Last Update: May 07, 2024
A Double-blind, Randomized, Placebo-controlled Trial to Evaluate the Efficacy, Pharmacokinetics and Safety of Remibrutinib (LOU064) for 24 Weeks in Adolescents From 12 to Less Than 18 Years of Age With Chronic Spontaneous Urticaria Inadequately Controlled by H1-antihistamines Followed by an Optional Open-label Extension for up to Another 3 Years and an Optional Safety Long-term Treatment-free Follow-up Period for up to an Additional 3 Years
ClinicalTrials.gov Identifier:
Novartis Reference Number:CLOU064F12301
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

The purpose of this trial is:

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
to collect long-term efficacy, safety and tolerability data on remibrutinib in adolescents after having completed 24 weeks of treatment
to collect safety data in this population for up to three years after the last dose of study treatment

This trial consists of 3 different periods:

the "core period", which is randomized and double-blind, during which 2/3 participants will receive remibrutinib and 1/3 will receive placebo for 24 weeks. Total duration: approximately 32 weeks (10 site visits).
an optional "open-label extension (OLE) period" proposed to all participants who completed 24 weeks of treatment of the "core period" and all scheduled assessments planned at week 24 visit . Depending on their CSU symptoms (as assessed by the doctor), participants will either receive remibrutinib for 24 weeks, or enter an observational treatment-free period for 1 year. If the CSU symptoms return during the observational period, the participants can switch to the treatment period at any time (decided by the doctor). At the end of the 24-week treatment period, if CSU is controlled, participants will enter the 1-year observational period, otherwise, they can continue with another cycle of 24-week remibrutinib treatment. The number of remibrutinib treatment or observational cycles will be limited to 6 times each. Total duration: from 1 year to approximately 3 years, and number of visits: from 3 to 15 (depending on the CSU symptoms).
an optional "long-term treatment-free follow-up period" proposed to all participants who completed at least 4 months treatment in the "OLE period". No treatment will be given. Duration: 3 years with 1 site visit and up to 4 phone call follow-up visits.

The primary clinical question of interest is what is the effect of remibrutinib treatment versus placebo on the change from baseline in UAS7, ISS7 and HSS7 scores after 12 weeks of treatment

Chronic Spontaneous Urticaria
Phase 3
Recruiting
84
Jul 11, 2023
Jan 22, 2032
All
12 Years - 17 Years (Child)

Interventions

Drug

LOU064 (blinded)

LOU064 (blinded) active treatment
Drug

placebo

matching active drug

Eligibility Criteria

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.

Study Location

Novartis Investigative Site

Recruiting

Rosario,Santa Fe,2000,Argentina

Novartis Investigative Site

Recruiting

Caba,Buenos Aires,C1181ACH,Argentina

Novartis Investigative Site

Recruiting

Caba,Buenos Aires,C1414AIF,Argentina

Novartis Investigative Site

Recruiting

Caba,Buenos Aires,C1425BEN,Argentina

Novartis Investigative Site

Recruiting

Montreal,Quebec,H3H 1P3,Canada

Novartis Investigative Site

Recruiting

Santiago,8420383,Chile

Novartis Investigative Site

Recruiting

Chengdu,Sichuan,610041,China

Novartis Investigative Site

Recruiting

Beijing,100050,China

Novartis Investigative Site

Recruiting

Beijing,100069,China

Novartis Investigative Site

Recruiting

Guangdong,Guangzhou,510091,China

Novartis Investigative Site

Recruiting

Mainz,55131,Germany

Novartis Investigative Site

Recruiting

Muenster,48149,Germany

Novartis Investigative Site

Recruiting

Berlin,13353,Germany

Novartis Investigative Site

Recruiting

Frankfurt,60590,Germany

Novartis Investigative Site

Recruiting

Tuebingen,72076,Germany

Novartis Investigative Site

Recruiting

Hong Kong,Hong Kong

Novartis Investigative Site

Recruiting

Bari,70126,Italy

Novartis Investigative Site

Recruiting

Napoli,80138,Italy

Novartis Investigative Site

Recruiting

Firenze,FI,50141,Italy

Novartis Investigative Site

Recruiting

Trieste,TS,34137,Italy

Novartis Investigative Site

Recruiting

Parma,PR,43100,Italy

Novartis Investigative Site

Recruiting

Pavia,PV,27100,Italy

Novartis Investigative Site

Recruiting

Itabashi-ku,Tokyo,173-8610,Japan

Novartis Investigative Site

Recruiting

Kamimashi-gun,Kumamoto,861-3106,Japan

Novartis Investigative Site

Recruiting

Kuching,Sarawak,93586,Malaysia

Novartis Investigative Site

Recruiting

Utrecht,3584 CX,Netherlands

Novartis Investigative Site

Recruiting

Deventer,7416 SE,Netherlands

Novartis Investigative Site

Recruiting

Warszawa,02-962,Poland

Novartis Investigative Site

Recruiting

Olsztyn,10-045,Poland

Novartis Investigative Site

Recruiting

Lodz,90-436,Poland

Novartis Investigative Site

Recruiting

Singapore,119074,Singapore

Novartis Investigative Site

Recruiting

Singapore,229899,Singapore

Novartis Investigative Site

Recruiting

Esplugues De Llobregat,Barcelona,08950,Spain

Novartis Investigative Site

Recruiting

Barcelona,Catalunya,08035,Spain

Novartis Investigative Site

Recruiting

Bangkoknoi,Bangkok,10700,Thailand

Novartis Investigative Site

Recruiting

Bangkok,10330,Thailand

Novartis Investigative Site

Recruiting

Istanbul,34093,Turkey

Novartis Investigative Site

Recruiting

Adana,01050,Turkey

Novartis Investigative Site

Recruiting

Ankara,06100,Turkey

Northshore University Health System .

Recruiting

Glenview,Madeline Snedden (847-657-5959) email: [email protected] -- Giselle Mosnaim,60077 - Illinois,United States

Allergy and Asthma Specialist P S C Main Center

Recruiting

Owensboro,Andrea Arthur (270-684-6144) email: [email protected] -- Lee Clore,42301 - Kentucky,United States

Pediatric Dermatology of Miami at the Pediatric CoE

Recruiting

Coral Gables,Karla Olivas (305-667-3152) email: [email protected] -- Mercedes E Gonzalez,33134 - Florida,United States

Allergy and Clinical Immunology Ass

Recruiting

Pittsburgh,Sherry Knoblock (412-833-4051) email: [email protected] -- Michael Palumbo,15241 - Pennsylvania,United States

RFSA Dermatology

Recruiting

San Antonio,Lindsey Finklea,78213 - Texas,United States

Allergy Associates of Utah

Recruiting

Murray,(801-263-8700) -- Andrew Smith,84107 - Utah,United States

Toledo Institute of Clinical Research

Recruiting

Toledo,Faheem Husain (419-843-8815) email: [email protected] -- Syed Rehman,43617 - Ohio,United States

Allergy Asthma and Clinical Research

Recruiting

Oklahoma City,Gabriella Day (405-752-0393) email: [email protected] -- Martha Tarpay,73120 - Oklahoma,United States

Treasure Valley Medical Research

Recruiting

Boise,Margaret Tracy email: [email protected] -- Neetu Talreja,83706 - Idaho,United States

Kern Research

Recruiting

Bakersfield,Andrea Estrada (661-864-7710) email: [email protected] -- Eric Boren,93301 - California,United States

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Novartis Pharmaceuticals

Novartis Pharmaceuticals