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Dubbelblinde, gerandomiseerde, placebogecontroleerde studie van 24 weken om de werkzaamheid, farmacokinetiek en veiligheid van LOU064 bij adolescenten te evalueren (12 -

14 oktober 2026 bijgewerkt door: Novartis Pharmaceuticals

Een dubbelblinde, gerandomiseerde, placebogecontroleerde studie om de werkzaamheid, farmacokinetiek en veiligheid van remibrutinib (LOU064) gedurende 24 weken te evalueren bij adolescenten van 12 tot jonger dan 18 jaar met chronische spontane urticaria die onvoldoende onder controle worden gehouden door H1-antihistaminica, gevolgd door een optionele open-label verlenging voor nog eens 3 jaar en een optionele veiligheid Langdurige behandelingsvrije follow-upperiode voor nog eens 3 jaar

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

Studie Overzicht

Toestand

Actief, niet wervend

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

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

Studietype

Ingrijpend

Inschrijving (Werkelijk)

100

Fase

  • Fase 3

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

  • Argentinië
      • CABA, Argentinië, C1181ACH
        • Novartis Investigative Site
      • San Miguel de Tucumán, Argentinië, T4000AXL
        • Novartis Investigative Site
    • Buenos Aires
      • CABA, Buenos Aires, Argentinië, C1414AIF
        • Novartis Investigative Site
    • Santa Fe Province
      • Rosario, Santa Fe Province, Argentinië, S2000DEJ
        • Novartis Investigative Site
  • Canada
    • Quebec
      • Montreal, Quebec, Canada, H4A 3J1
        • Novartis Investigative Site
  • Chili
    • Santiago Metropolitan
      • Santiago, Santiago Metropolitan, Chili, 8420383
        • Novartis Investigative Site
  • China
      • Beijing, China, 100050
        • Novartis Investigative Site
      • Beijing, China, 100069
        • Novartis Investigative Site
    • Guangdong
      • Guangzhou, Guangdong, China, 510091
        • Novartis Investigative Site
    • Sichuan
      • Chengdu, Sichuan, China, 610041
        • Novartis Investigative Site
  • Duitsland
      • Berlin, Duitsland, 13353
        • Novartis Investigative Site
      • Tübingen, Duitsland, 72076
        • Novartis Investigative Site
    • Hesse
      • Frankfurt am Main, Hesse, Duitsland, 60590
        • Novartis Investigative Site
  • Hongkong
    • Hong Kong
      • Hong Kong, Hong Kong, Hongkong, 999077
        • Novartis Investigative Site
  • Italië
    • FI
      • Florence, FI, Italië, 50139
        • Novartis Investigative Site
    • PV
      • Pavia, PV, Italië, 27100
        • Novartis Investigative Site
    • SI
      • Siena, SI, Italië, 53100
        • Novartis Investigative Site
  • Japan
    • Fukuoka
      • Kitakyushu, Fukuoka, Japan, 8078556
        • Novartis Investigative Site
    • Kumamoto
      • Kamimashi-gun, Kumamoto, Japan, 8613106
        • Novartis Investigative Site
    • Osaka
      • Sakai, Osaka, Japan, 5938324
        • Novartis Investigative Site
    • Shimane
      • Izumo, Shimane, Japan, 6938501
        • Novartis Investigative Site
    • Tokyo
      • tabashi City, Tokyo, Japan, 1738610
        • Novartis Investigative Site
  • Maleisië
    • Sarawak
      • Kuching, Sarawak, Maleisië, 93586
        • Novartis Investigative Site
  • Nederland
      • Utrecht, Nederland, 3584 CX
        • Novartis Investigative Site
  • Polen
      • Lodz, Polen, 90-436
        • Novartis Investigative Site
  • Spanje
      • Valencia, Spanje, 46014
        • Novartis Investigative Site
    • Barcelona
      • Esplugues, Barcelona, Spanje, 08950
        • Novartis Investigative Site
  • Thailand
      • Bangkok, Thailand, 10330
        • Novartis Investigative Site
      • Bangkok, Thailand, 10700
        • Novartis Investigative Site
    • Hat Yai
      • Songkhla, Hat Yai, Thailand, 90110
        • Novartis Investigative Site
  • Turkije (Türkiye)
      • Adana, Turkije (Türkiye), 01330
        • Novartis Investigative Site
    • Fatih
      • Istanbul, Fatih, Turkije (Türkiye), 34093
        • Novartis Investigative Site
    • Sihhiye Altindag
      • Ankara, Sihhiye Altindag, Turkije (Türkiye), 06230
        • Novartis Investigative Site
  • Verenigd Koninkrijk
      • Manchester, Verenigd Koninkrijk, M13 9WL
        • Novartis Investigative Site
      • Southampton, Verenigd Koninkrijk, SO16 6YD
        • Novartis Investigative Site
    • Cambridgeshire
      • Peterborough, Cambridgeshire, Verenigd Koninkrijk, PE3 9GZ
        • Novartis Investigative Site
  • Verenigde Staten
    • California
      • Bakersfield, California, Verenigde Staten, 93301
        • Kern Research
      • San Diego, California, Verenigde Staten, 92123
        • Allergy and Asthma Medical Group and Research Center
    • Florida
      • Miami, Florida, Verenigde Staten, 33156
        • Pediatric Dermatology of Miami at the Pediatric CoE
    • Idaho
      • Boise, Idaho, Verenigde Staten, 83706
        • Treasure Valley Medical Research
    • Illinois
      • Glenview, Illinois, Verenigde Staten, 60077
        • Endeavor Health
    • Kentucky
      • Owensboro, Kentucky, Verenigde Staten, 42301
        • Allergy and Asthma Specialist P S C
    • Ohio
      • Toledo, Ohio, Verenigde Staten, 43617
        • Toledo Institute of Clinical Research
    • Oklahoma
      • Oklahoma City, Oklahoma, Verenigde Staten, 73120
        • Allergy Asthma and Clinical Research
    • Pennsylvania
      • Pittsburgh, Pennsylvania, Verenigde Staten, 15241
        • Allergy and Clinical Immunology Associates
    • Texas
      • San Antonio, Texas, Verenigde Staten, 78213
        • RFSA Dermatology
    • Utah
      • Sandy City, Utah, Verenigde Staten, 84093
        • Allergy Associates of Utah
  • Zuid-Afrika
      • Cape Town, Zuid-Afrika, 7925
        • Novartis Investigative Site
    • Gauteng
      • Pretoria, Gauteng, Zuid-Afrika, 0181
        • Novartis Investigative Site

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

12 jaar tot 17 jaar (Kind)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

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.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Verviervoudigen

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: 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.
Geneesmiddel: LOU064 (blinded)

LOU064 (blinded) active treatment

Andere namen:
  • remibrutinib
Placebo-vergelijker: 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)
Geneesmiddel: placebo

matching active drug

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Change from baseline in UAS7
Tijdsspanne: 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.
Baseline, week 12
Change fron baseline in ISS7
Tijdsspanne: 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.
Baseline, Week 12
Change from baseline in HSS7
Tijdsspanne: 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.
Baseline, Week 12

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Cmax of remibrutinib
Tijdsspanne: At Week 12, before study drug intake, then after 30 min, 1 h, 2h, 3h and 4 h after study drug intake

The maximum (peak) observed blood drug concentration after single dose administration
At Week 12, before study drug intake, then after 30 min, 1 h, 2h, 3h and 4 h after study drug intake
Tmax of remibrutinib
Tijdsspanne: At Week 12, before study drug intake, then after 30 min, 1 h, 2h, 3h and 4 h after study drug intake

The time to reach maximum (peak) blood drug concentration after single dose administration
At Week 12, before study drug intake, then after 30 min, 1 h, 2h, 3h and 4 h after study drug intake
AUClast of remibrutinib
Tijdsspanne: At Week 12, before study drug intake, then after 30 min, 1 h, 2h, 3h and 4 h after study drug intake

The Area Under the Curve (AUC) from pre-dose to the last measurable concentration sampling time
At Week 12, before study drug intake, then after 30 min, 1 h, 2h, 3h and 4 h after study drug intake
Absolute change from baseline in ISS7
Tijdsspanne: 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.
Baseline - Week 12
Absolute change from baseline in HSS7
Tijdsspanne: 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.
Baseline - Week 12
Achievement of UAS7 ≤ 6 (yes/no)
Tijdsspanne: Week 12 and over time

Disease activity control is defined as UAS7 ≤ 6. 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.
Week 12 and over time
Achievement of UAS7 = 0 (yes/no)
Tijdsspanne: Week 12 and over time

Complete absence of hives and itch is defined as UAS7 = 0. 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.
Week 12 and over time
Absolute change from baseline in CDLQI score
Tijdsspanne: Week 12

The Children Dermatology life Quality Index (CDLQI) score range is 0 to 30, with 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).
Week 12
Number of weeks without angioedema, assessed by the cumulative number of weeks with an AAS7 = 0 response
Tijdsspanne: baseline - Week 12

Angioedema Activity Score (AAS7) is a measure of the frequency and intensity of angioedema episodes. The total possible range of scores over 7 days is 0-105 where higher scores indicate increased angioedema activity.
baseline - Week 12
Occurrence of treatment-emergent adverse events (AE) and serious adverse events (SAE) during the core period
Tijdsspanne: 28 weeks

To demonstrate the safety and tolerability of remibrutinib by assessing occurrence of treatment emergent adverse events and serious adverse events during the core period of the study.
28 weeks
Occurrence of treatment emergent AEs, and SAEs during the Open Label Extension (OLE) period
Tijdsspanne: 3 years

To demonstrate the safety and tolerability of remibrutinib by assessing occurrence of treatment emergent adverse events and serious adverse events during the OLE period of the study.
3 years

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Novartis Pharmaceuticals

Onderzoekers

  • Studie directeur: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

11 juli 2023

Primaire voltooiing (Geschat)

3 november 2026

Studie voltooiing (Geschat)

30 maart 2032

Studieregistratiedata

Eerst ingediend

16 december 2022

Eerst ingediend dat voldeed aan de QC-criteria

23 december 2022

Eerst geplaatst (Werkelijk)

10 januari 2023

Updates van studierecords

Laatste update geplaatst (Werkelijk)

15 oktober 2026

Laatste update ingediend die voldeed aan QC-criteria

14 oktober 2026

Laatst geverifieerd

1 oktober 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • CLOU064F12301
  • 2022-502159-78-00 (Andere identificatie: EU CT Number)

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

JA

Beschrijving IPD-plan

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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