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)

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)

This was a multicenter, single-arm, 24-week treatment, Phase 3 study. The purpose of this study was 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 included: Screening: 2 to 4 weeks; The treatment duration was 24 weeks; Follow-up period: 12 weeks; The study duration was 38 to 40 weeks (including screening and follow-up); The number of study visits was 6.

Study Overview

• Status: Completed
• Conditions: Chronic Spontaneous Urticaria
• Intervention / Treatment: Drug: Dupilumab

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2W 4X9
      • Investigational Site Number : 1240009
    • Edmonton, Alberta, Canada, T5J 3S9
      • Investigational Site Number : 1240010
  • Ontario
    • Hamilton, Ontario, Canada, L8S1G5
      • Investigational Site Number : 1240007
  • Quebec
    • Montreal, Quebec, Canada, H4A 3J1
      • Investigational Site Number : 1240001
• Japan
  • Kanagawa
    • Yokohama, Kanagawa, Japan, 232-8555
      • Investigational Site Number : 3920001
  • Mie-ken
    • Tsu, Mie-ken, Japan, 514-0125
      • Investigational Site Number : 3920002
• United States
  • Florida
    • Coral Gables, Florida, United States, 33146
      • Pediatric Dermatology of Miami Site Number : 8400015
  • Idaho
    • Boise, Idaho, United States, 83706
      • Treasure Valley Medical Research Site Number : 8400019
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Washington University School of Medicine- Site Number : 8400004
  • New York
    • Hawthorne, New York, United States, 10532
      • Boston Childrens Health Physicians Site Number : 8400017
    • New York, New York, United States, 10032
      • Columbia University Irving Medical Center Site Number : 8400003
  • Ohio
    • Akron, Ohio, United States, 44308
      • Childrens Hospital Medical Center of Akron- Site Number : 8400020
    • Cincinnati, Ohio, United States, 45229-3026
      • Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400001
  • Oklahoma
    • Tulsa, Oklahoma, United States, 74136
      • Vital Prospects Clinical Research Institute, P.C.- Site Number : 8400002
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Monroe Carell Jr. Childrens Hospital at Vanderbilt- Site Number : 8400005

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 11 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Participant must be ≥ 2 years to <12 years of age, at the time of signing the informed consent.
• Participants who had 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 were 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 participant'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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Dupilumab; 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; Injection solution Subcutaneous; Other Names: Dupixent

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Serum Concentration of Dupilumab at Weeks 12 and 24	Blood samples were collected at specified timepoints to obtain dupilumab concentration.	Weeks 12 and 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)	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.	From the first dose of study intervention (Day 1) up to end of follow-up, maximum up to 36 weeks
Number of Participants With Anti-drug Antibodies (ADAs) to Dupilumab	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.	From the first dose of study intervention (Day 1) up to end of follow-up, maximum up to 36 weeks
Change From Baseline in Children's Dermatology Life Quality Index (C-DLQI) in Participants Aged 4 Years to <12 Years at Week 24	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.	Baseline (Day 1) and Week 24
Change From Baseline in Infant's Dermatitis Quality of Life Index (IDQOL) in Participants Aged 2 Years to <4 Years at Week 24	The IDQOL questionnaire is completed by child's caregiver/guardian with a recall period of 7 days;consists of 10 questions focusing on life quality index (LQI) scored on 4-point Likert scale. An additional question on dermatitis severity is scored on a 5-point Likert scale (0 [none] to 4 [extremely severe]); it is not considered for calculating total IDQOL. For LQI, score ranges are as follows: 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 the sum of the score of each question of LQI, ranges from 0 (no impact) to 30 (maximum 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.	Baseline (Day 1) and Week 24
Change From Baseline in Modified Urticaria Activity Score Over 7 Days (mUAS7) at Week 24	A modified version of the UAS (mUAS) was used for the smaller body surface area of child and adolescent participants. The mUAS was derived from the 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 range from 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 the 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. Baseline was defined as sum of the 7 daily measurements obtained within the 7 days prior to first study intervention administration.	Baseline (Day -7 to Day 1) and Week 24
Change From Baseline in Itch Severity Score Over 7 Days (ISS7) at Week 24	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.	Baseline (Day -7 to Day 1) and Week 24
Change From Baseline in Hive Severity Score Over 7 Days (HSS7) at Week 24	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.	Baseline (Day -7 to Day 1) and Week 24

Collaborators and Investigators

• Sponsor: Sanofi
• Collaborators: Regeneron Pharmaceuticals

Publications and helpful links

Helpful Links

• PKM16982 Plain Language Results Summary

Study record dates

Study Major Dates

• Study Start (Actual): August 25, 2022
• Primary Completion (Actual): November 11, 2024
• Study Completion (Actual): February 3, 2025

Study Registration Dates

• First Submitted: August 31, 2022
• First Submitted That Met QC Criteria: August 31, 2022
• First Posted (Actual): September 2, 2022

Study Record Updates

• Last Update Posted (Estimated): November 25, 2025
• Last Update Submitted That Met QC Criteria: November 19, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Chronic Disease; Disease Attributes; Immune System Diseases; Hypersensitivity, Immediate; Hypersensitivity; Skin Diseases; Urticaria; Skin Diseases, Vascular; Pathological Conditions, Signs and Symptoms; Skin and Connective Tissue Diseases; Chronic Urticaria; dupilumab
• Other Study ID Numbers: PKM16982; U1111-1266-5669 (Registry Identifier: ICTRP); 2022-000260-22 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No