 4, 12 and 16 (and if applicable 20) weeks post injection. Patients completed the study at Week 16 if they had clinically significant (CS) pain. If the patient did not have CS pain at Week 16, the patient continued in the study for a further 4 weeks and completed the study at Week 20.

    Reporting group title
    Dysport® 10 units/kg/hip
    Reporting group description
    Dysport® was administered at 10 units/kg/hip, up to a maximum total dose of 1000 units in a single treatment. Only patients with bilateral hip pain were entered onto the study, therefore the total dosage was divided between both hips. Patients had follow-up visits at 4, 12 and 16 (and if applicable 20) weeks post injection. Patients completed the study at Week 16 if they had CS pain. If the patient did not have CS pain at Week 16, the patient continued in the study for a further 4 weeks and completed the study at Week 20.

    Reporting group title
    Dysport® 15 units/kg/hip
    Reporting group description
    Dysport® was administered at 15 units/kg/hip, up to a maximum total dose of 1000 units in a single treatment. Only patients with bilateral hip pain were entered onto the study, therefore the total dosage was divided between both hips. Patients had follow-up visits at 4, 12 and 16 (and if applicable 20) weeks post injection. Patients completed the study at Week 16 if they had CS pain. If the patient did not have CS pain at Week 16, the patient continued in the study for a further 4 weeks and completed the study at Week 20.

    Primary: Change in Score in the Paediatric Pain Profile (PPP) from Baseline to Week 4.

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    End point title
    Change in Score in the Paediatric Pain Profile (PPP) from Baseline to Week 4. [1]
    End point description
    The PPP completed by the patient’s parent/guardian was assessed by the Investigator at Screening, Baseline and at Weeks 4, 12, 16 and 20. The primary end point was the change in score in the PPP at Week 4 in comparison to Baseline.
    End point type
    Primary
    End point timeframe
    Week 4.
    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.
    End point values
    Dysport® 5 units/kg/hip Dysport® 10 units/kg/hip Dysport® 15 units/kg/hip
    Number of subjects analysed
    0 [2]
    0 [3]
    0 [4]
    Units: Not Applicable
    Notes
    [2] - Due to the small sample of patients, end point data was listed only. No summarised results reported.
    [3] - Due to the small sample of patients, end point data was listed only. No summarised results reported.
    [4] - Due to the small sample of patients, end point data was listed only. No summarised results reported.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to Week 20
    Adverse event reporting additional description
    Adverse events (AEs) were monitored from the time that the patient gave informed consent to the end of the study. Treatment-emergent AEs (TEAEs) are reported.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    11.0
    Reporting groups
    Reporting group title
    Dysport® 5 units/kg/hip
    Reporting group description
    Dysport® was administered at 5 units/kg/hip up to a maximum total dose of 1000 units in a single treatment. Only patients with bilateral hip pain were entered onto the study, therefore the total dosage was divided between both hips. Patients had follow-up visits at 4, 12 and 16 (and if applicable 20) weeks post injection. Patients completed the study at Week 16 if they had CS pain. If the patient did not have CS pain at Week 16, the patient continued in the study for a further 4 weeks and completed the study at Week 20.

    Reporting group title
    Dysport® 15 units/kg/hip
    Reporting group description
    Dysport® was administered at 15 units/kg/hip, up to a maximum total dose of 1000 units in a single treatment. Only patients with bilateral hip pain were entered onto the study, therefore the total dosage was divided between both hips. Patients had follow-up visits at 4, 12 and 16 (and if applicable 20) weeks post injection. Patients completed the study at Week 16 if they had CS pain. If the patient did not have CS pain at Week 16, the patient continued in the study for a further 4 weeks and completed the study at Week 20.

    Reporting group title
    Dysport® 10 units/kg/hip
    Reporting group description
    Dysport® was administered at 10 units/kg/hip, up to a maximum total dose of 1000 units in a single treatment. Only patients with bilateral hip pain were entered onto the study, therefore the total dosage was divided between both hips. Patients had follow-up visits at 4, 12 and 16 (and if applicable 20) weeks post injection. Patients completed the study at Week 16 if they had CS pain. If the patient did not have CS pain at Week 16, the patient continued in the study for a further 4 weeks and completed the study at Week 20.

    Serious adverse events
    Dysport® 5 units/kg/hip Dysport® 15 units/kg/hip Dysport® 10 units/kg/hip
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Dysport® 5 units/kg/hip Dysport® 15 units/kg/hip Dysport® 10 units/kg/hip
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    1 / 2 (50.00%)
    2 / 2 (100.00%)
    1 / 2 (50.00%)
    General disorders and administration site conditions
    Oedema peripheral
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrointestinal disorders
    Haemorrhoids
         subjects affected / exposed
    1 / 2 (50.00%)
    0 / 2 (0.00%)
    0 / 2 (0.00%)
         occurrences all number
    1
    0
    0
    Vomiting
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    Diarrhoea
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    Skin and subcutaneous tissue disorders
    Skin lesion
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 2 (0.00%)
    1 / 2 (50.00%)
         occurrences all number
    0
    0
    1
    Nasopharyngitis
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 2 (50.00%)
    0 / 2 (0.00%)
         occurrences all number
    0
    1
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    27 Jan 2006
    The study was altered to reduce the study to a single treatment with 1 of 3 doses of Dysport® for up to 20 weeks. • The protocol was amended to indicate that a single dose would be given and not a repeat dose. • The evaluation of effectiveness of Dysport® in the management of hip pain was altered to show that change in score in the PPP at all other assessment timepoints and not just the change from baseline to the Week 12 and Week 16 assessments would be assessed. • Assessment of the effect of Dysport® treatment on hip migration percentage as measured on X-ray on completion of the study (Week 28) in comparison to Baseline was removed. • Comparison of the duration of efficacy for the three doses by recording the return of CS pain was added. • The study design was changed to double-blind. • The study duration was changed from 32 to 24 weeks. • It was specified that if the patient was still free from CS pain at Week 16 they would return for a further study visit at Week 20 and that a new diary would be dispensed except at the patient’s final visit (Week 16 or Week 20). • The sleep questionnaire was to be completed at Week 12 in addition to Weeks 4 and 16. • Patients were to complete the study at Week 16. If the patient did not have CS pain at Week 16, the patient was to remain in the study for a further 4 weeks and complete an additional final study visit at Week 20. • Sample size considerations were revised. • A section detailing withdrawal criteria and procedures was added.
    07 Jul 2006
    The Independent Ethics Committee suggested that the study was amended to include patients with bilateral hip pain only. The protocol was altered to reflect these accepted changes. This change significantly reduced the original patient pool.
    24 Nov 2006
    Study was re-classified from Phase IV to Phase II as it fell outside the product license for Dysport® in the UK. This change had no impact on study conduct.

    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.
    Due to the small patient pool, it was difficult to find eligible patients and the study was prematurely terminated with only 6 patients recruited. No analyses of aggregated patient data was performed and only listings were produced for this study.
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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
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