24 Weeks Double-blind Randomized Placebo-controlled Trial to Evaluate Efficacy, PK, Safety of LOU064 in Adolescents (12 - <18) With CSU and Inadequate Response to H1-antihistamine Followed by Optional 3 Years Open-label Extension and an Optional 3 Years Safety Long-term Treatment-free Follow-up
LOU064 (blinded): LOU064 (blinded) active treatment
placebo: matching active drug
Study summary
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
Sex
ALL
Min age
12 Years
Max age
17 Years
Healthy volunteers
No
Key Inclusion Criteria:
* 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)
* Diagnosis of CSU inadequately controlled by second-generation H1-AH at the time of randomization defined as:
* 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)
* Documentation of hives within three months before randomization (either at screening and/or at randomization; or documented in the participants' medical history)
Key Exclusion criteria:
* 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
* History or current hepatic disease
* Evidence of clinically significant cardiovascular, neurological, psychiatric, pulmonary, renal, hepatic, endocrine, metabolic, hematological disorders, gastrointestinal disease or immunodeficiency that, in the investigator's opinion, would compromise the safety of the participant, interfere with the interpretation of the study results or otherwise preclude participation or protocol adherence of the participant
* History of hypersensitivity to any of the study drugs or its excipients or to drugs of similar chemical classes
* Participants having a clearly defined predominant or sole trigger of their chronic urticaria (chronic inducible urticaria) including urticaria factitia (symptomatic dermographism), cold-, heat-, solar-, pressure-, delayed pressure-, aquagenic-, cholinergic-, or contact-urticaria
* Other diseases with symptoms of urticaria or angioedema, including but not limited to urticaria vasculitis, urticaria pigmentosa, erythema multiforme, mastocytosis, hereditary angioedema, or drug-induced urticaria
* Any other skin disease associated with chronic itching that might influence in the investigator's opinion the study evaluations and results, e.g., atopic dermatitis, bullous pemphigoid, dermatitis herpetiformis, senile pruritus or psoriasis
Other protocol-defined inclusion/exclusion criteria may apply.
Primary outcome measure(s)
Change from baseline in UAS7 — Baseline, week 12 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 — Baseline, Week 12 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 — Baseline, Week 12 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.
Trial sites (50)
Facility
City
Region
Status
Kern Research
Bakersfield
California
Allergy and Asthma Medical Group and Research Center
San Diego
California
Pediatric Dermatology of Miami at the Pediatric CoE
Miami
Florida
Treasure Valley Medical Research
Boise
Idaho
Endeavor Health
Glenview
Illinois
Allergy and Asthma Specialist P S C
Owensboro
Kentucky
Toledo Institute of Clinical Research
Toledo
Ohio
Allergy Asthma and Clinical Research
Oklahoma City
Oklahoma
Allergy and Clinical Immunology Associates
Pittsburgh
Pennsylvania
RFSA Dermatology
San Antonio
Texas
Allergy Associates of Utah
Sandy City
Utah
Novartis Investigative Site
CABA
Buenos Aires
Novartis Investigative Site
Rosario
Santa Fe Province
Novartis Investigative Site
CABA
Argentina
Novartis Investigative Site
San Miguel de Tucumán
Argentina
Novartis Investigative Site
Montreal
Quebec
Novartis Investigative Site
Santiago
Santiago Metropolitan
Novartis Investigative Site
Guangzhou
Guangdong
Novartis Investigative Site
Chengdu
Sichuan
Novartis Investigative Site
Beijing
China
Novartis Investigative Site
Beijing
China
Novartis Investigative Site
Frankfurt am Main
Hesse
Novartis Investigative Site
Berlin
Germany
Novartis Investigative Site
Tübingen
Germany
Novartis Investigative Site
Hong Kong
Hong Kong
Novartis Investigative Site
Hong Kong
Hong Kong
Novartis Investigative Site
Florence
FI
Novartis Investigative Site
Pavia
PV
Novartis Investigative Site
Siena
SI
Novartis Investigative Site
Kitakyushu
Fukuoka
Novartis Investigative Site
Kamimashi-gun
Kumamoto
Novartis Investigative Site
Sakai
Osaka
Novartis Investigative Site
Izumo
Shimane
Novartis Investigative Site
Itabashi-ku
Tokyo
Novartis Investigative Site
Kuching
Sarawak
Novartis Investigative Site
Utrecht
Netherlands
Novartis Investigative Site
Lodz
Poland
Novartis Investigative Site
Pretoria
Gauteng
Novartis Investigative Site
Cape Town
South Africa
Novartis Investigative Site
Esplugues
Barcelona
+ 10 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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