About this trial
The purpose of this trial is:
1. to assess the efficacy, pharmacokinetics, and safety of remibrutinib vs. placebo in adolescents from 12 to \< 18 years of age suffering from chronic spontaneous urticaria inadequately controlled by H1-antihistamines 2. to collect long-term efficacy, safety and tolerability data on remibrutinib in adolescents after having completed 24 weeks of treatment 3. to collect safety data in this population for up to three years after the last dose of study treatment
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male and female adolescent participants aged >= 12 to < 18 years of age at the time of signing the informed consent
CSU duration for >= 6 months prior to screening (defined as the onset of CSU determined by the investigator based on all available supporting documentation)
The presence of itch and hives for ≥ 6 consecutive weeks prior to screening despite the use of second-generation H1-AH during this time period according to local treatment guidelines
UAS7 score (range 0 - 42) >= 16, ISS7 score (range 0 - 21) >= 6 and HSS7 score (range 0 - 21) >= 6 during the 7 days prior to randomization (Day 1)
Disqualifiers
Previous use of remibrutinib or other BTK inhibitors
Significant bleeding risk or coagulation disorders
History of gastrointestinal bleeding
Requirement for anti-platelet medication, except for acetylsalicylic acid up to 100 mg/d or clopidogrel up to 75 mg/d. The use of dual anti-platelet therapy (e.g., acetylsalicylic acid + clopidogrel) is prohibited
Trial design
Parallel
Treatments tested in this trial
LOU064 (blinded)
DrugLOU064 (blinded) active treatment
placebo
Drugmatching active drug
Treatment groups
Trial outcomes
Primary outcomes
Change from baseline in UAS7
The Urticaria Activity Score (UAS) is sum of the Hive Severity Score (HSS) and the Itch Severity Score (ISS). UAS7 is sum of the HSS7 and the ISS7 scores. Possible range of weekly UAS7 score is 0 to 42. Complete UAS7 response is UAS7 = 0. Negative change from baseline indicates improvement.
Change fron baseline in ISS7
Itch Severity Score (ISS) scale is 0 to 3. Score (ISS7) is derived by adding up average daily scores of 7 days preceding visit. Possible range of weekly score is therefore 0 to 21. Itch Severity Score scale: 0 - None 1 - Mild (minimal awareness, easily tolerated) 2 - Moderate (definite awareness, bothersome but tolerable) 3 - Severe (difficult to tolerate). Negative change from baseline indicates improvement.
Change from baseline in HSS7
Hives Severity Score (HSS) scale is 0 to 3. A weekly score (HSS7) is derived by adding up the average daily scores of the 7 days preceding the visit. Possible range of the weekly score is therefore 0 to 21. Hives Severity Score scale: 0 - None 1 - Mild (1-6 hives/12 hours) 2 - Moderate (7-12 hives/12 hours) 3 - Severe (\>12 hives/12 hours). Negative change from baseline indicates improvement.
Secondary outcomes
Cmax of remibrutinib
The maximum (peak) observed blood drug concentration after single dose administration
Tmax of remibrutinib
The time to reach maximum (peak) blood drug concentration after single dose administration
AUClast of remibrutinib
The Area Under the Curve (AUC) from pre-dose to the last measurable concentration sampling time
Absolute change from baseline in ISS7
Itch Severity Score (ISS) scale is 0 to 3. Score (ISS7) is derived by adding up average daily scores of 7 days preceding visit. Possible range of weekly score is therefore 0 to 21. Itch Severity Score scale: 0 - None 1 - Mild (minimal awareness, easily tolerated) 2 - Moderate (definite awareness, bothersome but tolerable) 3 - Severe (difficult to tolerate). Negative change from baseline indicates improvement.
Sponsors and contacts
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