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

NCT ID: NCT05677451

Last Updated: 2025-06-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-07-11

Study Completion Date

2032-03-30

Brief Summary

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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

Detailed Description

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This trial consists of 3 different periods:

1. 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).
2. 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).
3. 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

Conditions

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Chronic Spontaneous Urticaria

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Arm 1: LOU064 (blinded)

LOU064 (blinded) taken orally b.i.d. for 24 weeks, followed by LOU064 (open-label) taken orally b.i.d. for up to 6 cycles of 24 weeks.

Group Type EXPERIMENTAL

LOU064 (blinded)

Intervention Type DRUG

LOU064 (blinded) active treatment

Arm 2: LOU064 placebo (blinded)

LOU064 placebo (blinded) taken orally b.i.d. for 24 weeks (randomized in a 2:1 ratio arm 1: arm 2)

Group Type PLACEBO_COMPARATOR

placebo

Intervention Type DRUG

matching active drug

Interventions

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LOU064 (blinded)

LOU064 (blinded) active treatment

Intervention Type DRUG

placebo

matching active drug

Intervention Type DRUG

Other Intervention Names

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remibrutinib

Eligibility Criteria

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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)

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
Minimum Eligible Age

12 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Kern Research

Bakersfield, California, United States

Site Status RECRUITING

Allergy and Asthma Medical Group and Research Center

San Diego, California, United States

Site Status RECRUITING

Pediatric Dermatology of Miami at the Pediatric CoE

Coral Gables, Florida, United States

Site Status RECRUITING

Treasure Valley Medical Research

Boise, Idaho, United States

Site Status RECRUITING

Endeavor Health

Glenview, Illinois, United States

Site Status RECRUITING

Allergy and Asthma Specialist P S C

Owensboro, Kentucky, United States

Site Status RECRUITING

Toledo Institute of Clinical Research

Toledo, Ohio, United States

Site Status RECRUITING

Allergy Asthma and Clinical Research

Oklahoma City, Oklahoma, United States

Site Status RECRUITING

Allergy and Clinical Immunology Associates

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

RFSA Dermatology

San Antonio, Texas, United States

Site Status RECRUITING

Allergy Associates of Utah

Sandy City, Utah, United States

Site Status RECRUITING

Seattle Allergy and Asthma Rsch

Seattle, Washington, United States

Site Status WITHDRAWN

Novartis Investigative Site

CABA, Buenos Aires, Argentina

Site Status RECRUITING

Novartis Investigative Site

CABA, Buenos Aires, Argentina

Site Status RECRUITING

Novartis Investigative Site

CABA, Buenos Aires, Argentina

Site Status RECRUITING

Novartis Investigative Site

Rosario, Santa Fe Province, Argentina

Site Status RECRUITING

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Montreal, Quebec, Canada

Site Status RECRUITING

Novartis Investigative Site

Santiago, , Chile

Site Status ACTIVE_NOT_RECRUITING

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Guangdong, Guangzhou, China

Site Status RECRUITING

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Chengdu, Sichuan, China

Site Status RECRUITING

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Beijing, , China

Site Status RECRUITING

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Beijing, , China

Site Status RECRUITING

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Frankfurt am Main, Hesse, Germany

Site Status RECRUITING

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Berlin, , Germany

Site Status RECRUITING

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Mainz, , Germany

Site Status RECRUITING

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Münster, , Germany

Site Status RECRUITING

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Tübingen, , Germany

Site Status RECRUITING

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Hong Kong, , Hong Kong

Site Status RECRUITING

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Pokfulam, , Hong Kong

Site Status RECRUITING

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Florence, FI, Italy

Site Status RECRUITING

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Parma, PR, Italy

Site Status RECRUITING

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Pavia, PV, Italy

Site Status RECRUITING

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Siena, SI, Italy

Site Status RECRUITING

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Trieste, TS, Italy

Site Status RECRUITING

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Bari, , Italy

Site Status RECRUITING

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Napoli, , Italy

Site Status RECRUITING

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Kitakyushu, Fukuoka, Japan

Site Status RECRUITING

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Kamimashi-gun, Kumamoto, Japan

Site Status RECRUITING

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Sakai, Osaka, Japan

Site Status RECRUITING

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Izumo, Shimane, Japan

Site Status RECRUITING

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Itabashi-ku, Tokyo, Japan

Site Status RECRUITING

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Kuching, Sarawak, Malaysia

Site Status RECRUITING

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Deventer, , Netherlands

Site Status RECRUITING

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Utrecht, , Netherlands

Site Status RECRUITING

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Lodz, , Poland

Site Status RECRUITING

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Olsztyn, , Poland

Site Status RECRUITING

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Warsaw, , Poland

Site Status RECRUITING

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Singapore, , Singapore

Site Status RECRUITING

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Singapore, , Singapore

Site Status RECRUITING

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Pretoria, Gauteng, South Africa

Site Status RECRUITING

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Cape Town, Western Cape, South Africa

Site Status RECRUITING

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Esplugues de Llobregat, Barcelona, Spain

Site Status RECRUITING

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Barcelona, Catalonia, Spain

Site Status RECRUITING

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Valencia, Valencia, Spain

Site Status RECRUITING

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Songkhla, Hat Yai, Thailand

Site Status RECRUITING

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Bangkok, , Thailand

Site Status RECRUITING

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Bangkok, , Thailand

Site Status RECRUITING

Novartis Investigative Site

Adana, , Turkey (Türkiye)

Site Status RECRUITING

Novartis Investigative Site

Ankara, , Turkey (Türkiye)

Site Status RECRUITING

Novartis Investigative Site

Istanbul, , Turkey (Türkiye)

Site Status RECRUITING

Novartis Investigative Site

Peterborough, Cambridgeshire, United Kingdom

Site Status RECRUITING

Novartis Investigative Site

Manchester, , United Kingdom

Site Status RECRUITING

Novartis Investigative Site

Southampton, , United Kingdom

Site Status RECRUITING

Countries

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United States Argentina Canada Chile China Germany Hong Kong Italy Japan Malaysia Netherlands Poland Singapore South Africa Spain Thailand Turkey (Türkiye) United Kingdom

Central Contacts

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Novartis Pharmaceuticals

Role: CONTACT

1-888-669-6682

Novartis Pharmaceuticals

Role: CONTACT

+41613241111

Facility Contacts

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Federico Navarro

Role: primary

661-864-7710

Mary Vales

Role: primary

858-268-2368

Karla Olivas

Role: primary

305-667-3152

Summer Reames

Role: primary

Madeline Snedden

Role: primary

847-657-5959

Andrea Arthur

Role: primary

270-684-6144

Faheem Husain

Role: primary

419-843-8815

Role: primary

405-752-0393

Sherry Knoblock

Role: primary

412-833-4051

Deann Sims

Role: primary

801-263-8700

Other Identifiers

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2022-502159-78-00

Identifier Type: OTHER

Identifier Source: secondary_id

CLOU064F12301

Identifier Type: -

Identifier Source: org_study_id

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