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A Study to Investigate the Efficacy of Fenebrutinib in Relapsing Multiple Sclerosis (RMS)

Phase 2
Active, not recruiting
Conditions
Relapsing Multiple Sclerosis
Interventions
Drug: Placebo
Registration Number
NCT05119569
Lead Sponsor
Hoffmann-La Roche
Brief Summary

This is a study evaluating the effect of fenebrutinib on brain magnetic resonance imaging (MRI) in participants with RMS. The safety and pharmacokinetics of fenebrutinib will also be evaluated. Participants will be randomized to receive either fenebrutinib or placebo. This study consists of two parts: Double-blind treatment (DBT) phase and an optional Open-label extension (OLE) phase.

Detailed Description

Not available

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
109
Inclusion Criteria
  • A diagnosis of RMS in accordance with the revised 2017 McDonald Criteria.
  • Expanded Disability Status Scale (EDSS) score of 0 - 5.5 at screening.
  • For female participants of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, and refrain from donating eggs.
  • For male participants: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, and refrain from donating sperm.
Exclusion Criteria
  • Disease duration of > 10 years from the onset of symptoms and an EDSS score at screening < 2.0.
  • Female participants who are pregnant or breastfeeding, or intending to become pregnant.
  • Male participants who intend to father a child during the study.
  • A diagnosis of Primary Progressive Multiple Sclerosis (PPMS) or non-active Secondary Progressive Multiple Sclerosis (SPMS).
  • Any known or suspected active infection at screening, including but not limited to a positive screening tests for Hepatitis B and C, an active or latent or inadequately treated infection with tuberculosis (TB), a confirmed or suspected progressive multifocal leukoencephalopathy (PML).
  • History of cancer including hematologic malignancy and solid tumors within 10 years of screening.
  • Presence of other neurological disorders that could interfere with the diagnosis of MS or with the assessments of safety or efficacy during the study.
  • Clinically significant cardiovascular, psychiatric, pulmonary, renal, hepatic, endocrine, metabolic or gastrointestinal disease.
  • Any concomitant disease that may require chronic treatment with systemic corticosteroids or immunosuppressants during the course of the study.
  • History of alcohol or other drug abuse within 12 months prior to screening.
  • History of or currently active primary or secondary (non-drug-related) immunodeficiency, including known history of HIV infection.
  • Inability to complete an MRI scan.
  • Adrenocorticotropic hormone or systemic corticosteroid therapy within 4 weeks prior to screening.
  • Receipt of a live-attenuated vaccine within 6 weeks prior to randomization.
  • Any previous treatment with immunomodulatory or immunosuppressive medication without an appropriate washout period.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
FenebrutinibFenebrutinibParticipants will receive oral fenebrutinib.
PlaceboPlaceboParticipants will receive oral placebo.
Primary Outcome Measures
NameTimeMethod
DBT Phase: New Gadolinium (Gd) - Enhancing T1 Lesion Rate Observed on Magnetic Resonance Imaging (MRI) Scans of the Brain Over 12 WeeksMRI scans performed at Weeks 4, 8 and 12

Radiologic evaluation for Gd enhancing T1 lesion rate was performed using a standardized MRI protocol at screening, and at Weeks 4, 8, and 12. All MRI scans were read by a centralized reading center for efficacy endpoints. The total number of new Gd-enhancing T1 lesions were calculated as the sum of the individual number of new lesions observed at Weeks 4, 8 and 12. The lesion rate (new lesions per scan) was estimated from a negative binomial regression model for the total number of events and was adjusted for the covariate 'presence or absence of T1 Gd+ lesions on the screening MRI'. Log-transformed number of scans were included in the negative binomial model as an "offset" variable to account for different number of scans.

Secondary Outcome Measures
NameTimeMethod
DBT Phase: New or Enlarging T2 - Weighted Lesion Rate Observed on MRI Scans of the Brain Over 12 WeeksMRI scans performed at Weeks 4, 8 and 12

Radiologic evaluation for new or enlarging T2 - weighted lesion rate was performed using a standardized MRI protocol at screening, and at Weeks 4, 8, and 12. All MRI scans were read by a centralized reading center for efficacy endpoints. Total number of new or enlarging T2-weighted lesions were calculated as the sum of the individual number of new or enlarging lesions at Weeks 4, 8, and 12. The lesion rate (new/enlarging lesions per scan) was estimated from a negative binomial regression model for the total number of events and was adjusted for the covariate 'presence or absence of T1 Gd+ lesions on the screening MRI'. Log-transformed number of scans were included in the negative binomial model as an "offset" variable to account for different numbers of scans.

DBT Phase: Proportion of Participants Free From Any New Gd - Enhancing T1 Lesions and New or Enlarging T2 - Weighted Lesions Observed on MRI Scans of the Brain Over 12 WeeksMRI scans performed at Weeks 4, 8 and 12

Radiologic evaluation for new Gd - enhancing T1 lesion and new or enlarging T2 - weighted lesions were performed using a standardized MRI protocol at screening, and at Weeks 4, 8, and 12. All MRI scans were read by a centralized reading center for efficacy endpoints. The total number of new Gd-enhancing T1 lesions and new or enlarging T2 - weighted lesions were calculated as the sum of the individual number of lesions observed at Weeks 4, 8 and 12. Analysis was performed using a logistic regression model performed on the status of both new T1 Gd+ lesion and new or enlarging T2-weighted lesions post-baseline (present or not) adjusted for the stratification factor(s) presence or absence of T1 Gd+ lesions on the screening MRI.

DBT Phase: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Up to Week 12

An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not considered related to the medicinal (investigational) product. A SAE is defined as any untoward medical occurrence that, at any dose results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability or incapacity, is a congenital anomaly or birth defect.

OLE Phase: Number of Participants With AEs and SAEsOLE Baseline (DBT Week 12) up to Week 192

An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not considered related to the medicinal (investigational) product. A SAE is defined as any untoward medical occurrence that, at any dose results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability or incapacity, is a congenital anomaly or birth defect.

Number of Participants With Post-baseline Suicidal Ideation or Suicidal Behavior as Measured Using Columbia-Suicide Severity Rating Scale (C-SSRS)Up to Week 192

C-SSRS=assessment tool used to assess lifetime suicidality of a participant (at baseline) as well as any new instances of suicidality (C-SSRS since last visit). Structured interview prompts recollection of suicidal ideation, including intensity of ideation, behavior, \& attempts with actual/potential lethality. Categories have binary responses (yes/no) \& include Wish to be Dead; Non-specific Active Suicidal Thoughts; Active Suicidal Ideation with Any Methods (Not Plan) without Intent to Act; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; Active Suicidal Ideation with Specific Plan and Intent, Preparatory Acts and Behavior; Aborted Attempt; Interrupted Attempt; Actual Attempt (non-fatal); Completed Suicide. Suicidal ideation/behavior is indicated by a "yes" answer to any of the listed categories. Score of 0 is assigned if no suicide risk is present. Score of 1 or higher= suicidal ideation or behavior.

Plasma Concentrations of Fenebrutinib at Specified TimepointsUp to Week 192

Trial Locations

Locations (17)

Fullerton Neurology and Headache Center

🇺🇸

Fullerton, California, United States

Cleveland Clinic Lou Ruvo

🇺🇸

Las Vegas, Nevada, United States

University Clinical Centre of the Republic of Srpska

🇧🇦

Banja Luka, Bosnia and Herzegovina

Clinical Hospital Center Rijeka - PPDS

🇭🇷

Rijeka, Croatia

General Hospital Varazdin

🇭🇷

Varazdin, Croatia

Klinicki bolnicki centar Zagreb

🇭🇷

Zagreb, Croatia

Poliklinika Solmed

🇭🇷

Zagreb, Croatia

Fakultni nemocnice u sv. Anny v Brne

🇨🇿

Brno, Czechia

Fakultni nemocnice Hradec Kralove

🇨🇿

Hradec Kralove, Czechia

Nemocnice Jihlava

🇨🇿

Jihlava, Czechia

Vseobecna Fakultni Nemocnice V Praze - CRC - PPDS

🇨🇿

Praha, Czechia

Clinical Center of Serbia

🇷🇸

Belgrade, Serbia

Military Medical Academy

🇷🇸

Belgrade, Serbia

Clinical center Kragujevac

🇷🇸

Kragujevac, Serbia

Clinical Center Nis

🇷🇸

NIS, Serbia

Clinical Hospital Centre Zemun

🇷🇸

Zemun, Serbia

Univerzitna nemocnica Bratislava

🇸🇰

Bratislava, Slovakia

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