RecruitingPhase 3NCT05677451

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

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


Sponsor

Novartis Pharmaceuticals

Enrollment

100 participants

Start Date

Jul 11, 2023

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 12 YearsMax Age: 17 Years

Inclusion Criteria6

  • 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)

Exclusion Criteria10

  • 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

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Interventions

DRUGLOU064 (blinded)

LOU064 (blinded) active treatment

DRUGplacebo

matching active drug


Locations(63)

Kern Research

Bakersfield, California, United States

Allergy and Asthma Medical Group and Research Center

San Diego, California, United States

Pediatric Dermatology of Miami at the Pediatric CoE

Coral Gables, Florida, United States

Treasure Valley Medical Research

Boise, Idaho, United States

Endeavor Health

Glenview, Illinois, United States

Allergy and Asthma Specialist P S C

Owensboro, Kentucky, United States

Toledo Institute of Clinical Research

Toledo, Ohio, United States

Allergy Asthma and Clinical Research

Oklahoma City, Oklahoma, United States

Allergy and Clinical Immunology Associates

Pittsburgh, Pennsylvania, United States

RFSA Dermatology

San Antonio, Texas, United States

Allergy Associates of Utah

Sandy City, Utah, United States

Seattle Allergy and Asthma Rsch

Seattle, Washington, United States

Novartis Investigative Site

CABA, Buenos Aires, Argentina

Novartis Investigative Site

CABA, Buenos Aires, Argentina

Novartis Investigative Site

CABA, Buenos Aires, Argentina

Novartis Investigative Site

Rosario, Santa Fe Province, Argentina

Novartis Investigative Site

Montreal, Quebec, Canada

Novartis Investigative Site

Santiago, Chile

Novartis Investigative Site

Guangdong, Guangzhou, China

Novartis Investigative Site

Chengdu, Sichuan, China

Novartis Investigative Site

Beijing, China

Novartis Investigative Site

Beijing, China

Novartis Investigative Site

Frankfurt am Main, Hesse, Germany

Novartis Investigative Site

Berlin, Germany

Novartis Investigative Site

Mainz, Germany

Novartis Investigative Site

Münster, Germany

Novartis Investigative Site

Tübingen, Germany

Novartis Investigative Site

Hong Kong, Hong Kong

Novartis Investigative Site

Pokfulam, Hong Kong

Novartis Investigative Site

Florence, FI, Italy

Novartis Investigative Site

Parma, PR, Italy

Novartis Investigative Site

Pavia, PV, Italy

Novartis Investigative Site

Siena, SI, Italy

Novartis Investigative Site

Trieste, TS, Italy

Novartis Investigative Site

Bari, Italy

Novartis Investigative Site

Naples, Italy

Novartis Investigative Site

Kitakyushu, Fukuoka, Japan

Novartis Investigative Site

Kamimashi-gun, Kumamoto, Japan

Novartis Investigative Site

Sakai, Osaka, Japan

Novartis Investigative Site

Izumo, Shimane, Japan

Novartis Investigative Site

Itabashi-ku, Tokyo, Japan

Novartis Investigative Site

Kuching, Sarawak, Malaysia

Novartis Investigative Site

Deventer, Netherlands

Novartis Investigative Site

Utrecht, Netherlands

Novartis Investigative Site

Lodz, Poland

Novartis Investigative Site

Olsztyn, Poland

Novartis Investigative Site

Warsaw, Poland

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Singapore, Singapore

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Singapore, Singapore

Novartis Investigative Site

Pretoria, Gauteng, South Africa

Novartis Investigative Site

Cape Town, Western Cape, South Africa

Novartis Investigative Site

Esplugues de Llobregat, Barcelona, Spain

Novartis Investigative Site

Barcelona, Catalonia, Spain

Novartis Investigative Site

Valencia, Valencia, Spain

Novartis Investigative Site

Songkhla, Hat Yai, Thailand

Novartis Investigative Site

Bangkok, Thailand

Novartis Investigative Site

Bangkok, Thailand

Novartis Investigative Site

Adana, Turkey (Türkiye)

Novartis Investigative Site

Ankara, Turkey (Türkiye)

Novartis Investigative Site

Istanbul, Turkey (Türkiye)

Novartis Investigative Site

Peterborough, Cambridgeshire, United Kingdom

Novartis Investigative Site

Manchester, United Kingdom

Novartis Investigative Site

Southampton, United Kingdom

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