Back to resultsA 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)
Primary Purpose
Chronic Spontaneous Urticaria
Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Dupilumab
Sponsored by
About this trial
This is an interventional treatment trial for Chronic Spontaneous Urticaria
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 a documented diagnosis of CSU or CICU >6 months prior to screening visit.
- Participants with CSU (characterized by recurrent itchy wheals with or without angioedema for >6 consecutive weeks) who remain symptomatic at the time of screening despite regular H1-antihistamine treatment.
- Participants with CICU (characterized by recurrent itchy wheals with or without angioedema due to cold for >6 weeks) who remain symptomatic at the time of screening despite the regular or as-needed use of H1-antihistamine or appropriate preventive measures.
- Participants with CICU with a positive ice cube provocation test, presenting with a confluent hive/wheal on the exposed skin area, at the screening visit.
- 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/CICU.
- Presence of skin morbidities other than CSU/CICU that may interfere with the assessment of the study outcomes.
- Participants with a concurrent diagnosis of both CSU and CICU.
- 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.
Sites / Locations
- The Allergy Group-Site Number:8400019Recruiting
- Washington University School of Medicine-Site Number:8400004Recruiting
- Columbia University Irving Medical Center-Site Number:8400003Recruiting
- Cincinnati Children's Hospital Medical Center - PIN-Site Number:8400001Recruiting
- Vital Prospects Clinical Research Institute, P.C.-Site Number:8400002Recruiting
- Investigational Site Number :1240009Recruiting
- Investigational Site Number :1240010Recruiting
- Investigational Site Number :1240006Recruiting
- Investigational Site Number :1240007Recruiting
- Investigational Site Number :1240001Recruiting
- Investigational Site Number :3920001Recruiting
- Investigational Site Number :3920002Recruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Dupilumab
Arm Description
Administered subcutaneously (SC) every 4 weeks (Q4W) or every 2 weeks (Q2W) with or without an initial loading dose based on weight and age
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Incidence of treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs)
Incidence of treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs).
Incidence of anti-drug antibodies (ADA) to dupilumab over time
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
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
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
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 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
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05526521
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)
Study Type
Interventional
2. Study Status
Record Verification Date
August 25, 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 25, 2022 (Actual)
Primary Completion Date
March 1, 2025 (Anticipated)
Study Completion Date
July 7, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sanofi
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chronic Spontaneous Urticaria
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
24 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Dupilumab
Arm Type
Experimental
Arm Description
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 Type
Drug
Intervention Name(s)
Dupilumab
Other Intervention Name(s)
Dupixent
Intervention Description
Injection solution Subcutaneous
Primary Outcome Measure Information:
Title
Concentration of dupilumab in serum over time including Ctrough at Week 12 and Week 24
Description
Concentration of dupilumab in serum over time including Ctrough at Week 12 and Week 24.
Time Frame
Week 12 and Week 24
Secondary Outcome Measure Information:
Title
Incidence of treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs)
Description
Incidence of treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs).
Time Frame
Baseline to Week 36
Title
Incidence of anti-drug antibodies (ADA) to dupilumab over time
Description
Incidence of anti-drug antibodies (ADA) to dupilumab over time.
Time Frame
Baseline to Week 36
Title
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
Description
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.
Time Frame
Baseline to Week 24
Title
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
Description
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.
Time Frame
Baseline to Week 24
Title
Change from baseline in the modified Urticaria Activity Score (UAS7) at Week 24
Description
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.
Time Frame
Baseline to Week 24
Title
Change from baseline in the modified itch severity score over 7 days (ISS7) at Week 24
Description
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.
Time Frame
Baseline to Week 24
Title
Change from baseline in the modified Hive Severity Score over 7 days (HSS7) at Week 24
Description
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.
Time Frame
Baseline to Week 24
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Trial Transparency email recommended (Toll free number for US & Canada)
Phone
800-633-1610
Ext
option 6
Email
Contact-US@sanofi.com
Facility Information:
Facility Name
The Allergy Group-Site Number:8400019
City
Boise
State/Province
Idaho
ZIP/Postal Code
83706
Country
United States
Individual Site Status
Recruiting
Facility Name
Washington University School of Medicine-Site Number:8400004
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Individual Site Status
Recruiting
Facility Name
Columbia University Irving Medical Center-Site Number:8400003
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Individual Site Status
Recruiting
Facility Name
Cincinnati Children's Hospital Medical Center - PIN-Site Number:8400001
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229-3039
Country
United States
Individual Site Status
Recruiting
Facility Name
Vital Prospects Clinical Research Institute, P.C.-Site Number:8400002
City
Tulsa
State/Province
Oklahoma
ZIP/Postal Code
74136
Country
United States
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :1240009
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2W 4X9
Country
Canada
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :1240010
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T5J 3S9
Country
Canada
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :1240006
City
Winnipeg
State/Province
Manitoba
ZIP/Postal Code
R3A 1M3
Country
Canada
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :1240007
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8S1G5
Country
Canada
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :1240001
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H4A 3J1
Country
Canada
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :3920001
City
Yokohama-shi
State/Province
Kanagawa
ZIP/Postal Code
232-8555
Country
Japan
Individual Site Status
Recruiting
Facility Name
Investigational Site Number :3920002
City
Tsu-shi
State/Province
Mie
ZIP/Postal Code
514-0125
Country
Japan
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing 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
Learn more about this trial
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)
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