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

Not Applicable

Recruiting

• Conditions: Relapsing Multiple Sclerosis
• Interventions: Drug: Fenebrutinib

• Registration Number: NCT07161258
• Lead Sponsor: Hoffmann-La Roche

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-08-20
• Status Verified: 2025-09
• Study First Submitted QC: 2025-09-01
• Study First Posted: 2025-09-08
• Last Update Submitted: 2025-09-19
• Last Update Posted: 2025-09-22
• Study Start: 2025-10-03
• Primary Completion: 2027-06-08
• Study Completion: 2029-02-13

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 12

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
• Peripheral venous access that precludes venous blood sampling as required per study protocol
• 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Fenebrutinib	Fenebrutinib	Participants will receive fenebrutinib orally.

Primary Outcome Measures

Name	Time	Method
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

Secondary Outcome Measures

Name	Time	Method
Percentage of Participants With Suicidal Ideation (SI) or Behavior, as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS)	Up to approximately 100 weeks	The C-SSRS is an interview-based instrument used to assess baseline incidence of SI and behavior. The assessment includes yes/no (0/1) responses for 5 questions, each related to SI.
Number of Participants With Adverse Events (AEs)	Up to approximately 100 weeks
Change From Baseline in Vital Signs - Pulse Rate	Baseline up to approximately 100 Weeks
Change From Baseline in Vital Signs - Blood Pressure	Baseline up to approximately 100 Weeks
Change From Baseline in Single 12-lead Electrocardiogram (ECG) Parameter - QTc Interval	Baseline up to approximately 100 Weeks
Change From Baseline in Single 12-lead ECG Parameter - PR Interval	Baseline up to approximately 100 Weeks
Change from Baseline in Clinical Laboratory Test Results - Alanine Aminotransferase (ALT)	Baseline up to approximately 100 Weeks
Change from Baseline in Clinical Laboratory Test Results - Aspartate Aminotransferase (AST)	Baseline up to approximately 100 Weeks

Trial Locations

Locations (5)

Sanatorio del Sur S.A.; 🇦🇷 San Miguel de Tucumán, Argentina

Instituto de Neurologia de Curitiba - Hospital Ecoville; 🇧🇷 Curitiba, Paraná, Brazil

Centro de Pesquisas Clinicas - CPCLIN; 🇧🇷 São Paulo, São Paulo, Brazil

CNE of Lviv Reg Clin Hospital Dept of Neurology D.Halytskyi Lviv NMU; 🇺🇦 Lviv, KIEV Governorate, Ukraine

Nucleo de Pesquisa Clinica do Rio Grande do Sul NPCR; 🇧🇷 Porto Alegre, Rio Grande do Sul, Brazil