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) or chronic inducible cold urticaria (CICU)

JRCT ID: jRCT2031220733

Registered date:26/03/2023

Basic Information

• Recruitment status: Recruiting

Health condition(s) or Problem(s) studied

Chronic Cold Urticaria
Chronic Spontaneous Urticaria

• Date of first enrollment: 30/03/2023
• Target sample size: 24
• Countries of recruitment: Canada,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

Drug: Dupilumab
Pharmaceutical form: Injection solution Subcutaneous
Other Name: Dupixent

Outcome(s)

Primary Outcome

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

Secondary Outcome

1.Incidence of treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs)
[Time Frame baseline:Baseline to Week 36]
2.Incidence of anti-drug antibodies (ADA) to dupilumab over time
[Time Frame baseline:Baseline to Week 36]
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: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: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.
5.Only for participants with CSU: Change from baseline in the modified Urticaria Activity Score (UAS7) at Week 24
[Time Frame baseline: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.Only for participants with CSU: Change from baseline in the modified itch severity score over 7 days (ISS7) at Week 24
[Time Frame baseline:Baseline to Week 24]
ISS7 ranges from 0 (absent) to 3 (intense).
7.Only for participants with CSU: Change from baseline in the modified Hive Severity Score over 7 days (HSS7) at Week 24
[Time Frame baseline:Baseline to Week 24]
HSS7 ranges from 0 (absent) to 3 (intense), (>30 wheals/24 hours or large confluent areas of wheals)
8.Only for participants with CICU: Proportion of participants with negative ice cube provocation test at Week 24
[Time Frame baseline:Week 24]
9.Only for participants with CICU: Change from baseline in Wheal Intensity Likert Scale at Week 24
[Time Frame baseline: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.

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: <= 11age old
• Gender: Both

Include 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.

Exclude criteria

Participants are excluded from the study if any of the following criteria apply:
Medical conditions
- 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.

Related Information

• Primary Sponsor: Tanaka Tomoyuki
• Secondary ID(s): NCT05526521,2022-000260-22