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    Clinical Trial Results:
    A Remote, Double-Blind, Randomized, Placebo-Controlled Study of Rotigotine Transdermal System in Adolescent Subjects With Idiopathic Restless Legs Syndrome

    Summary
    EudraCT number
    		 2018-001445-13
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    07 Apr 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    21 Oct 2023
    First version publication date
    21 Oct 2023
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    SP1006
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03728933
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    UCB Biopharma SRL
    Sponsor organisation address
    Allée de la Recherche 60, Brussels, Belgium, 1070
    Public contact
    Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
    Scientific contact
    Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    26 Apr 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    23 Jul 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Apr 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To demonstrate the efficacy of rotigotine against placebo in adolescent subjects with idiopathic Restless Legs Syndrome (RLS) over a 12-week Maintenance Period.
    Protection of trial subjects
    During the conduct of the study all participants were closely monitored.
    Background therapy
    		 Background therapy as permitted in the protocol.
    Evidence for comparator
    		 Not applicable
    Actual start date of recruitment
    20 Dec 2018
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Regulatory reason
    Long term follow-up duration
    		 1 Years
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 23
    Worldwide total number of subjects
    23
    EEA total number of subjects
    0
    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)
    23
    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 study started to enroll participants in December 2018 and concluded prematurely in July 2022.

    Pre-assignment
    Screening details
    		 The Participant Flow refers to the Randomized Set.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Participants randomized to this arm received placebo as a comparator matched to rotigotine during 3 week titration period and is continued throughout the 12-week Maintenance Period.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Transdermal patch
    Routes of administration
    Transdermal use
    Dosage and administration details
    Participants received placebo as a comparator matched to rotigotine during 3 week titration period and is continued throughout the 12-week Maintenance Period.

    Arm title
    		 Rotigotine 2 mg/24h
    Arm description
    		 Participants randomized to this arm were initiated on 1 milligram (mg)/24 hours (h) rotigotine and up-titrated to a maximum of 2 mg/24 h rotigotine during 3 week titration period and the same dose is continued throughout the 12-week Maintenance Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Rotigotine
    Investigational medicinal product code
    Other name
    Neupro
    Pharmaceutical forms
    Transdermal patch
    Routes of administration
    Transdermal use
    Dosage and administration details
    Participants randomized to this arm were initiated on 1 mg/24 h rotigotine and up-titrated to a maximum of 2 mg/24 h rotigotine during 3 week titration period and the same dose is continued throughout the 12-week Maintenance Period.

    Arm title
    		 Rotigotine 3 mg/24h
    Arm description
    		 Participants randomized to this arm were initiated on 1 mg/24 h rotigotine and up-titrated to a maximum of 3 mg/24 h rotigotine during 3 week titration period and the same dose is continued throughout the 12-week Maintenance Period.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Rotigotine
    Investigational medicinal product code
    Other name
    Neupro
    Pharmaceutical forms
    Transdermal patch
    Routes of administration
    Transdermal use
    Dosage and administration details
    Participants randomized to this arm were initiated on 1 mg/24 h rotigotine and up-titrated to a maximum of 3 mg/24 h rotigotine during 3 week titration period and the same dose is continued throughout the 12-week Maintenance Period.

    Number of subjects in period 1
    		Placebo Rotigotine 2 mg/24h Rotigotine 3 mg/24h
    Started
    8
    8
    7
    Completed
    5
    7
    6
    Not completed
    3
    1
    1
         Consent withdrawn by subject
    -
    -
    1
         Adverse event, non-fatal
    1
    -
    -
         Withdrawal by Parent/Guardian
    2
    -
    -
         Lost to follow-up
    -
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants randomized to this arm received placebo as a comparator matched to rotigotine during 3 week titration period and is continued throughout the 12-week Maintenance Period.

    Reporting group title
    Rotigotine 2 mg/24h
    Reporting group description
    		 Participants randomized to this arm were initiated on 1 milligram (mg)/24 hours (h) rotigotine and up-titrated to a maximum of 2 mg/24 h rotigotine during 3 week titration period and the same dose is continued throughout the 12-week Maintenance Period.

    Reporting group title
    Rotigotine 3 mg/24h
    Reporting group description
    		 Participants randomized to this arm were initiated on 1 mg/24 h rotigotine and up-titrated to a maximum of 3 mg/24 h rotigotine during 3 week titration period and the same dose is continued throughout the 12-week Maintenance Period.

    Reporting group values
    		 Placebo Rotigotine 2 mg/24h Rotigotine 3 mg/24h Total
    Number of subjects
    		 8 8 7 23
    Age Categorical
    Units: Subjects
        Adolescents (12-17 years)
    		 8 8 7 23
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 15.4 ( 1.3 ) 16.1 ( 1.1 ) 15.6 ( 1.0 ) -
    Gender Categorical
    Units: Subjects
        Female
    		 6 6 2 14
        Male
    		 2 2 5 9

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants randomized to this arm received placebo as a comparator matched to rotigotine during 3 week titration period and is continued throughout the 12-week Maintenance Period.

    Reporting group title
    Rotigotine 2 mg/24h
    Reporting group description
    		 Participants randomized to this arm were initiated on 1 milligram (mg)/24 hours (h) rotigotine and up-titrated to a maximum of 2 mg/24 h rotigotine during 3 week titration period and the same dose is continued throughout the 12-week Maintenance Period.

    Reporting group title
    Rotigotine 3 mg/24h
    Reporting group description
    		 Participants randomized to this arm were initiated on 1 mg/24 h rotigotine and up-titrated to a maximum of 3 mg/24 h rotigotine during 3 week titration period and the same dose is continued throughout the 12-week Maintenance Period.

    Primary: Change from Baseline in Clinical Global Impressions (CGI) Item 1 to the end of the Maintenance Period

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    End point title
    		 Change from Baseline in Clinical Global Impressions (CGI) Item 1 to the end of the Maintenance Period [1]
    End point description
    The Clinical Global Impressions Item 1 (Severity of Illness) score ranges from 0 to 7 as follows: 0=not assessed, 1=normal, not ill at all, 2=borderline ill, 3=mildly ill, 4=moderately ill, 5=markedly ill, 6=severely ill, 7=among the most extremely ill. The CGI Item 1 was completed during an interview between the participant and the investigator or designee. A negative change from Baseline indicates improvement. The FAS consisted of all participants from the Safety Set who had a valid IRLS score and a valid CGI Item 1 score at Baseline and a valid post-Baseline IRLS score and a valid post-Baseline CGI Item 1 score. Here, Number of Subjects analyzed signifies those who were evaluable for this endpoint.
    End point type
    Primary
    End point timeframe
    From Baseline to the end of the Maintenance Period (Day 106)
    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 formal statistical hypothesis testing was planned due to early stopping of this study. Results were summarized in tables as descriptive statistics only.
    End point values
    		 Placebo Rotigotine 2 mg/24h Rotigotine 3 mg/24h
    Number of subjects analysed
    5
    7
    5
    Units: score on a scale
        arithmetic mean (standard deviation)
    -1.0 ( 1.4 )
    -1.7 ( 1.1 )
    -0.8 ( 0.8 )
    No statistical analyses for this end point

    Primary: Change from Baseline to the end of the Maintenance Period in International Restless Legs Rating Scale (IRLS) sum score

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    End point title
    		 Change from Baseline to the end of the Maintenance Period in International Restless Legs Rating Scale (IRLS) sum score [2]
    End point description
    The IRLS consisted of 10 questions, each scored using a 5-point scale ranging from 0=not present to 4=very severe. The IRLS sum score was calculated by summing up the single scores of all applicable questions, i.e., the total sum score ranged from 0 (no RLS symptoms present) to 40 (maximum severity in all symptoms). A score between 31 and 40, indicates very severe RLS. A score between 21 and 30 indicates severe RLS. A score between 11 and 20 indicates moderate RLS. A score between 1 and 10 indicates mild RLS and a score of 0 means no RLS. A negative change from Baseline indicates improvement. The Full Analysis Set (FAS) consisted of all participants from the Safety Set who had a valid IRLS score and a valid clinical global impressions (CGI) Item 1 score at Baseline and a valid post-Baseline IRLS score and a valid post-Baseline CGI Item 1 score. Here, Number of Subjects analyzed signifies those who were evaluable for this endpoint.
    End point type
    Primary
    End point timeframe
    From Baseline to the end of the Maintenance Period (Day 106)
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No formal statistical hypothesis testing was planned due to early stopping of this study. Results were summarized in tables as descriptive statistics only.
    End point values
    		 Placebo Rotigotine 2 mg/24h Rotigotine 3 mg/24h
    Number of subjects analysed
    5
    7
    5
    Units: score on a scale
        arithmetic mean (standard deviation)
    -8.2 ( 6.8 )
    -14.0 ( 8.2 )
    -6.4 ( 5.8 )
    No statistical analyses for this end point

    Primary: Percentage of participants with treatment-emergent adverse events (TEAEs) leading to withdrawals

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    End point title
    		 Percentage of participants with treatment-emergent adverse events (TEAEs) leading to withdrawals [3]
    End point description
    TEAEs were defined as events that started during the Treatment Period or within 30 days following the end of the Treatment Period (i.e., on or after the date of first patch application and within 30 days following the date of last patch removal + 1 day), or those events where the intensity worsened within this time frame. The Safety Set consisted of all participants from the RS who had at least one patch (rotigotine or placebo) applied.
    End point type
    Primary
    End point timeframe
    From Baseline to Safety Follow-Up (up to Week 20)
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No formal statistical hypothesis testing was planned due to early stopping of this study. Results were summarized in tables as descriptive statistics only.
    End point values
    		 Placebo Rotigotine 2 mg/24h Rotigotine 3 mg/24h
    Number of subjects analysed
    8
    8
    7
    Units: percentage of participants
        number (not applicable)
    12.5
    0
    0
    No statistical analyses for this end point

    Primary: Percentage of participants with treatment-emergent adverse events (TEAEs)

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    End point title
    		 Percentage of participants with treatment-emergent adverse events (TEAEs) [4]
    End point description
    TEAEs were defined as events that started during the Treatment Period or within 30 days following the end of the Treatment Period (i.e., on or after the date of first patch application and within 30 days following the date of last patch removal + 1 day), or those events where the intensity worsened within this time frame. The Safety Set consisted of all participants from the Randomized Set (RS) who had at least one patch (rotigotine or placebo) applied.
    End point type
    Primary
    End point timeframe
    From Baseline to Safety Follow-Up (up to Week 20)
    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: No formal statistical hypothesis testing was planned due to early stopping of this study. Results were summarized in tables as descriptive statistics only.
    End point values
    		 Placebo Rotigotine 2 mg/24h Rotigotine 3 mg/24h
    Number of subjects analysed
    8
    8
    7
    Units: percentage of participants
        number (not applicable)
    87.5
    87.5
    71.4
    No statistical analyses for this end point

    Secondary: Change from Baseline in Restless Legs-6 Rating Scales (RLS-6) to the end of the Maintenance Period

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    End point title
    		 Change from Baseline in Restless Legs-6 Rating Scales (RLS-6) to the end of the Maintenance Period
    End point description
    The RLS-6 Rating Scales was designed to assess the severity of RLS and consisted of 6 subscales. The subscales assessed severity of symptoms at the following times of the day/evening: falling asleep, during the night, during the day at rest, and during the day when engaged in daytime activities. In addition, the subscales assessed satisfaction with sleep and severity of daytime tiredness/sleepiness. Scores for each of the 6 subscales ranged from 0 (completely satisfied) to 10 (completely dissatisfied). The change from baseline was derived for each of the subscales and reported in this endpoint. A negative change from Baseline indicates improvement. The FAS consisted of all participants from the Safety Set who had a valid IRLS score and a valid CGI Item 1 score at Baseline and a valid post-Baseline IRLS score and a valid post-Baseline CGI Item 1 score. Here, Number of Subjects analyzed signifies those who were evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    From Baseline to the end of the Maintenance Period (Day 106)
    End point values
    		 Placebo Rotigotine 2 mg/24h Rotigotine 3 mg/24h
    Number of subjects analysed
    5
    7
    5
    Units: score on a scale
    arithmetic mean (standard deviation)
        Satisfaction with sleep
    -1.8 ( 4.1 )
    -4.7 ( 2.3 )
    -2.0 ( 2.8 )
        Severity: RLS symptoms at falling asleep
    -2.8 ( 3.6 )
    -5.6 ( 1.6 )
    -2.8 ( 1.8 )
        Severity: RLS symptoms during the night
    -1.0 ( 2.5 )
    -2.9 ( 3.0 )
    -2.4 ( 2.2 )
        Severity: RLS symptoms during the day - at rest
    -1.4 ( 1.3 )
    -3.6 ( 3.5 )
    -2.4 ( 2.6 )
        Severity: RLS symptoms during the day-not at rest
    -3.8 ( 1.9 )
    -4.3 ( 3.5 )
    -0.4 ( 1.1 )
        How tired or sleepy during the day
    -3.0 ( 2.8 )
    -5.6 ( 2.1 )
    -3.0 ( 1.9 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Baseline to Safety Follow-Up (up to Week 20)
    Adverse event reporting additional description
    TEAEs were defined as events that started during Treatment Period or within 30 days following the end of Treatment Period (i.e., on or after the date of first patch application and within 30 days following the date of last patch removal + 1 day), or those events where intensity worsened within this time frame. TEAEs were analyzed for Safety Set.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants randomized to this arm received placebo as a comparator matched to rotigotine during 3 week titration period and is continued throughout the 12-week Maintenance Period.

    Reporting group title
    Rotigotine 3 mg/24h
    Reporting group description
    		 Participants randomized to this arm were initiated on 1 mg/24 h rotigotine and up-titrated to a maximum of 3 mg/24 h rotigotine during 3 week titration period and the same dose is continued throughout the 12-week Maintenance Period.

    Reporting group title
    Rotigotine 2 mg/24h
    Reporting group description
    		 Participants randomized to this arm were initiated on 1 mg/24 h rotigotine and up-titrated to a maximum of 2 mg/24 h rotigotine during 3 week titration period and the same dose is continued throughout the 12-week Maintenance Period.

    Serious adverse events
    		 Placebo Rotigotine 3 mg/24h Rotigotine 2 mg/24h
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 8 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Metabolism and nutrition disorders
    Type 2 diabetes mellitus
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 8 (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
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Placebo Rotigotine 3 mg/24h Rotigotine 2 mg/24h
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 8 (87.50%)
    5 / 7 (71.43%)
    7 / 8 (87.50%)
    Surgical and medical procedures
    Wisdom teeth removal
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    1
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    0
    Fatigue
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    1
    0
    1
    Application site pain
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    1
    0
    1
    Application site irritation
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    1
    Application site pruritus
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    3 / 8 (37.50%)
         occurrences all number
    0
    1
    3
    Application site erythema
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 7 (14.29%)
    4 / 8 (50.00%)
         occurrences all number
    2
    1
    5
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    Reproductive system and breast disorders
    Amenorrhoea
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Upper-airway cough syndrome
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    Dyspnoea
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    Attention deficit hyperactivity disorder
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    1
    Investigations
    Serum ferritin decreased
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    Transferrin saturation decreased
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    Thyroid function test abnormal
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    Injury, poisoning and procedural complications
    Wound
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    1
    Skin abrasion
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    Nervous system disorders
    Migraine
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    Dizziness
         subjects affected / exposed
    2 / 8 (25.00%)
    1 / 7 (14.29%)
    0 / 8 (0.00%)
         occurrences all number
    2
    1
    0
    Headache
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    1 / 8 (12.50%)
         occurrences all number
    0
    4
    1
    Blood and lymphatic system disorders
    Lymphadenopathy
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    Eye disorders
    Eye pain
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    0
    Nausea
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    2 / 8 (25.00%)
         occurrences all number
    1
    0
    4
    Vomiting
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    2 / 8 (25.00%)
         occurrences all number
    1
    0
    2
    Diarrhoea
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    2
    Haemorrhoids
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    Skin and subcutaneous tissue disorders
    Eczema
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    Urticaria papular
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    Skin irritation
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 7 (0.00%)
    2 / 8 (25.00%)
         occurrences all number
    0
    0
    3
    Pruritus
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    2 / 8 (25.00%)
         occurrences all number
    0
    1
    2
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    0
    Gastroenteritis viral
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 8 (12.50%)
    3 / 7 (42.86%)
    0 / 8 (0.00%)
         occurrences all number
    1
    3
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    19 Mar 2019
    The main purpose of Amendment 1 (dated 19 Mar 2019) was to update Exclusion Criterion with a change in the ferritin level at Visit 1/Screening from <50ng/mL to below the lower limit of normal. Other changes included in this amendment were as follows: • Sponsor contact information was updated. • Added headings for primary and other safety variables. Clarified that AEs are treatment emergent. Categorized occurrence of TEAEs and TEAEs leading to withdrawal as primary safety variables. The remaining safety variables were categorized as other. • Changed the eC-SSRS to the C-SSRS. • Addition of urine drug screen at Unscheduled Visits. • Clarified that the smartphone technology was to be used in combination with visits from mobile study personnel to subjects’/legal representatives’ homes. Visits to local health care providers or Patient Service Centers were not conducted during this study. • Clarified that a serum pregnancy test was to be performed in females at Screening and urine pregnancy test at all other visits. A positive urine pregnancy test must have been confirmed by a serum pregnancy test. • Added a description of the Where’s My Patch (WMP) app. • Removed BMI of <95th percentile for his or her age group, according to the Child and Teen BMI calculator as Inclusion Criterion. • Clarified that the washout period for supplemental iron is 1 month prior to Baseline in Exclusion Criterion. • Added secondary RLS (eg, due to renal insufficiency [uremia], iron deficiency, or rheumatoid arthritis as Exclusion Criterion. • Added a lifetime history of suicide attempts or suicidal ideation in the past 6 months as indicated by a positive response (“Yes”) to either Question 4 or Question 5 of the C-SSRS at Screening as Exclusion Criterion. • Added taking a prohibited concomitant medication and the washout period as Exclusion Criterion.
    19 Mar 2019
    Continuation of Amendment 1 rationale: • Clarified that subjects who have been screened but not randomized may be rescreened with the permission of the Study Physician or representative. • Added RBC indices (mean corpuscular hemoglobin; mean corpuscular hemoglobin concentration; mean cell volume; cell distribution width) to the laboratory measurements. • Typographic errors and changes of an editorial nature were made.

    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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