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    Clinical Trial Results:
    A Phase 3, Open-Label, Multi-Center Trial to Evaluate the Long-Term Safety and Efficacy of Repeat Treatments of DaxibotulinumtoxinA for Injection in Adults with Isolated Cervical Dystonia (ASPEN-OLS)

    Summary
    EudraCT number
    		 2018-000447-11
    Trial protocol
    		 GB   AT   PL   IT  
    Global end of trial date
    25 May 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 May 2023
    First version publication date
    04 May 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    1720304
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03617367
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Revance Therapeutics Inc
    Sponsor organisation address
    7555 Gateway Boulevard, Newark, California, United States, 94560
    Public contact
    Regulatory Affairs Manager, Revance Therapeutics Inc, +1 5107423557, jlintao@revance.com
    Scientific contact
    Senior Director, Clinical Development, Revance Therapeutics Inc, +1 5107423400, dvitarella@revance.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
    25 May 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    25 May 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main purpose of this study is to evaluate the long-term safety of multiple continuous treatments of daxibotulinumtoxinA (DAXI) for injection and to assess immunogenicity to botulinum neurotoxin type A (BoNTA) and revance novel excipient (RTP004) after multiple treatments of DAXI for injection.
    Protection of trial subjects
    This study was conducted in accordance with the accepted version of the Declaration of Helsinki, in compliance with International Council for Harmonisation (ICH), Good Clinical Practice (GCP) guidelines, and according to the appropriate regulatory requirements in the countries where the study was conducted.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    05 Sep 2018
    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
    Poland: 65
    Country: Number of subjects enrolled
    Spain: 12
    Country: Number of subjects enrolled
    United Kingdom: 6
    Country: Number of subjects enrolled
    Austria: 1
    Country: Number of subjects enrolled
    Czechia: 26
    Country: Number of subjects enrolled
    France: 8
    Country: Number of subjects enrolled
    Germany: 18
    Country: Number of subjects enrolled
    Canada: 1
    Country: Number of subjects enrolled
    United States: 220
    Worldwide total number of subjects
    357
    EEA total number of subjects
    130
    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)
    240
    From 65 to 84 years
    117
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The study was conducted at 64 sites in 9 countries (Austria, Canada, Czech Republic, France, Germany, Poland, Spain, United Kingdom, and the United States from 05 September 2018 to 25 May 2021. A total of 387 subjects were screened and 357 subjects were enrolled.

    Pre-assignment
    Screening details
    		 A total of 387 subjects were screened, of which 357 subjects were enrolled and treated. Few subjects had multiple movement within the arms in the treatment cycles so, only overall subjects data was planned, analysed and reported to avoid double-counting in disposition and baseline.

    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
    		 DaxibotulinumtoxinA (DAXI)
    Arm description
    		 Subjects received 4 continuous treatments of multi-dose of DAXI intramuscular (IM) injection (125 U, 200 U, 250 U, and 300 U). In cycle 1 subjects received DAXI IM injection (DAXI 125 U or 250 U) based on clinical factors, CD disease severity, and prior BoNT treatment history, using the dose selection criteria on Day 1. In treatment cycles 2 to 4, subjects received same DAXI dose received in cycle 1 (125 U or 250 U), or there was an increase or decrease in the DAXI dose by 1 predefined dose step per subsequent cycle based on the subject’s treatment response in the prior cycle, as determined by the Investigator. The 2 additional DAXI doses for these dose steps were started in cycle 2: DAXI 200 U and DAXI 300 U. If subjects had received retreatment at 12 weeks during each of the first 2 cycles, a 3rd cycle could not have a duration greater than 28 weeks, and if subjects had received 12 weeks of treatment during each of the first 3 cycles, a 4 cycle could not go longer than 16 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    DAXI
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intramuscular use
    Dosage and administration details
    Subjects received 4 multi-dose of DAXI IM injection (125 U, 200 U, 250 U, and 300 U).

    Number of subjects in period 1
    		DaxibotulinumtoxinA (DAXI)
    Started
    357
    Treatment Cycle 1
    357
    Treatment Cycle 2
    329
    Treatment Cycle 3
    234 [1]
    Treatment Cycle 4
    65 [2]
    Completed
    297
    Not completed
    60
         Consent withdrawn by subject
    15
         Adverse Event
    2
         Protocol violation
    6
         Death
    1
         Other
    2
         Lost to follow-up
    6
         Progressive disease
    4
         Lack of efficacy
    24
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: Few subjects had multiple movement within the arms in the treatment cycles.
    [2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: Few subjects had multiple movement within the arms in the treatment cycles.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Study
    Reporting group description
    		 Subjects received 4 continuous treatments of multi-dose of DAXI intramuscular (IM) injection (125 U, 200 U, 250 U, and 300 U). In cycle 1 subjects received DAXI IM injection (DAXI 125 U or 250 U) based on clinical factors, CD disease severity, and prior BoNT treatment history, using the dose selection criteria on Day 1. In treatment cycles 2 to 4, subjects received same DAXI dose received in cycle 1 (125 U or 250 U), or there was an increase or decrease in the DAXI dose by 1 predefined dose step per subsequent cycle based on the subject’s treatment response in the prior cycle, as determined by the Investigator. The 2 additional DAXI doses for these dose steps were started in cycle 2: DAXI 200 U and DAXI 300 U. If subjects had received retreatment at 12 weeks during each of the first 2 cycles, a 3rd cycle could not have a duration greater than 28 weeks, and if subjects had received 12 weeks of treatment during each of the first 3 cycles, a 4 cycle could not go longer than 16 weeks.

    Reporting group values
    		 Overall Study Total
    Number of subjects
    		 357 357
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 57.6 ( 11.86 ) -
    Gender categorical
    Units: Subjects
        Female
    		 238 238
        Male
    		 119 119
    Race
    Units: Subjects
        White
    		 342 342
        Black
    		 6 6
        Asian
    		 4 4
        American Indian or Alaska Native
    		 1 1
        Native Hawaiian or other Pacific Islander
    		 1 1
        Other
    		 3 3
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 19 19
        Not Hispanic or Latino
    		 333 333
        Not provided
    		 5 5

    End points

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    End points reporting groups
    Reporting group title
    DaxibotulinumtoxinA (DAXI)
    Reporting group description
    		 Subjects received 4 continuous treatments of multi-dose of DAXI intramuscular (IM) injection (125 U, 200 U, 250 U, and 300 U). In cycle 1 subjects received DAXI IM injection (DAXI 125 U or 250 U) based on clinical factors, CD disease severity, and prior BoNT treatment history, using the dose selection criteria on Day 1. In treatment cycles 2 to 4, subjects received same DAXI dose received in cycle 1 (125 U or 250 U), or there was an increase or decrease in the DAXI dose by 1 predefined dose step per subsequent cycle based on the subject’s treatment response in the prior cycle, as determined by the Investigator. The 2 additional DAXI doses for these dose steps were started in cycle 2: DAXI 200 U and DAXI 300 U. If subjects had received retreatment at 12 weeks during each of the first 2 cycles, a 3rd cycle could not have a duration greater than 28 weeks, and if subjects had received 12 weeks of treatment during each of the first 3 cycles, a 4 cycle could not go longer than 16 weeks.

    Subject analysis set title
    Treatment Cycle 1: DAXI 125 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 125 U solution, IM injection in treatment cycle 1.

    Subject analysis set title
    Treatment Cycle 2: DAXI 125 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 125 U solution, IM injection in treatment cycle 2.

    Subject analysis set title
    Treatment Cycle 3: DAXI 125 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 125 U solution, IM injection in treatment cycle 3.

    Subject analysis set title
    Treatment Cycle 4: DAXI 125 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 125 U solution, IM injection in treatment cycle 4.

    Subject analysis set title
    Treatment Cycle 2: DAXI 200 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 200 U solution, IM injection in treatment cycle 2.

    Subject analysis set title
    Treatment Cycle 3: DAXI 200 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 200 U solution, IM injection in treatment cycle 3.

    Subject analysis set title
    Treatment Cycle 4: DAXI 200 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 200 U solution, IM injection in treatment cycle 4.

    Subject analysis set title
    Treatment Cycle 1: DAXI 250 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 250 U solution, IM injection in treatment cycle 1.

    Subject analysis set title
    Treatment Cycle 2: DAXI 250 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 250 U solution, IM injection in treatment cycle 2.

    Subject analysis set title
    Treatment Cycle 3: DAXI 250 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 250 U solution, IM injection in treatment cycle 3.

    Subject analysis set title
    Treatment Cycle 4: DAXI 250 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 250 U solution, IM injection in treatment cycle 4.

    Subject analysis set title
    Treatment Cycle 2: DAXI 300 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 300 U solution, IM injection in treatment cycle 2.

    Subject analysis set title
    Treatment Cycle 3: DAXI 300 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 300 U solution, IM injection in treatment cycle 3.

    Subject analysis set title
    Treatment Cycle 4: DAXI 300 U
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects received DAXI 300 U solution, IM injection in treatment cycle 4.

    Subject analysis set title
    Treatment Cycle 1 Total
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Total number of subjects who received treatment (DAXI 125U or DAXI 250U) in Cycle 1.

    Subject analysis set title
    Treatment Cycle 2 Total
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Total number of subjects who received treatment (DAXI 125 U, DAXI 200 U, DAXI 250 U or DAXI 300 U) in Cycle 2.

    Subject analysis set title
    Treatment Cycle 3 Total
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Total number of subjects who received treatment (DAXI 125 U, DAXI 200 U, DAXI 250 U or DAXI 300 U) in cycle 3.

    Subject analysis set title
    Treatment Cycle 4 Total
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Total number of subjects who received treatment (DAXI 125 U, DAXI 200 U, DAXI 250 U or DAXI 300 U) in cycle 4.

    Primary: Number of Subjects with Drug-related Treatment-emergent Adverse Events (TEAEs) and Study Drug Discontinuation due to Drug-related TEAEs

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    End point title
    		 Number of Subjects with Drug-related Treatment-emergent Adverse Events (TEAEs) and Study Drug Discontinuation due to Drug-related TEAEs [1]
    End point description
    An AE was defined as any untoward medical occurrence (e.g., sign, symptom, disease, syndrome, intercurrent illness, clinically significant abnormal laboratory finding, injury, or accident) that emerged or worsened following administration of the study drug; AEs were recorded until the end of study participation. The untoward medical occurrence may not necessarily have had a causal relationship to the administration of the study drug. A TEAE was one that occurred after any exposure to study drug. Number of subjects with drug-related TEAEs and study drug discontinuation due to drug-related TEAEs were reported. Safety population was defined as all enrolled subjects who receive at least one dose of study drug. Here, "number of subjects analysed" refer to the subjects evaluable for this endpoint.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 52
    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 analysed for this endpoint.
    End point values
    		 Treatment Cycle 1 Total Treatment Cycle 2 Total Treatment Cycle 3 Total Treatment Cycle 4 Total
    Number of subjects analysed
    357
    329
    234
    65
    Units: subjects
    number (not applicable)
        Any treatment-related TEAE
    75
    56
    46
    9
        TEAE that led to study drug discontinuation
    1
    0
    2
    0
    No statistical analyses for this end point

    Primary: Number of Subjects who Developed Anti-drug Antibodies (ADAs) to RTP004 by Treatment Cycles and Dose

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    End point title
    		 Number of Subjects who Developed Anti-drug Antibodies (ADAs) to RTP004 by Treatment Cycles and Dose [2]
    End point description
    Subjects who developed ADAs (negative and positive) to analyte RTP004 were reported. Positive ADAs consisted of Treatment-induced, treatment unaffected, and treatment-boosted RTP004 ADAs. The safety population was defined as all enrolled subjects who received at least 1 dose of study drug. Here, "number of subjects analysed" refer to the subjects evaluable for this endpoint.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 52
    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: Only descriptive data was planned to be analysed for this endpoint.
    End point values
    		 Treatment Cycle 1: DAXI 125 U Treatment Cycle 2: DAXI 125 U Treatment Cycle 3: DAXI 125 U Treatment Cycle 4: DAXI 125 U Treatment Cycle 2: DAXI 200 U Treatment Cycle 3: DAXI 200 U Treatment Cycle 4: DAXI 200 U Treatment Cycle 1: DAXI 250 U Treatment Cycle 2: DAXI 250 U Treatment Cycle 3: DAXI 250 U Treatment Cycle 4: DAXI 250 U Treatment Cycle 2: DAXI 300 U Treatment Cycle 3: DAXI 300 U Treatment Cycle 4: DAXI 300 U
    Number of subjects analysed
    109
    43
    16
    1
    68
    29
    10
    234
    103
    87
    15
    102
    98
    38
    Units: subjects
        Treatment-induced ADA
    0
    0
    0
    0
    1
    0
    0
    2
    0
    1
    0
    0
    1
    0
        Treatment-unaffected ADA
    1
    0
    0
    0
    1
    1
    0
    3
    2
    2
    0
    1
    2
    2
        Treatment-boosted ADA
    0
    0
    0
    0
    0
    0
    0
    0
    0
    0
    0
    0
    0
    0
        ADA-negative and treatment-induced negative
    108
    43
    16
    1
    66
    28
    10
    229
    101
    84
    15
    101
    95
    36
    No statistical analyses for this end point

    Primary: Number of Subjects who Developed Anti-drug Antibodies (ADAs) to RTT150 by Treatment Cycles and Dose

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    End point title
    		 Number of Subjects who Developed Anti-drug Antibodies (ADAs) to RTT150 by Treatment Cycles and Dose [3]
    End point description
    Subjects who developed ADAs (negative and positive) to analyte RTT150 were reported. Positive ADAs consisted of Treatment-induced, treatment unaffected, and treatment-boosted RTT150 ADAs. The safety population was defined as all enrolled subjects who received at least 1 dose of study drug. Here, "number of subjects analysed" refer to the subjects evaluable for this endpoint.
    End point type
    Primary
    End point timeframe
    Baseline up to Week 52
    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: Only descriptive data was planned to be analysed for this endpoint.
    End point values
    		 Treatment Cycle 1: DAXI 125 U Treatment Cycle 2: DAXI 125 U Treatment Cycle 3: DAXI 125 U Treatment Cycle 4: DAXI 125 U Treatment Cycle 2: DAXI 200 U Treatment Cycle 3: DAXI 200 U Treatment Cycle 4: DAXI 200 U Treatment Cycle 1: DAXI 250 U Treatment Cycle 2: DAXI 250 U Treatment Cycle 3: DAXI 250 U Treatment Cycle 4: DAXI 250 U Treatment Cycle 2: DAXI 300 U Treatment Cycle 3: DAXI 300 U Treatment Cycle 4: DAXI 300 U
    Number of subjects analysed
    109
    43
    16
    1
    69
    29
    10
    237
    103
    88
    15
    104
    100
    39
    Units: subjects
        Treatment-induced ADA
    1
    0
    0
    0
    1
    0
    0
    0
    1
    1
    1
    0
    0
    0
        Treatment-unaffected ADA
    2
    1
    1
    0
    1
    0
    0
    5
    3
    3
    0
    3
    2
    2
        Treatment-boosted ADA
    0
    0
    0
    0
    0
    0
    0
    1
    1
    1
    0
    0
    0
    0
        ADA-negative and treatment-induced negative
    106
    42
    15
    1
    67
    29
    10
    231
    98
    83
    14
    101
    98
    37
    No statistical analyses for this end point

    Primary: Number of Subjects who Developed Neutralizing Anti-drug Antibodies (ADAs) to RTT150 by Treatment Cycles and Dose

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    End point title
    		 Number of Subjects who Developed Neutralizing Anti-drug Antibodies (ADAs) to RTT150 by Treatment Cycles and Dose [4]
    End point description
    Subjects who developed NAbs (negative and positive) to analyte RTT150 were reported. The safety population was defined as all enrolled subjects who received at least 1 dose of study drug. Here, "number of subjects analysed" refer to the subjects evaluable for this endpoint.
    End point type
    Primary
    End point timeframe
    Treatment Cycle 1: Up to Week 36, Treatment Cycle 2: Up to Week 28, Treatment Cycle 3: Up to Week 16 and Treatment Cycle 4: Up to Week 16
    Notes
    [4] - 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 analysed for this endpoint.
    End point values
    		 Treatment Cycle 1: DAXI 125 U Treatment Cycle 2: DAXI 125 U Treatment Cycle 3: DAXI 125 U Treatment Cycle 4: DAXI 125 U Treatment Cycle 2: DAXI 200 U Treatment Cycle 3: DAXI 200 U Treatment Cycle 4: DAXI 200 U Treatment Cycle 1: DAXI 250 U Treatment Cycle 2: DAXI 250 U Treatment Cycle 3: DAXI 250 U Treatment Cycle 4: DAXI 250 U Treatment Cycle 2: DAXI 300 U Treatment Cycle 3: DAXI 300 U Treatment Cycle 4: DAXI 300 U
    Number of subjects analysed
    0 [5]
    0 [6]
    0 [7]
    0 [8]
    0 [9]
    0 [10]
    0 [11]
    1
    1
    1
    0 [12]
    0 [13]
    0 [14]
    1
    Units: Subjects
        NAb positive
    0
    1
    1
    1
        NAb negative
    1
    0
    0
    0
    Notes
    [5] - No subjects were analysed at this timepoint (Week 36).
    [6] - No subjects were analysed at this timepoint (Week 28).
    [7] - No subjects were analysed at this timepoint (Week 16).
    [8] - No subjects were analysed at this timepoint (Week 16).
    [9] - No subjects were analysed at this timepoint (Week 28).
    [10] - No subjects were analysed at this timepoint (Week 16).
    [11] - No subjects were analysed at this timepoint (Week 16).
    [12] - No subjects were analysed at this timepoint (Week 16).
    [13] - No subjects were analysed at this timepoint (Week 28).
    [14] - No subjects were analysed at this timepoint (Week 16).
    No statistical analyses for this end point

    Secondary: Average Change from Baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score at Weeks 4 and 6 by Treatment Cycles and Dose

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    End point title
    		 Average Change from Baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score at Weeks 4 and 6 by Treatment Cycles and Dose
    End point description
    The TWSTRS was an assessment scale used to measure the impact of CD on subjects and comprises 3 subscales: Severity (0–35), disability (0–30) and pain (0-20), each of which was scored independently. The total score from the 3 subscales gives the TWSTRS total score with a value from 0 to 85 (best to worst). The higher score indicates worst outcomes, and a negative change indicates better outcomes. The safety population was defined as all enrolled subjects who received at least 1 dose of study drug. Here, "number of subjects analysed" refer to the subjects evaluable for this endpoint. Here "99999" refers to data not available and we have added it as space-fillers.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 4 and 6
    End point values
    		 Treatment Cycle 1: DAXI 125 U Treatment Cycle 2: DAXI 125 U Treatment Cycle 3: DAXI 125 U Treatment Cycle 4: DAXI 125 U Treatment Cycle 2: DAXI 200 U Treatment Cycle 3: DAXI 200 U Treatment Cycle 4: DAXI 200 U Treatment Cycle 1: DAXI 250 U Treatment Cycle 2: DAXI 250 U Treatment Cycle 3: DAXI 250 U Treatment Cycle 4: DAXI 250 U Treatment Cycle 2: DAXI 300 U Treatment Cycle 3: DAXI 300 U Treatment Cycle 4: DAXI 300 U
    Number of subjects analysed
    107
    41
    15
    1
    71
    29
    8
    243
    100
    87
    14
    103
    96
    39
    Units: Score on scale
        arithmetic mean (standard deviation)
    -15.0 ( 10.43 )
    -15.6 ( 12.26 )
    -15.0 ( 11.55 )
    -9.7 ( 99999 )
    -19.5 ( 10.34 )
    -19.6 ( 11.07 )
    -13.9 ( 11.31 )
    -15.6 ( 10.26 )
    -19.9 ( 10.78 )
    -18.1 ( 10.99 )
    -21.4 ( 12.63 )
    -15.2 ( 11.67 )
    -17.5 ( 11.71 )
    -20.9 ( 14.31 )
    No statistical analyses for this end point

    Secondary: Duration of Effect (Time to Loss of Efficacy Based on TWSTRS Total Score) by Treatment Cycles and Dose

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    End point title
    		 Duration of Effect (Time to Loss of Efficacy Based on TWSTRS Total Score) by Treatment Cycles and Dose
    End point description
    The duration of effect was defined as the time in weeks after each treatment until loss of at least 80% of the peak treatment effect based on TWSTRS total score (loss of efficacy). The peak treatment effect was defined as the average change from baseline at Weeks 4 and 6 in the TWSTRS total score. Duration of effect is only evaluable in Cycles 1 and 2; due to the 52-week limit on study participation, Cycles 3 and 4 are artificially truncated and therefore not provided valid estimates of duration. The safety population was defined as all enrolled subjects who received at least 1 dose of study drug. Here "99999" refers to data not available and we have added it as space-fillers. Here, "number of subjects analysed" refer to the subjects evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 4 and 6
    End point values
    		 Treatment Cycle 1: DAXI 125 U Treatment Cycle 2: DAXI 125 U Treatment Cycle 2: DAXI 200 U Treatment Cycle 1: DAXI 250 U Treatment Cycle 2: DAXI 250 U Treatment Cycle 2: DAXI 300 U
    Number of subjects analysed
    111
    44
    71
    246
    107
    107
    Units: weeks
        median (confidence interval 95%)
    21.3 (19.3 to 24.4)
    26.0 (20.1 to 32.1)
    21.0 (19.0 to 24.0)
    19.9 (17.1 to 20.9)
    20.1 (17.1 to 28.1)
    20.1 (17.7 to 24.4)
    No statistical analyses for this end point

    Secondary: Percentage of Subjects with at least “moderate” (a 2-point) improvement on Clinical Global Impression of Change (CGIC) at Week 4 or Week 6 of Each Treatment Cycle

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    End point title
    		 Percentage of Subjects with at least “moderate” (a 2-point) improvement on Clinical Global Impression of Change (CGIC) at Week 4 or Week 6 of Each Treatment Cycle
    End point description
    The CGIC was a questionnaire that captures the clinician’s overall impression of the subject’s response to study treatment. The clinician’s selected response maps to a 7-point scale: -3 (very much worse), 0 (about the same), to +3 (very much better). The higher score indicates better outcomes. A 2+ point improvement was defined as a response of moderately better (+2) or very much better (+3) at Week 4 or Week 6. The safety population was defined as all enrolled subjects who received at least 1 dose of study drug. Here, "number of subjects analysed" refer to the subjects evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    At Week 4 or 6
    End point values
    		 Treatment Cycle 1: DAXI 125 U Treatment Cycle 2: DAXI 125 U Treatment Cycle 3: DAXI 125 U Treatment Cycle 4: DAXI 125 U Treatment Cycle 2: DAXI 200 U Treatment Cycle 3: DAXI 200 U Treatment Cycle 4: DAXI 200 U Treatment Cycle 1: DAXI 250 U Treatment Cycle 2: DAXI 250 U Treatment Cycle 3: DAXI 250 U Treatment Cycle 4: DAXI 250 U Treatment Cycle 2: DAXI 300 U Treatment Cycle 3: DAXI 300 U Treatment Cycle 4: DAXI 300 U
    Number of subjects analysed
    109
    42
    15
    1
    71
    29
    8
    244
    103
    87
    14
    104
    96
    39
    Units: Percentage of subjects
        number (not applicable)
    75.7
    79.5
    75.0
    100
    84.5
    90.0
    80.0
    70.7
    86.0
    77.3
    80.0
    76.6
    76.0
    84.6
    No statistical analyses for this end point

    Secondary: Percentage of Subjects with at least “moderate” (a 2-point) improvement on Patient Global Impression of Change (PGIC) at Week 4 or Week 6 of Each Treatment Cycle

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    End point title
    		 Percentage of Subjects with at least “moderate” (a 2-point) improvement on Patient Global Impression of Change (PGIC) at Week 4 or Week 6 of Each Treatment Cycle
    End point description
    The PGIC was a questionnaire that captures the patient’s overall impression of their response to study treatment. The subject’s selected response maps to a 7-point scale: -3 (very much worse), 0 (about the same), to +3 (very much better). A 2+ point improvement was defined as a response of moderately better (+2) or very much better (+3) at Week 4 or Week 6. The safety population was defined as all enrolled subjects who received at least 1 dose of study drug. Here, "number of subjects analysed" refer to the subjects evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    At Week 4 or 6
    End point values
    		 Treatment Cycle 1: DAXI 125 U Treatment Cycle 2: DAXI 125 U Treatment Cycle 3: DAXI 125 U Treatment Cycle 4: DAXI 125 U Treatment Cycle 2: DAXI 200 U Treatment Cycle 3: DAXI 200 U Treatment Cycle 4: DAXI 200 U Treatment Cycle 1: DAXI 250 U Treatment Cycle 2: DAXI 250 U Treatment Cycle 3: DAXI 250 U Treatment Cycle 4: DAXI 250 U Treatment Cycle 2: DAXI 300 U Treatment Cycle 3: DAXI 300 U Treatment Cycle 4: DAXI 300 U
    Number of subjects analysed
    109
    42
    15
    1
    71
    29
    8
    244
    103
    87
    14
    104
    96
    39
    Units: Percentage of subjects
        number (not applicable)
    69.4
    68.2
    43.8
    100
    80.3
    93.3
    60.0
    66.3
    80.4
    68.2
    60.0
    58.9
    57.0
    74.4
    No statistical analyses for this end point

    Secondary: Percent Change from Baseline in Quality of Life (QOL) measured based on the Cervical Dystonia Impact Profile (CDIP-58) at Week 6

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    End point title
    		 Percent Change from Baseline in Quality of Life (QOL) measured based on the Cervical Dystonia Impact Profile (CDIP-58) at Week 6
    End point description
    The CDIP-58 assesses the health impact of CD. The CDIP-58 is composed of eight domains: head and neck (6 items; 6 to 30 points), pain and discomfort (5 items; 5 to 25 points), upper limb activities (9 items; 9 to 45 points), walking (9 items; 9 to 45 points), sleep (4 items; 4 to 20 points), annoyance (8 items; 8 to 40 points), mood (7 items; 7 to 35 points), and psychosocial functioning (10 items; 10 to 50 points). Subscale scores were transformed to a common theoretical range of 0 (no impact) to 100 (most impact). The safety population was defined as all enrolled subjects who received at least 1 dose of study drug. Here, “n= number analysed” signifies to subjects evaluable at given category.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 6
    End point values
    		 Treatment Cycle 1 Total Treatment Cycle 2 Total Treatment Cycle 3 Total Treatment Cycle 4 Total
    Number of subjects analysed
    357
    329
    234
    65
    Units: Percent Change
    arithmetic mean (standard deviation)
        Annoyance (n=309,273,201,56)
    -29.46 ( 98.827 )
    -49.54 ( 57.094 )
    -40.35 ( 68.949 )
    -49.25 ( 54.633 )
        Head and neck symptoms (n=333,292,208,57)
    -36.83 ( 35.884 )
    -46.13 ( 33.539 )
    -40.19 ( 37.735 )
    -48.60 ( 40.805 )
        Mood (n=273,242,179,51)
    -29.09 ( 93.496 )
    -45.73 ( 73.222 )
    -45.42 ( 68.085 )
    -37.11 ( 139.591 )
        Pain and discomfort symptoms (n=326,284,204,55)
    -36.82 ( 49.805 )
    -48.75 ( 48.029 )
    -39.24 ( 46.514 )
    -50.65 ( 37.450 )
        Psychosocial functioning (n=302,268,200,55)
    -35.82 ( 58.520 )
    -48.75 ( 48.029 )
    -41.47 ( 65.214 )
    -47.73 ( 46.804 )
        Sleep (n=264,231,167,53)
    -47.03 ( 78.131 )
    -56.09 ( 52.852 )
    -47.26 ( 59.372 )
    -56.84 ( 55.075 )
        Upper limb activity symptoms (n=313,277,199,56)
    -26.84 ( 69.077 )
    -38.20 ( 62.693 )
    -36.31 ( 57.008 )
    -45.91 ( 43.393 )
        Walking (n=262,231,170,46)
    -35.51 ( 95.256 )
    -36.20 ( 105.067 )
    -25.15 ( 110.519 )
    -16.07 ( 128.252 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to Week 52
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23
    Reporting groups
    Reporting group title
    Treatment Cycle 1 Total
    Reporting group description
    		 Total number of subjects who received treatment (DAXI 125U or DAXI 250U) in Cycle 1.

    Reporting group title
    Treatment Cycle 2 Total
    Reporting group description
    		 Total number of subjects who received treatment (DAXI 125 U, DAXI 200 U, DAXI 250 U or DAXI 300 U) in Cycle 2.

    Reporting group title
    Treatment Cycle 3 Total
    Reporting group description
    		 Total number of subjects who received treatment (DAXI 125 U, DAXI 200 U, DAXI 250 U or DAXI 300 U) in Cycle 3.

    Reporting group title
    Treatment Cycle 4 Total
    Reporting group description
    		 Total number of subjects who received treatment (DAXI 125 U, DAXI 200 U, DAXI 250 U or DAXI 300 U) in Cycle 4.

    Serious adverse events
    		 Treatment Cycle 1 Total Treatment Cycle 2 Total Treatment Cycle 3 Total Treatment Cycle 4 Total
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 357 (0.56%)
    6 / 329 (1.82%)
    8 / 234 (3.42%)
    1 / 65 (1.54%)
         number of deaths (all causes)
    0
    1
    0
    0
         number of deaths resulting from adverse events
    0
    1
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Breast cancer
         subjects affected / exposed
    0 / 357 (0.00%)
    0 / 329 (0.00%)
    1 / 234 (0.43%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Intraductal proliferative breast lesion
         subjects affected / exposed
    0 / 357 (0.00%)
    1 / 329 (0.30%)
    0 / 234 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Squamous cell carcinoma of lung
         subjects affected / exposed
    0 / 357 (0.00%)
    0 / 329 (0.00%)
    1 / 234 (0.43%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Accidental overdose
         subjects affected / exposed
    0 / 357 (0.00%)
    0 / 329 (0.00%)
    0 / 234 (0.00%)
    1 / 65 (1.54%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Fall
         subjects affected / exposed
    0 / 357 (0.00%)
    1 / 329 (0.30%)
    0 / 234 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Head injury
         subjects affected / exposed
    0 / 357 (0.00%)
    0 / 329 (0.00%)
    1 / 234 (0.43%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Intracranial aneurysm
         subjects affected / exposed
    1 / 357 (0.28%)
    0 / 329 (0.00%)
    0 / 234 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Blood loss anaemia
         subjects affected / exposed
    0 / 357 (0.00%)
    1 / 329 (0.30%)
    0 / 234 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Non-cardiac chest pain
         subjects affected / exposed
    1 / 357 (0.28%)
    0 / 329 (0.00%)
    0 / 234 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Hiatus hernia
         subjects affected / exposed
    0 / 357 (0.00%)
    0 / 329 (0.00%)
    1 / 234 (0.43%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary embolism
         subjects affected / exposed
    0 / 357 (0.00%)
    0 / 329 (0.00%)
    1 / 234 (0.43%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    0 / 357 (0.00%)
    1 / 329 (0.30%)
    0 / 234 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Chronic kidney disease
         subjects affected / exposed
    0 / 357 (0.00%)
    1 / 329 (0.30%)
    0 / 234 (0.00%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    0 / 357 (0.00%)
    0 / 329 (0.00%)
    2 / 234 (0.85%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Clostridium difficile colitis
         subjects affected / exposed
    0 / 357 (0.00%)
    0 / 329 (0.00%)
    1 / 234 (0.43%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Diverticulitis
         subjects affected / exposed
    0 / 357 (0.00%)
    1 / 329 (0.30%)
    1 / 234 (0.43%)
    0 / 65 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 3%
    Non-serious adverse events
    		 Treatment Cycle 1 Total Treatment Cycle 2 Total Treatment Cycle 3 Total Treatment Cycle 4 Total
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    63 / 357 (17.65%)
    48 / 329 (14.59%)
    40 / 234 (17.09%)
    10 / 65 (15.38%)
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    5 / 357 (1.40%)
    2 / 329 (0.61%)
    3 / 234 (1.28%)
    2 / 65 (3.08%)
         occurrences all number
    5
    2
    3
    2
    Nervous system disorders
    Headache
         subjects affected / exposed
    11 / 357 (3.08%)
    9 / 329 (2.74%)
    3 / 234 (1.28%)
    1 / 65 (1.54%)
         occurrences all number
    11
    9
    4
    1
    General disorders and administration site conditions
    Injection site erythema
         subjects affected / exposed
    9 / 357 (2.52%)
    6 / 329 (1.82%)
    7 / 234 (2.99%)
    1 / 65 (1.54%)
         occurrences all number
    9
    6
    7
    1
    Injection site pain
         subjects affected / exposed
    19 / 357 (5.32%)
    9 / 329 (2.74%)
    5 / 234 (2.14%)
    2 / 65 (3.08%)
         occurrences all number
    19
    10
    6
    2
    Gastrointestinal disorders
    Dysphagia
         subjects affected / exposed
    15 / 357 (4.20%)
    14 / 329 (4.26%)
    12 / 234 (5.13%)
    2 / 65 (3.08%)
         occurrences all number
    15
    14
    12
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    4 / 357 (1.12%)
    1 / 329 (0.30%)
    0 / 234 (0.00%)
    2 / 65 (3.08%)
         occurrences all number
    4
    1
    0
    2
    Muscular weakness
         subjects affected / exposed
    16 / 357 (4.48%)
    17 / 329 (5.17%)
    15 / 234 (6.41%)
    2 / 65 (3.08%)
         occurrences all number
    16
    18
    15
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Feb 2018
    Protocol Amendment 1 •Modified Protocol title •Changed Medical Monitor •Modified Study design o Removed active comparator and changed dose range to fixed dose o Changed sample size from 600 to 290 subjects o Expanded participating countries to include Europe •Changed total number of sites from 50 to approximately 80 sites •Modified the study objectives •Modified primary, secondary, and exploratory endpoints •Modified screening window and modified screening period from 2 to 3 weeks •Modified schedule of assessments •Modified Patient Global Impression of Change (PGIC) and Clinical Global Impression of Change (CGIC) scales from 9-point to 7-point scales •Updated safety evaluations (added Spirometry and Dysphagia Severity Scale) •Modified and updated Inclusion and exclusion criteria •Limited dose of drug to be injected into each targeted muscle to a minimum and maximum range •Modified scientific rationale to match updates •Modified dose justification •Updated the list of prohibited medications •Modified the injectable muscles and injection volume by muscle table (Appendix A) •Added appendices for Cervical Dystonia Impact Profile (CDIP-58), Treatment Satisfaction Questionnaire (TSQ), Work Productivity and Activity Impairment (WPAI), Questionnaire and Short Form-36 (SF-36) Survey.
    27 Mar 2018
    Protocol Amendment 2 •Clarified inclusion and exclusion criteria; added 1 exclusion criterion. •Clarified sections describing study intervention. •Updated secondary and exploratory endpoints. •Added more instructions for dose selection and clarified study product description. •Clarified that subjects could discontinue study product due to safety or at any time during the study as well as when there was no treatment benefit. •Clarified study assessment sections and moved some examples and scales to the appendices. •Reorganized the statistical analysis section to align with headings of ICH E9 guidance. •Added prior treatment experience, age, and gender as subgroup analyses in the exploratory endpoints. •Made wording changes to endpoints for clarification. •Changed sample size to approximately 300. •Clarified muscles for injections and added mandatory parameters. •Clarified BoNT, BoNTA and made global terminology changes for clarification: DAXI to DAXI for injection, total TWSTRS score to TWSTRS total score, medical judgment to clinical judgment.
    04 Jun 2018
    Protocol Amendment 3 •Added more information about Study RT002-CL005 to the introduction. •Added dysphagia as a known potential risk. •Updated study design section for clarification. •Clarified and updated inclusion and exclusion criteria. •Updated text about allowed concomitant medications; for focal dystonia treatments, amended time for stable dose requirement from 3 months to 4 weeks. •Amended recruitment and retention strategies section. •Clarified study intervention sections. •Amended pregnancy section. •Updated UPs section to align with guidance from regulatory authorities and added suspected unexpected serious adverse reaction (SUSAR) information. •Clarified sections about EOS Visit, TWSTRS, AE expectedness, follow-up of nonserious AEs, and follow-up of post-study SAEs. •In the statistical analysis section, added objectives with endpoints and clarified efficacy endpoints. •Updated information for key study personnel. •Updated study oversight text to reflect that the Data Safety Monitoring Board (DSMB) was to review unblinded data, as this was an open-label study.
    27 Jun 2018
    Protocol Amendment 4 •Increased sample size to approximately 350. •Clarified sample size justification and Increased sample size in 4.1 Overall design. •Clarified that a subject who has no reduction or an increase from baseline in the average TWSTRS-total score at Weeks 4 and 6 is the same as a subject who has a lack of efficacy. •Updated Figure 1. •Clarified major inclusion criteria. •Clarified that a subject who has no reduction or an increase from baseline in the average TWSTRS-total score at Weeks 4 and 6 is the same as a subject who has a lack of efficacy in 4.1. Overall design, and in 4.6 End of study definition and 7.2 Subject discontinuation/withdrawal from the study. •Updated 4.5 Justification for dose.
    10 Jul 2019
    Protocol Amendment 5 •Added National Clinical Trial Identified Number NCT03617367. •Added “Blood sample for antibody testing” at Week 4 after baseline injection and Week 4 after all retreatments. •Updated Medical Monitor’s information.

    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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