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Essai randomisé en double aveugle contrôlé par placebo de 24 semaines pour évaluer l'efficacité, la pharmacocinétique et l'innocuité de LOU064 chez les adolescents (12 -

14 octobre 2026 mis à jour par: Novartis Pharmaceuticals

Un essai en double aveugle, randomisé et contrôlé par placebo pour évaluer l'efficacité, la pharmacocinétique et l'innocuité du remibrutinib (LOU064) pendant 24 semaines chez des adolescents de 12 à moins de 18 ans atteints d'urticaire chronique spontanée insuffisamment contrôlée par des antihistaminiques H1 suivis de une extension facultative en ouvert jusqu'à 3 ans supplémentaires et une période de suivi facultative de sécurité à long terme sans traitement jusqu'à 3 ans supplémentaires

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

Aperçu de l'étude

Statut

Actif, ne recrute pas

Les conditions

Intervention / Traitement

Description détaillée

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

Type d'étude

Interventionnel

Inscription (Réel)

100

Phase

  • Phase 3

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

  • Afrique du Sud
      • Cape Town, Afrique du Sud, 7925
        • Novartis Investigative Site
    • Gauteng
      • Pretoria, Gauteng, Afrique du Sud, 0181
        • Novartis Investigative Site
  • Allemagne
      • Berlin, Allemagne, 13353
        • Novartis Investigative Site
      • Tübingen, Allemagne, 72076
        • Novartis Investigative Site
    • Hesse
      • Frankfurt am Main, Hesse, Allemagne, 60590
        • Novartis Investigative Site
  • Argentine
      • CABA, Argentine, C1181ACH
        • Novartis Investigative Site
      • San Miguel de Tucumán, Argentine, T4000AXL
        • Novartis Investigative Site
    • Buenos Aires
      • CABA, Buenos Aires, Argentine, C1414AIF
        • Novartis Investigative Site
    • Santa Fe Province
      • Rosario, Santa Fe Province, Argentine, 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
  • Chine
      • Beijing, Chine, 100050
        • Novartis Investigative Site
      • Beijing, Chine, 100069
        • Novartis Investigative Site
    • Guangdong
      • Guangzhou, Guangdong, Chine, 510091
        • Novartis Investigative Site
    • Sichuan
      • Chengdu, Sichuan, Chine, 610041
        • Novartis Investigative Site
  • Espagne
      • Valencia, Espagne, 46014
        • Novartis Investigative Site
    • Barcelona
      • Esplugues, Barcelona, Espagne, 08950
        • Novartis Investigative Site
  • Hong Kong
    • Hong Kong
      • Hong Kong, Hong Kong, Hong Kong, 999077
        • Novartis Investigative Site
  • Italie
    • FI
      • Florence, FI, Italie, 50139
        • Novartis Investigative Site
    • PV
      • Pavia, PV, Italie, 27100
        • Novartis Investigative Site
    • SI
      • Siena, SI, Italie, 53100
        • Novartis Investigative Site
  • Japon
    • Fukuoka
      • Kitakyushu, Fukuoka, Japon, 8078556
        • Novartis Investigative Site
    • Kumamoto
      • Kamimashi-gun, Kumamoto, Japon, 8613106
        • Novartis Investigative Site
    • Osaka
      • Sakai, Osaka, Japon, 5938324
        • Novartis Investigative Site
    • Shimane
      • Izumo, Shimane, Japon, 6938501
        • Novartis Investigative Site
    • Tokyo
      • tabashi City, Tokyo, Japon, 1738610
        • Novartis Investigative Site
  • Malaisie
    • Sarawak
      • Kuching, Sarawak, Malaisie, 93586
        • Novartis Investigative Site
  • Pays-Bas
      • Utrecht, Pays-Bas, 3584 CX
        • Novartis Investigative Site
  • Pologne
      • Lodz, Pologne, 90-436
        • Novartis Investigative Site
  • Royaume-Uni
      • Manchester, Royaume-Uni, M13 9WL
        • Novartis Investigative Site
      • Southampton, Royaume-Uni, SO16 6YD
        • Novartis Investigative Site
    • Cambridgeshire
      • Peterborough, Cambridgeshire, Royaume-Uni, PE3 9GZ
        • Novartis Investigative Site
  • Thaïlande
      • Bangkok, Thaïlande, 10330
        • Novartis Investigative Site
      • Bangkok, Thaïlande, 10700
        • Novartis Investigative Site
    • Hat Yai
      • Songkhla, Hat Yai, Thaïlande, 90110
        • Novartis Investigative Site
  • Turquie (Türkiye)
      • Adana, Turquie (Türkiye), 01330
        • Novartis Investigative Site
    • Fatih
      • Istanbul, Fatih, Turquie (Türkiye), 34093
        • Novartis Investigative Site
    • Sihhiye Altindag
      • Ankara, Sihhiye Altindag, Turquie (Türkiye), 06230
        • Novartis Investigative Site
  • États-Unis
    • California
      • Bakersfield, California, États-Unis, 93301
        • Kern Research
      • San Diego, California, États-Unis, 92123
        • Allergy and Asthma Medical Group and Research Center
    • Florida
      • Miami, Florida, États-Unis, 33156
        • Pediatric Dermatology of Miami at the Pediatric CoE
    • Idaho
      • Boise, Idaho, États-Unis, 83706
        • Treasure Valley Medical Research
    • Illinois
      • Glenview, Illinois, États-Unis, 60077
        • Endeavor Health
    • Kentucky
      • Owensboro, Kentucky, États-Unis, 42301
        • Allergy and Asthma Specialist P S C
    • Ohio
      • Toledo, Ohio, États-Unis, 43617
        • Toledo Institute of Clinical Research
    • Oklahoma
      • Oklahoma City, Oklahoma, États-Unis, 73120
        • Allergy Asthma and Clinical Research
    • Pennsylvania
      • Pittsburgh, Pennsylvania, États-Unis, 15241
        • Allergy and Clinical Immunology Associates
    • Texas
      • San Antonio, Texas, États-Unis, 78213
        • RFSA Dermatology
    • Utah
      • Sandy City, Utah, États-Unis, 84093
        • Allergy Associates of Utah

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

12 ans à 17 ans (Enfant)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Tout

La description

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.

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Randomisé
  • Modèle interventionnel: Affectation parallèle
  • Masquage: Quadruple

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: 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.
Médicament: LOU064 (blinded)

LOU064 (blinded) active treatment

Autres noms:
  • rémibrutinib
Comparateur placebo: 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)
Médicament: placebo

matching active drug

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Change from baseline in UAS7
Délai: 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
Délai: 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
Délai: 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

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Cmax of remibrutinib
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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)
Délai: 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)
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Novartis Pharmaceuticals

Les enquêteurs

  • Directeur d'études: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

11 juillet 2023

Achèvement primaire (Estimé)

3 novembre 2026

Achèvement de l'étude (Estimé)

30 mars 2032

Dates d'inscription aux études

Première soumission

16 décembre 2022

Première soumission répondant aux critères de contrôle qualité

23 décembre 2022

Première publication (Réel)

10 janvier 2023

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

15 octobre 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

14 octobre 2026

Dernière vérification

1 octobre 2026

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • CLOU064F12301
  • 2022-502159-78-00 (Autre identifiant: EU CT Number)

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

OUI

Description du régime IPD

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

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Oui

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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