Clinical trial NCT05526521: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Chronic Spontaneous Urticaria
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2022-09-02
Enrollment: 15
Sex: ALL
Minimum age (years): 2
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Concentration of dupilumab in serum over time including Ctrough at Week 12 and Week 24
timeFrame: Week 12 and Week 24
Secondary outcomes:
measure: Incidence of treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs)
timeFrame: Baseline to Week 36
measure: Incidence of anti-drug antibodies (ADA) to dupilumab over time
timeFrame: Baseline to Week 36
measure: 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
timeFrame: Baseline to Week 24
measure: 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
timeFrame: Baseline to Week 24
measure: Change from baseline in the modified Urticaria Activity Score (UAS7) at Week 24
timeFrame: Baseline to Week 24
measure: Change from baseline in the modified itch severity score over 7 days (ISS7) at Week&nbsp;24
timeFrame: Baseline to Week 24
measure: Change from baseline in the modified Hive Severity Score over 7 days (HSS7) at Week 24
timeFrame: Baseline to Week 24
Other outcomes:
Locations:
city: Coral Gables
country: United States
facility: Pediatric Dermatology of Miami Site Number : 8400015
state: Florida
city: Boise
country: United States
facility: Treasure Valley Medical Research Site Number : 8400019
state: Idaho
city: Saint Louis
country: United States
facility: Washington University School of Medicine- Site Number : 8400004
state: Missouri
city: Hawthorne
country: United States
facility: Boston Childrens Health Physicians Site Number : 8400017
state: New York
city: New York
country: United States
facility: Columbia University Irving Medical Center Site Number : 8400003
state: New York
city: Akron
country: United States
facility: Childrens Hospital Medical Center of Akron- Site Number : 8400020
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400001
state: Ohio
city: Tulsa
country: United States
facility: Vital Prospects Clinical Research Institute, P.C.- Site Number : 8400002
state: Oklahoma
city: Nashville
country: United States
facility: Monroe Carell Jr. Childrens Hospital at Vanderbilt- Site Number : 8400005
state: Tennessee
city: Calgary
country: Canada
facility: Investigational Site Number : 1240009
state: Alberta
city: Edmonton
country: Canada
facility: Investigational Site Number : 1240010
state: Alberta
city: Hamilton
country: Canada
facility: Investigational Site Number : 1240007
state: Ontario
city: Montreal
country: Canada
facility: Investigational Site Number : 1240001
state: Quebec
city: Yokohama-shi
country: Japan
facility: Investigational Site Number : 3920001
state: Kanagawa
city: Tsu-shi
country: Japan
facility: Investigational Site Number : 3920002
state: Mie
Study officials:
References: