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: August 28, 2023
• Sponsor: Sanofi
• Information provided by (Responsible Party): Sanofi

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Chronic Spontaneous Urticaria	Drug: Dupilumab	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 24 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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)
• Actual Study Start Date: August 25, 2022
• Estimated Primary Completion Date: March 1, 2025
• Estimated Study Completion Date: July 7, 2025

Arms and Interventions

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 Name: Dupixent

Outcome Measures

Primary Outcome Measures :

1. 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.

Secondary Outcome Measures :

1. 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).
2. 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.
3. 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.
4. 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.
5. 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.
6. 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.
7. 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.

Eligibility Criteria

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

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.

Contacts and Locations

Contacts

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

Locations

United States, Idaho

The Allergy Group-Site Number:8400019; Recruiting

Boise, Idaho, United States, 83706

United States, Missouri

Washington University School of Medicine-Site Number:8400004; Recruiting

Saint Louis, Missouri, United States, 63110

United States, New York

Columbia University Irving Medical Center-Site Number:8400003; Recruiting

New York, New York, United States, 10032

United States, Ohio

Cincinnati Children's Hospital Medical Center - PIN-Site Number:8400001; Recruiting

Cincinnati, Ohio, United States, 45229-3039

United States, Oklahoma

Vital Prospects Clinical Research Institute, P.C.-Site Number:8400002; Recruiting

Tulsa, Oklahoma, United States, 74136

Canada, Alberta

Investigational Site Number :1240009; Recruiting

Calgary, Alberta, Canada, T2W 4X9

Investigational Site Number :1240010; Recruiting

Edmonton, Alberta, Canada, T5J 3S9

Canada, Manitoba

Investigational Site Number :1240006; Recruiting

Winnipeg, Manitoba, Canada, R3A 1M3

Canada, Ontario

Investigational Site Number :1240007; Recruiting

Hamilton, Ontario, Canada, L8S1G5

Canada, Quebec

Investigational Site Number :1240001; Recruiting

Montreal, Quebec, Canada, H4A 3J1

Japan

Investigational Site Number :3920001; Recruiting

Yokohama-shi, Kanagawa, Japan, 232-8555

Investigational Site Number :3920002; Recruiting

Tsu-shi, Mie, Japan, 514-0125

Sponsors and Collaborators

Sanofi

More Information

• Responsible Party: Sanofi
• ClinicalTrials.gov Identifier: NCT05526521
• Other Study ID Numbers: PKM16982; U1111-1266-5669 ( Registry Identifier: ICTRP ); 2022-000260-22 ( EudraCT Number )
• First Posted: September 2, 2022
• Last Update Posted: August 28, 2023
• Last Verified: August 25, 2023

Individual Participant Data (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

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

Additional relevant MeSH terms:

Urticaria; Chronic Urticaria; Skin Diseases, Vascular; Skin Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes