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)

• ClinicalTrials.gov Identifier: NCT05526521
• Recruitment Status: Recruiting
• First Posted: September 2, 2022
• Last Update Posted: March 12, 2024
• Sponsor: Sanofi
• Information provided by (Responsible Party): Sanofi

Tracking Information

• First Submitted Date: August 31, 2022
• First Posted Date: September 2, 2022
• Last Update Posted Date: March 12, 2024
• Actual Study Start Date: August 25, 2022
• Estimated Primary Completion Date: March 1, 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 31, 2022)

Concentration of dupilumab in serum over time including Ctrough at Week 12 and Week 24 [ Time Frame: Week 12 and Week 24 ]

Concentration of dupilumab in serum over time including Ctrough at Week 12 and Week 24.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: August 23, 2023)

• Incidence of treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs) [ Time Frame: Baseline to Week 36 ]
  Incidence of treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs).
• Incidence of anti-drug antibodies (ADA) to dupilumab over time [ Time Frame: Baseline to Week 36 ]
  Incidence of anti-drug antibodies (ADA) to dupilumab over time.
• 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 [ Time Frame: Baseline to 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.
• 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 [ Time Frame: Baseline to 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.
• Change from baseline in the modified Urticaria Activity Score (UAS7) at Week 24 [ Time Frame: Baseline to 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.
• Change from baseline in the modified itch severity score over 7 days (ISS7) at Week&nbsp;24 [ Time Frame: Baseline to Week 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.
• Change from baseline in the modified Hive Severity Score over 7 days (HSS7) at Week 24 [ Time Frame: Baseline to 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.

Original Secondary Outcome Measures (submitted: August 31, 2022)

• Incidence of treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs) [ Time Frame: Baseline to Week 36 ]
  Incidence of treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs).
• Incidence of anti-drug antibodies (ADA) to dupilumab over time [ Time Frame: Baseline to Week 36 ]
  Incidence of anti-drug antibodies (ADA) to dupilumab over time.
• 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 [ Time Frame: Baseline to 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.
• 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 [ Time Frame: Baseline to Week 24 ]
  The IDQOL is a validated questionnaire designed for use in children aged <4 years. The C-DLQI total score ranges 0 to 30. The higher the score, the greater the impact is on the child's HRQoL.
• Only for participants with CSU: Change from baseline in the modified Urticaria Activity Score (UAS7) at Week 24 [ Time Frame: Baseline to 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.
• Only for participants with CSU: Change from baseline in the modified itch severity score over 7 days (ISS7) at Week 24 [ Time Frame: Baseline to Week 24 ]
  ISS7 ranges from 0 (absent) to 3 (intense).
• Only for participants with CSU: Change from baseline in the modified Hive Severity Score over 7 days (HSS7) at Week 24 [ Time Frame: Baseline to Week 24 ]
  HSS7 ranges from 0 (absent) to 3 (intense, (>30 wheals/24 hours or large confluent areas of wheals).
• Only for participants with CICU: Proportion of participants with negative ice cube provocation test at Week 24 [ Time Frame: Week 24 ]
• Only for participants with CICU: Change from baseline in Wheal Intensity Likert Scale at Week 24 [ Time Frame: Baseline to Week 24 ]
  The Wheal Intensity Likert Scale is a clinician-reported outcome measure comprised of a single item assessing the intensity of patients' cutaneous reaction rated as follows: 0 = no wheals; 1 = numerous small, non-coalescent wheals; 2 = a large, regular, slightly edematous, coalescent wheal; 3 = a large and moderately edematous wheal; 4 = a large, regular, and significantly edematous wheal without pseudopodia; and 5 = a large, very edematous wheal with pseudopodia.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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)
• Official Title: 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)
• Brief Summary: 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Chronic Spontaneous Urticaria

Intervention

Drug: Dupilumab

Injection solution Subcutaneous

Other Name: Dupixent

Study Arms

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

Intervention: Drug: Dupilumab

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: August 31, 2022): 24
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: July 7, 2025
• Estimated Primary Completion Date: March 1, 2025 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years to 11 Years (Child)
• Accepts Healthy Volunteers: No

Contacts

Contact: Trial Transparency email recommended (Toll free number for US & Canada); 800-633-1610 ext option 6; Contact-US@sanofi.com

• Listed Location Countries: Canada, Japan, United States

Administrative Information

• NCT Number: NCT05526521
• Other Study ID Numbers: PKM16982; U1111-1266-5669 ( Registry Identifier: ICTRP ); 2022-000260-22 ( EudraCT Number )
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Yes
• 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

• Current Responsible Party: Sanofi
• Original Responsible Party: Same as current
• Current Study Sponsor: Sanofi
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Sanofi
• Verification Date: March 2024