A Study to Investigate the Pharmacokinetics and Safety of Dupilumab in Participants ≥2 Years to <12 Years of Age With Uncontrolled Chronic Spontaneous Urticaria (CSU) (LIBERTY-CSU CUPIDKids)

Study Overview
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Brief Summary
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This is a multicenter, single-arm, 24-week treatment, Phase 3 study. The purpose of this study is to investigate the PK and safety of dupilumab in children diagnosed with CSU who remain symptomatic despite the use of H1-antihistamine treatment. Study details include: Screening: 2 to 4 weeks; The treatment duration will be 24 weeks; Follow-up period: 12 weeks; The study duration will be 38 to 40 weeks (including screening and follow-up); The number of study visits will be 6.

Official Title
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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)

Conditions
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Chronic Spontaneous Urticaria

Intervention / Treatment
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* Drug: Dupilumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participant must be ≥ 2 years to <12 years of age, at the time of signing the informed consent. Participants who have history of a diagnosis of CSU prior to screening (Visit 1) or symptoms consistent with a diagnosis of CSU for at least 3 months in the Investigator's opinion. Participants with CSU (characterized by recurrent itchy wheals with or without angioedema for >6 weeks) who remain symptomatic at the time of screening despite regular H1-antihistamine treatment. Body weight within ≥5 kg to <60 kg. Participant/parent(s)/caregiver(s)/participant's legally authorized representative, as appropriate, willing and able to comply with study visits and related procedures. Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply: Underlying etiology for chronic urticarias other than CSU. Presence of skin morbidities other than CSU that may interfere with the assessment of the study outcomes. Participants with a diagnosis of chronic inducible cold urticaria. Participants with active AD. Severe concomitant illness(es) that, in the Investigator's judgment, would adversely affect the patient's participation in the study. Participants with active tuberculosis (TB) or non-tuberculous mycobacterial infection, or a history of incompletely treated. Diagnosed with, suspected of, or at high risk of endoparasitic infection. Active chronic or acute infection requiring treatment with systemic antibiotics, antivirals, or antifungals within 2 weeks before the screening visit or during the screening period. Known or suspected immunodeficiency. Active malignancy or history of malignancy within 5 years before the baseline visit. History of systemic hypersensitivity or anaphylaxis to dupilumab including any excipient. Participation in prior dupilumab clinical study or have been treated with commercially available dupilumab. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 11 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Dupilumab<br>Administered subcutaneously (SC) every 4 weeks (Q4W) or every 2 weeks (Q2W) with or without an initial loading dose based on weight and age | Drug: Dupilumab<br>* Injection solution Subcutaneous<br>* Other names: Dupixent;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Concentration of dupilumab in serum over time including Ctrough at Week 12 and Week 24 | Concentration of dupilumab in serum over time including Ctrough at Week 12 and Week 24. | Week 12 and Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs) | Incidence of treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs). | Baseline to Week 36 | 
| Incidence of anti-drug antibodies (ADA) to dupilumab over time | Incidence of anti-drug antibodies (ADA) to dupilumab over time. | Baseline to Week 36 | 
| Change from baseline in Children's Dermatology Quality Life Index (C-DLQI) in children from 4 years to less than 12 years of age at Week 24 | The C-DLQI is a validated questionnaire designed to measure the impact of skin disease on children's Health-Related Quality of Life (HRQoL). The questionnaire is validated for children aged ≥4 to <16 years. The C-DLQI total score ranges 0 to 30. The higher the score, the greater the impact is on the child's HRQoL. | Baseline to Week 24 | 
| Change from baseline in Infants' Dermatitis Quality of Life Index (IDQOL) in children from 2 years to less than 4 years of age at Week 24 | The IDQOL is a validated questionnaire designed for use in children aged <4 years. The IDLQI total score ranges 0 to 30. The higher the score, the greater the impact is on the child's HRQoL. | Baseline to Week 24 | 
| Change from baseline in the modified Urticaria Activity Score (UAS7) at Week 24 | The UAS is a validated patient reported outcome (PRO) measure. A modified version of the UAS (mUAS) will be used in this study to account for the smaller body surface area of child and adolescent patients. Daily mUAS scores are summed over 7-day period to create the mUAS7, ranging from 0 to 42. The higher score indicates the greater urticaria activity. | Baseline to Week 24 | 
| Change from baseline in the modified itch severity score over 7 days (ISS7) at Week&nbsp;24 | ISS ranges from 0 (absent) to 3 (intense). Daily ISS scores are summed over 7-day period to create the ISS7, ranging from 0 to 21. | Baseline to Week 24 | 
| Change from baseline in the modified Hive Severity Score over 7 days (HSS7) at Week 24 | HSS ranges from 0 (absent) to 3 (intense, (>30 wheals/24 hours or large confluent areas of wheals). Daily HSS scores are summed over 7-day period to create the HSS7, ranging from 0 to 21. | Baseline to Week 24 |