Last Update: Nov 19, 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:

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 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

Chronic Spontaneous Urticaria
Phase3
Recruiting
100
Jul 11, 2023
Feb 04, 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.

Novartis Investigative Site

Recruiting

Caba,Buenos Aires,C1425ben,Argentina

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Rosario,Santa Fe,2000,Argentina

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Caba,Buenos Aires,C1181ach,Argentina

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Caba,Buenos Aires,C1414aif,Argentina

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Montreal,Quebec,H4a 3j1,Canada

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Guangdong,Guangzhou,510091,China

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Chengdu,Sichuan,610041,China

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Beijing,100050,China

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Beijing,100069,China

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Berlin,13353,Germany

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Frankfurt,60590,Germany

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Mainz,55131,Germany

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Muenster,48149,Germany

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Tuebingen,72076,Germany

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Pokfulam,Hong Kong

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Napoli,80138,Italy

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Firenze,FI,50139,Italy

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Parma,PR,43100,Italy

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Pavia,PV,27100,Italy

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Siena,SI,53100,Italy

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Trieste,TS,34137,Italy

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Bari,70126,Italy

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Kitakyushu,Fukuoka,807-8556,Japan

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Kamimashi-gun,Kumamoto,861-3106,Japan

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Itabashi-ku,Tokyo,173-8610,Japan

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Kuching,Sarawak,93586,Malaysia

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Deventer,7416 se,Netherlands

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Utrecht,3584 cx,Netherlands

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Lodz,90-436,Poland

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Olsztyn,10-045,Poland

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Warszawa,02-962,Poland

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Singapore,119074,Singapore

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Singapore,229899,Singapore

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Pretoria,Gauteng,0009,South Africa

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Esplugues De Llobregat,Barcelona,08950,Spain

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Barcelona,Catalunya,08035,Spain

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Valencia,Comunidad Valenciana,46014,Spain

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Bangkoknoi,Bangkok,10700,Thailand

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Songkhla,Hat Yai,90110,Thailand

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Bangkok,10330,Thailand

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Ankara,06230,Turkey

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Istanbul,34093,Turkey

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Adana,01050,Turkey

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Peterborough,Cambridgeshire,Pe3 9gz,United Kingdom

Treasure Valley Medical Research

Recruiting

Boise,Idaho,83706,United States

Margaret Tracy
Neetu Talreja

Northshore University Health System

Recruiting

Glenview,Illinois,60077,United States

Giselle Mosnaim
Madeline Snedden

Allergy and Clinical Immunology Associates

Recruiting

Pittsburgh,Pennsylvania,15241,United States

Sherry Knoblock
Michael Palumbo

RFSA Dermatology

Recruiting

San Antonio,Texas,78213,United States

Lindsey Finklea

Toledo Institute of Clinical Research

Recruiting

Toledo,Ohio,43617,United States

Faheem Husain
Syed Rehman

Allergy and Asthma Specialist P S C

Recruiting

Owensboro,Kentucky,42301,United States

Andrea Arthur
Lee Clore

Kern Research

Recruiting

Bakersfield,California,93301,United States

Eric Boren
Mariya Mendoza

Allergy Associates of Utah

Recruiting

Sandy,Utah,84093,United States

Andrew Smith
Deann Sims

Allergy Asthma and Clinical Research

Recruiting

Oklahoma City,Oklahoma,73120,United States

Martha Tarpay

Pediatric Dermatology of Miami at the Pediatric CoE

Recruiting

Coral Gables,Florida,33134,United States

Karla Olivas
Mercedes E Gonzalez

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