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A Pharmacokinetics (PK), Pharmacodynamics (PD), Safety and Tolerability Study of Fenebrutinib in Children and Adolescents With Relapsing Multiple Sclerosis (RMS)

NCT07161258 · Hoffmann-La Roche
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Official title
An Open-label, Single-arm Study to Evaluate Pharmacokinetics, Pharmacodynamic Effects, Safety and Tolerability of Fenebrutinib in Children and Adolescents With Relapsing Multiple Sclerosis
About this study
This open label, single arm study will evaluate the PK and PD effects of fenebrutinib in children and adolescents with RMS aged between 10 and \< 18 years. This study consists of a Dose Exploration Period and an Optional Extension Period. Eligible participants may choose to continue treatment with fenebrutinib in the optional extension period after completing the dose exploration period.
Eligibility criteria
Inclusion Criteria: * A diagnosis of RMS in accordance with the International Pediatric Multiple Sclerosis Study Group (IPMSSG) criteria for pediatric MS, Version 2012, and the revised 2017 McDonald Criteria and one or more of the following: at least one MS relapse during the previous year or two MS relapses in the previous 2 years or evidence of at least one Gd enhancing lesion on MRI within 6 month * Expanded Disability Status Scale (EDSS) at screening from 0 to 5.5 points, inclusive * Children and adolescents must have received all childhood vaccinations as per local/national recommendations for childhood vaccination against infectious diseases Exclusion Criteria: * A diagnosis of primary progressive multiple sclerosis (PPMS) or non-active secondary progressive multiple sclerosis (SPMS) * Co-morbid Conditions: * Potentially confounding neurological, somatic, or metabolic disorders * Current clinically significant psychiatric or medical illness * History of cancer, transplants, or bleeding disorders * Inability to complete an MRI scan or get gadolinium * Abnormal liver function tests or blood counts * Sensitivity or intolerance to any ingredient (including excipients) of fenebrutinib tablets * Active, recurrent, or chronic infections * Recent or anticipated use of prohibited medications/treatments: * Certain disease-modifying therapy (DMT) and other immunosuppressants * Drugs interacting with fenebrutinib (Cytochrome P450 3A4 \[CYP3A4\] inhibitors) * Any other investigational therapy, anticoagulants, certain vaccines * A score of 4 or 5 on the "last 6 months" section of the screening SI section or "yes" on any item of the "last 6 months" Suicidal Behavior (SB) section of the C-SSRS or a positive answer on Question 9 of the Patient Health Questionnaire-9 Modified for Adolecents (PHQ-A) or significant risk of suicide, in the investigator's judgment
Study design
Enrollment target: 12 participants
Allocation: na
Masking: none
Age groups: child
Timeline
Starts: 2025-10-06
Estimated completion: 2029-02-13
Last updated: 2026-04-13
Interventions
Drug: Fenebrutinib
Primary outcomes
  • Plasma Concentration of Fenebrutinib (Up to Week 96)
  • Total Number of New T1 Gadolinium (Gd)-enhancing Lesions on Brain Observed Through Magnetic Resonance Imaging (MRI) Scans (At Week 12)
Sponsor
Hoffmann-La Roche · industry
Contacts & investigators
ContactReference Study ID Number: CN45847 https://forpatients.roche.com/ · contact · global-roche-genentech-trials@gene.com · 888-662-6728 (U.S.)
InvestigatorClinical Trials · study_director, Hoffmann-La Roche
All locations (17)
INECO Neurociencias OronoRecruiting
Rosario, Santa Fe Province, Argentina
Sanatorio del Sur S.A.Recruiting
San Miguel de Tucumán, Argentina
L2IP ?Instituto de Pesquisas Clínicas Ltda.Recruiting
Brasília, Federal District, Brazil
Instituto de Neurologia de Curitiba - Hospital EcovilleRecruiting
Curitiba, Paraná, Brazil
Nucleo de Pesquisa Clinica do Rio Grande do Sul NPCRRecruiting
Porto Alegre, Rio Grande do Sul, Brazil
Centro de Pesquisas Clinicas - CPCLINRecruiting
São Paulo, São Paulo, Brazil
Clinstile S.A de C.V.Recruiting
Mexico City, Mexico CITY (federal District), Mexico
Neurociencias Estudios Clinicos S.C.Recruiting
Culiacán, Sinaloa, Mexico
Uniwersyteckie Centrum KliniczneRecruiting
Gdansk, Poland
Uniwersytecki Szpital Kliniczny w PoznaniuRecruiting
Poznan, Poland
Instytut "Pomnik - Centrum Zdrowia Dziecka"Recruiting
Warsaw, Poland
Centro Clnico Acadmico - Braga, Associao (2CA-Braga)Recruiting
Braga, Portugal
Unidade Local de Saude de Coimbra E P ERecruiting
Coimbra, Portugal
Hospital Universitario de La PrincesaRecruiting
Madrid, Spain
Hospital Universitario Ramon y CajalRecruiting
Madrid, Spain
Hospital Universitario Virgen MacarenaRecruiting
Seville, Spain
CNE of Lviv Reg Clin Hospital Dept of Neurology D.Halytskyi Lviv NMURecruiting
Lviv, KIEV Governorate, Ukraine
A Pharmacokinetics (PK), Pharmacodynamics (PD), Safety and Tolerability Study of Fenebrutinib in Children and Adolescents With Relapsing Multiple Sclerosis (RMS) · TrialPath