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    Clinical Trial Results:
    A multi-center, single-arm study to investigate the pharmacokinetics and safety of dupilumab in male and female participants >=2 years to <12 years of age with uncontrolled chronic spontaneous urticaria (CSU)

    Summary
    EudraCT number
    		 2022-000260-22
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    03 Feb 2025

    Results information
    Results version number
    		 v1(current)
    This version publication date
    16 Aug 2025
    First version publication date
    16 Aug 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    PKM16982
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05526521
    WHO universal trial number (UTN)
    		 U1111-1266-5669
    Sponsors
    Sponsor organisation name
    Sanofi aventis recherche & développement
    Sponsor organisation address
    82 Avenue Raspail, Gentilly, France, 94250
    Public contact
    Trial Transparency Team, Sanofi aventis recherche & développement, Contact-US@sanofi.com
    Scientific contact
    Trial Transparency Team, Sanofi aventis recherche & développement, Contact-US@sanofi.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001501-PIP07-20
    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
    28 Feb 2025
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    03 Feb 2025
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To characterize the serum concentration of dupilumab over time.
    Protection of trial subjects
    The study was conducted by investigators experienced in the treatment of pediatric participants. The parent(s) or guardian(s) as well as the children were fully informed of all pertinent aspects of the clinical trial as well as the possibility to discontinue at any time. In addition to the consent form for the parent(s)/guardian(s), an assent form in child-appropriate language was provided and explained to the child. Repeated invasive procedures were minimized. The number of blood samples as well as the amount of blood drawn were adjusted according to age and weight. A topical anesthesia might have been used to minimize distress and discomfort.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    25 Aug 2022
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Canada: 4
    Country: Number of subjects enrolled
    Japan: 3
    Country: Number of subjects enrolled
    United States: 8
    Worldwide total number of subjects
    15
    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)
    15
    Adolescents (12-17 years)
    0
    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
    		 A total of 23 participants were screened from 25-Aug-2022 to 02-May-2024 of which 8 were screen failures mainly due to not meeting eligibility criteria.

    Pre-assignment
    Screening details
    		 A total of 15 participants were enrolled in the study to receive dupilumab at 1 of 4 dose regimens based on the body weight and age.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Dupilumab 200 mg Q4W
    Arm description
    		 Participants received dupilumab 200 milligrams (mg) subcutaneous (SC) injection every 4 weeks (Q4W) with no loading dose in children aged >=2 years to <12 years with body weight >=5 kilograms (kg) and <15 kg from Day 1 up to 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Dupilumab
    Investigational medicinal product code
    SAR231893
    Other name
    REGN668, Dupixent
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Dupilumab was administered for 24 weeks as specified in the protocol.

    Arm title
    		 Dupilumab 300 mg Q4W
    Arm description
    		 Participants received dupilumab 300 mg SC injection Q4W with no loading dose in children aged >=2 years to <6 years with body weight >=15 kg and <30 kg from Day 1 up to 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Dupilumab
    Investigational medicinal product code
    SAR231893
    Other name
    REGN668, Dupixent
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Dupilumab was administered for 24 weeks as specified in the protocol.

    Arm title
    		 Dupilumab 300 mg Q4W, 600 mg Loading Dose
    Arm description
    		 Participants received dupilumab 300 mg SC injection Q4W with an initial 600 mg loading dose in children aged >=6 years to <12 years with body weight >=15 kg and <30 kg from Day 1 up to 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Dupilumab
    Investigational medicinal product code
    SAR231893
    Other name
    REGN668, Dupixent
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Dupilumab was administered for 24 weeks as specified in the protocol.

    Arm title
    		 Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Arm description
    		 Participants received dupilumab 200 mg SC injection every 2 weeks (Q2W) with an initial 400 mg loading dose in children aged >=2 years to <12 years with body weight >=30 kg and <60 kg from Day 1 up to 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Dupilumab
    Investigational medicinal product code
    SAR231893
    Other name
    REGN668, Dupixent
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Dupilumab was administered for 24 weeks as specified in the protocol.

    Number of subjects in period 1
    		Dupilumab 200 mg Q4W Dupilumab 300 mg Q4W Dupilumab 300 mg Q4W, 600 mg Loading Dose Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Started
    1
    4
    2
    8
    Completed
    1
    3
    2
    8
    Not completed
    0
    1
    0
    0
         Consent withdrawn by subject
    -
    1
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Dupilumab 200 mg Q4W
    Reporting group description
    		 Participants received dupilumab 200 milligrams (mg) subcutaneous (SC) injection every 4 weeks (Q4W) with no loading dose in children aged >=2 years to <12 years with body weight >=5 kilograms (kg) and <15 kg from Day 1 up to 24 weeks.

    Reporting group title
    Dupilumab 300 mg Q4W
    Reporting group description
    		 Participants received dupilumab 300 mg SC injection Q4W with no loading dose in children aged >=2 years to <6 years with body weight >=15 kg and <30 kg from Day 1 up to 24 weeks.

    Reporting group title
    Dupilumab 300 mg Q4W, 600 mg Loading Dose
    Reporting group description
    		 Participants received dupilumab 300 mg SC injection Q4W with an initial 600 mg loading dose in children aged >=6 years to <12 years with body weight >=15 kg and <30 kg from Day 1 up to 24 weeks.

    Reporting group title
    Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Reporting group description
    		 Participants received dupilumab 200 mg SC injection every 2 weeks (Q2W) with an initial 400 mg loading dose in children aged >=2 years to <12 years with body weight >=30 kg and <60 kg from Day 1 up to 24 weeks.

    Reporting group values
    		 Dupilumab 200 mg Q4W Dupilumab 300 mg Q4W Dupilumab 300 mg Q4W, 600 mg Loading Dose Dupilumab 200 mg Q2W, 400 mg Loading Dose Total
    Number of subjects
    		 1 4 2 8 15
    Age Categorical
    Units: participants
        >=2 to <6 years
    		 0 4 0 0 4
        >=6 to 12 years
    		 1 0 2 8 11
    Sex: Female, Male
    Units: participants
        Female
    		 1 2 2 6 11
        Male
    		 0 2 0 2 4
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0 0 0 0
        Asian
    		 1 0 1 2 4
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0 0
        Black or African American
    		 0 0 0 1 1
        White
    		 0 4 1 4 9
        More than one race
    		 0 0 0 0 0
        Unknown or Not Reported
    		 0 0 0 1 1

    End points

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    End points reporting groups
    Reporting group title
    Dupilumab 200 mg Q4W
    Reporting group description
    		 Participants received dupilumab 200 milligrams (mg) subcutaneous (SC) injection every 4 weeks (Q4W) with no loading dose in children aged >=2 years to <12 years with body weight >=5 kilograms (kg) and <15 kg from Day 1 up to 24 weeks.

    Reporting group title
    Dupilumab 300 mg Q4W
    Reporting group description
    		 Participants received dupilumab 300 mg SC injection Q4W with no loading dose in children aged >=2 years to <6 years with body weight >=15 kg and <30 kg from Day 1 up to 24 weeks.

    Reporting group title
    Dupilumab 300 mg Q4W, 600 mg Loading Dose
    Reporting group description
    		 Participants received dupilumab 300 mg SC injection Q4W with an initial 600 mg loading dose in children aged >=6 years to <12 years with body weight >=15 kg and <30 kg from Day 1 up to 24 weeks.

    Reporting group title
    Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Reporting group description
    		 Participants received dupilumab 200 mg SC injection every 2 weeks (Q2W) with an initial 400 mg loading dose in children aged >=2 years to <12 years with body weight >=30 kg and <60 kg from Day 1 up to 24 weeks.

    Primary: Serum Concentration of Dupilumab at Weeks 12 and 24

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    End point title
    		 Serum Concentration of Dupilumab at Weeks 12 and 24 [1]
    End point description
    Blood samples were collected at specified timepoints to obtain dupilumab concentration. The pharmacokinetic (PK) population included all enrolled and treated participants (safety population) with at least 1 post-baseline PK result. Only those participants with data collected at Weeks 12 or 24 are reported. Here, n= number of participants with data collected for specified categories. 99999=standard deviation (SD) cannot be calculated for 1 participant.
    End point type
    Primary
    End point timeframe
    Weeks 12 and 24
    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: As the endpoint is descriptive in nature, no statistical analysis have been presented.
    End point values
    		 Dupilumab 200 mg Q4W Dupilumab 300 mg Q4W Dupilumab 300 mg Q4W, 600 mg Loading Dose Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Number of subjects analysed
    1
    3
    2
    8
    Units: nanogram/milliliter
    arithmetic mean (standard deviation)
        Week 12 (n=1,1,2,8)
    67700 ( 99999 )
    105000 ( 99999 )
    45800 ( 12400 )
    91600 ( 23000 )
        Week 24 (n=1,3,2,8)
    116000 ( 99999 )
    141000 ( 30100 )
    51100 ( 20100 )
    78500 ( 31600 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

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    End point title
    		 Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
    End point description
    An adverse event (AE) was defined as any untoward medical occurrence in a participant or clinical trial participant, temporally associated with the use of study intervention, whether or not considered related to study intervention. Serious adverse event (SAE) was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. TEAEs were defined as AEs that developed, worsened or became serious during the TE period. The safety population included all enrolled participants who took at least 1 dose of the study intervention, regardless of the amount of intervention administered.
    End point type
    Secondary
    End point timeframe
    From the first dose of study intervention (Day 1) up to end of follow-up, maximum up to 36 weeks
    End point values
    		 Dupilumab 200 mg Q4W Dupilumab 300 mg Q4W Dupilumab 300 mg Q4W, 600 mg Loading Dose Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Number of subjects analysed
    1
    4
    2
    8
    Units: participants
        TEAEs
    1
    2
    2
    7
        TESAEs
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants With Anti-drug Antibodies (ADAs) to Dupilumab

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    End point title
    		 Number of Participants With Anti-drug Antibodies (ADAs) to Dupilumab
    End point description
    Blood samples were collected at specified timepoints and ADA samples were assayed using validated methods. Treatment-emergent ADA response was defined as a positive response in the ADA assay post first dose when baseline results were negative or missing. Number of participants with treatment-emergent ADA response is presented. The ADA population included all enrolled participants treated with dupilumab with at least 1 post-baseline ADA result (positive, negative or inconclusive).
    End point type
    Secondary
    End point timeframe
    From the first dose of study intervention (Day 1) up to end of follow-up, maximum up to 36 weeks
    End point values
    		 Dupilumab 200 mg Q4W Dupilumab 300 mg Q4W Dupilumab 300 mg Q4W, 600 mg Loading Dose Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Number of subjects analysed
    1
    3
    2
    8
    Units: participants
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Change From Baseline in Children’s Dermatology Life Quality Index (C-DLQI) in Participants Aged 4 Years to <12 Years at Week 24

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    End point title
    		 Change From Baseline in Children’s Dermatology Life Quality Index (C-DLQI) in Participants Aged 4 Years to <12 Years at Week 24
    End point description
    The C-DLQI assesses impact of skin disease on children’s health-related quality of life (HRQoL) over the previous week, contains 10 questions related to symptoms feelings associated with disease, impact of disease on leisure, school or holidays, personal relationships, sleep, and side effects of treatment for the skin disease. All questions were scored on 4-point Likert scale:0 (not at all),1 (a little),2 (a lot),3 (very much). Total C-DLQI was calculated by summing the score of each question and ranged from 0 (no impact) to 30 (severe impact). Higher scores indicated poor HRQoL. Mean is presented. Baseline was defined as closest assessment to first study intervention administration on or prior to Day 1 but no later than Day 4. Analysis was performed on intent-to-treat (ITT) population. Only those participants with data collected at Baseline and Week 24 are reported.99999=SD cannot be calculated for 1 participant.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and Week 24
    End point values
    		 Dupilumab 200 mg Q4W Dupilumab 300 mg Q4W Dupilumab 300 mg Q4W, 600 mg Loading Dose Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Number of subjects analysed
    1
    1
    2
    7
    Units: score on a scale
        arithmetic mean (standard deviation)
    -3.0 ( 99999 )
    -6.0 ( 99999 )
    -10.5 ( 3.5 )
    -7.3 ( 4.3 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Infant’s Dermatitis Quality of Life Index (IDQOL) in Participants Aged 2 Years to <4 Years at Week 24

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    End point title
    		 Change From Baseline in Infant’s Dermatitis Quality of Life Index (IDQOL) in Participants Aged 2 Years to <4 Years at Week 24
    End point description
    The IDQOL questionnaire is completed by child’s caregiver/guardian with recall period of 7 days;consists of 10 questions focusing on life quality index (LQI) scored on 4-point Likert scale. Additional question on dermatitis severity scored on a 5-point Likert scale (0 [none] to 4 [extremely severe]) is not considered for calculating total IDQOL.For LQI, score ranges are: Questions 1, 5 to 10: 0 (none) to 3 (all the time/very much). Question 2: 0 (happy), 1 (slightly fretful), 2 (very fretful),3 (always crying). Question 3: 0 (0-15 minutes), 1 (15 minutes-1 hour), 2 (1-2 hours),3 (>2 hours).Question 4: 0 (<1 hour), 1 (1-2 hours), 2 (3-4 hours),3 (>=5 hours). IDQOL total score is sum of score of each question of LQI, ranges from 0 (no impact) to 30 (maximum impact). Higher scores indicated poor HRQoL. Mean is presented. Analysis performed on ITT population. Only those participants with data collected at Baseline and Week 24 are reported. 99999=SD cannot be calculated for 1 participant.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and Week 24
    End point values
    		 Dupilumab 200 mg Q4W Dupilumab 300 mg Q4W Dupilumab 300 mg Q4W, 600 mg Loading Dose Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Number of subjects analysed
    0 [2]
    1
    0 [3]
    0 [4]
    Units: score on a scale
        arithmetic mean (standard deviation)
    ( )
    -3.0 ( 99999 )
    ( )
    ( )
    Notes
    [2] - No participants were analyzed.
    [3] - No participants were analyzed.
    [4] - No participants were analyzed.
    No statistical analyses for this end point

    Secondary: Change From Baseline in Modified Urticaria Activity Score Over 7 Days (mUAS7) at Week 24

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    End point title
    		 Change From Baseline in Modified Urticaria Activity Score Over 7 Days (mUAS7) at Week 24
    End point description
    Modified version of UAS (mUAS) was used for smaller body surface area of child and adolescent participants. The mUAS was derived from sum of daily hives severity score (HSS) (ranging from 0 to 3 [0 = absent; 1 = mild {1 to <10 wheals/24 hours}; 2 = moderate: {10 to 30 wheals/24 hours}; and 3 = intense: {>30 wheals/24 hours or large confluent areas of wheals}]) and daily itch severity score (ISS) (ranging from 0 = none to 3 = intense).Daily mUAS total scores:0 to 6 (0 to 3 for ISS and 0 to 3 for HSS). Daily mUAS scores were summed over 7-day period to create total score ranging from 0 (no urticaria) to 42 (severe urticaria). Completion of mUAS7 was done by child or parent(s)/caregiver(s)/legal guardian(s) for participants aged >=4 years and by parent(s)/caregiver(s) for participants aged <4 years.Mean is presented. Analysis performed on ITT population.Only those participants with data collected at Baseline and Week 24 are reported. 99999=SD cannot be calculated for 1 participant.
    End point type
    Secondary
    End point timeframe
    Baseline (Day -7 to Day 1) and Week 24
    End point values
    		 Dupilumab 200 mg Q4W Dupilumab 300 mg Q4W Dupilumab 300 mg Q4W, 600 mg Loading Dose Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Number of subjects analysed
    1
    2
    2
    5
    Units: score on a scale
        arithmetic mean (standard deviation)
    -8.0 ( 99999 )
    -21.4 ( 1.9 )
    -2.8 ( 1.8 )
    -7.8 ( 6.6 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Itch Severity Score (ISS7) Over 7 Days at Week 24

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    End point title
    		 Change From Baseline in Itch Severity Score (ISS7) Over 7 Days at Week 24
    End point description
    The ISS represents severity of itch on a scale ranging from 0 (none) to 3 (intense). The ISS7 score was the sum of daily ISS scores recorded by a participant at the same time each day over 7 days with an overall scale of 0 (no impact) to 21 (severe impact). Higher scores indicated greater intensity of itch. Mean is presented. Baseline was defined as sum of the 7 daily measurements obtained within the 7 days prior to first study intervention administration. The ITT population included all enrolled participants. Only those participants with data collected at Baseline and Week 24 are reported. 99999=SD cannot be calculated for 1 participant.
    End point type
    Secondary
    End point timeframe
    Baseline (Day -7 to Day 1) and Week 24
    End point values
    		 Dupilumab 200 mg Q4W Dupilumab 300 mg Q4W Dupilumab 300 mg Q4W, 600 mg Loading Dose Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Number of subjects analysed
    1
    2
    2
    5
    Units: score on a scale
        arithmetic mean (standard deviation)
    -5.0 ( 99999 )
    -9.6 ( 3.7 )
    -1.6 ( 0.6 )
    -3.4 ( 3.2 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Hive Severity Score Over 7 Days (HSS7) at Week 24

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    End point title
    		 Change From Baseline in Hive Severity Score Over 7 Days (HSS7) at Week 24
    End point description
    The HSS7 score is the sum of daily HSS ranging from ranging from 0 to 3 (0 = absent; 1 = mild [1 to <10 wheals/24 hours]; 2 = moderate [10 to 30 wheals/24 hours]; and 3 = intense: [>30 wheals/24 hours or large confluent areas of wheals]) recorded by a participant at the same time of each day over 7 days with an overall scale of 0 (no hives) to 21 (severe hives). Higher scores indicate greater intensity of hives. Mean is presented. Baseline was defined as sum of the 7 daily measurements obtained within the 7 days prior to first study intervention administration. The ITT population included all enrolled participants. Only those participants with data collected at Baseline and Week 24 are reported. 99999=SD cannot be calculated for 1 participant.
    End point type
    Secondary
    End point timeframe
    Baseline (Day -7 to Day 1) and Week 24
    End point values
    		 Dupilumab 200 mg Q4W Dupilumab 300 mg Q4W Dupilumab 300 mg Q4W, 600 mg Loading Dose Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Number of subjects analysed
    1
    2
    2
    5
    Units: score on a scale
        arithmetic mean (standard deviation)
    -3.0 ( 99999 )
    -11.8 ( 1.8 )
    -1.2 ( 1.2 )
    -4.4 ( 3.6 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From the first dose of study intervention (Day 1) up to end of follow-up, maximum up to 36 weeks.
    Adverse event reporting additional description
    Analysis was performed on the safety population.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.1
    Reporting groups
    Reporting group title
    Dupilumab 200 mg Q4W
    Reporting group description
    		 Participants received dupilumab 200 mg SC injection Q4W with no loading dose in children aged >=2 years to <12 years with body weight >=5 kg and <15 kg from Day 1 up to 24 weeks.

    Reporting group title
    Dupilumab 300 mg Q4W
    Reporting group description
    		 Participants received dupilumab 300 mg SC injection Q4W with no loading dose in children aged >=2 years to <6 years with body weight >=15 kg and <30 kg from Day 1 up to 24 weeks.

    Reporting group title
    Dupilumab 300 mg Q4W, 600 mg Loading Dose
    Reporting group description
    		 Participants received dupilumab 300 mg SC injection Q4W with an initial 600 mg loading dose in children aged >=6 years to <12 years with body weight >=15 kg and <30 kg from Day 1 up to 24 weeks.

    Reporting group title
    Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Reporting group description
    		 Participants received dupilumab 200 mg SC injection Q2W with an initial 400 mg loading dose in children aged >=2 years to <12 years with body weight >=30 kg and <60 kg from Day 1 up to 24 weeks.

    Serious adverse events
    		 Dupilumab 200 mg Q4W Dupilumab 300 mg Q4W Dupilumab 300 mg Q4W, 600 mg Loading Dose Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 8 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Dupilumab 200 mg Q4W Dupilumab 300 mg Q4W Dupilumab 300 mg Q4W, 600 mg Loading Dose Dupilumab 200 mg Q2W, 400 mg Loading Dose
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    1 / 1 (100.00%)
    2 / 4 (50.00%)
    2 / 2 (100.00%)
    7 / 8 (87.50%)
    Injury, poisoning and procedural complications
    Accidental Overdose
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Vascular disorders
    Internal Haemorrhage
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    2 / 8 (25.00%)
         occurrences all number
    0
    0
    0
    2
    Migraine
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    General disorders and administration site conditions
    Injection Site Reaction
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    2
    Fatigue
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Swelling Face
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Pyrexia
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Immune system disorders
    Seasonal Allergy
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    0
    1
    Lip Swelling
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Diarrhoea
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Abdominal Pain Upper
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Abdominal Discomfort
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Rhinorrhoea
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Skin and subcutaneous tissue disorders
    Skin Discolouration
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Psoriasis
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Infections and infestations
    Gastroenteritis Viral
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    2
    Folliculitis
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Conjunctivitis Bacterial
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Covid-19
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Hand-Foot-And-Mouth Disease
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    1 / 2 (50.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hordeolum
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    1 / 2 (50.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Influenza
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    1 / 2 (50.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    1
    1
    Viral Upper Respiratory Tract Infection
         subjects affected / exposed
    1 / 1 (100.00%)
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    2 / 8 (25.00%)
         occurrences all number
    1
    1
    0
    3
    Viral Rash
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    2
    Upper Respiratory Tract Infection
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Subcutaneous Abscess
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    2 / 2 (100.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    3
    0
    Otitis Media
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    1 / 2 (50.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pharyngitis
         subjects affected / exposed
    0 / 1 (0.00%)
    0 / 4 (0.00%)
    0 / 2 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Rhinovirus Infection
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 4 (25.00%)
    0 / 2 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    31 May 2023
    The purpose of this amendment was to remove the inclusion of participants with chronic inducible cold urticaria (CICU) following the results of the phase 3 study EFC16720, which evaluated the efficacy and safety of dupilumab in adult participants with CICU who remained symptomatic despite the use of H1-antihistamine treatment. This study did not meet the required efficacy endpoints to continue this program, including the development in the pediatric CICU population. In addition, this amendment modified the inclusion criteria and the PK sampling schedule to increase flexibility and to reduce participant burden related to study visits and procedures.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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