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A Study to Evaluate Safety and Efficacy of BIIB091 in Participants With Relapsing Forms of Multiple Sclerosis

Phase 2
Recruiting
Conditions
Relapsing Forms of Multiple Sclerosis
Interventions
Drug: DRF
Drug: Placebo
Registration Number
NCT05798520
Lead Sponsor
Biogen
Brief Summary

The primary objectives are to investigate the safety and tolerability of BIIB091 monotherapy in participants with relapsing multiple sclerosis (RMS) (Part 1), and to evaluate the effects of BIIB091 combination therapy with Diroximel Fumarate (DRF) compared with the DRF monotherapy arm, on the key Magnetic Resonance Imaging (MRI) measure of active Central Nervous System (CNS) inflammation (Part 2). The secondary objectives are to evaluate the effects of BIIB091 monotherapy on the MRI measures of active CNS inflammation, to evaluate the effects of BIIB091 combination therapy with DRF compared with the DRF monotherapy arm on additional MRI measures of active CNS inflammation, to investigate the safety and tolerability of BIIB091 combination therapy with DRF in participants with RMS.

Detailed Description

The primary objectives of this study are to investigate the safety and tolerability of BIIB091 monotherapy in participants with relapsing multiple sclerosis (RMS) in Part 1 and to evaluate the effects of BIIB091 combination therapy with DRF compared with the DRF monotherapy arm on the key MRI measure of active central nervous system (CNS) inflammation in Part 2.

The secondary objectives of Part 1 of the study are to evaluate the effects of BIIB091 monotherapy on the MRI measures of active CNS inflammation and to assess the effect of BIIB091 monotherapy on QTc and other ECG parameters. The secondary objectives of Part 2 are to evaluate the effects of BIIB091 combination therapy with DRF compared with the DRF monotherapy arm on additional MRI measures of active CNS inflammation, the safety and tolerability of BIIB091 combination therapy with DRF in participants with RMS, and the effect of BIIB091 combination therapy with DRF on QTc and other ECG parameters.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
275
Inclusion Criteria
  1. Diagnosis of RMS [relapsing-remitting multiple sclerosis (RRMS) or active secondary progressive multiple sclerosis (SPMS)] in accordance with the 2017 Revised McDonald criteria.

  2. Time since MS symptom onset is <20 years.

  3. Must have expanded disability status scale (EDSS) score of 0 through 5.0 at screening and baseline.

  4. Must have at least 1 of the following occurring prior to Baseline (Day 1):

    • ≥2 clinical relapses in the last 24 months (but not within 30 days prior to Baseline [Day 1]) with at least 1 relapse during the last 12 months prior to randomization.
    • ≥1 clinical relapse within the past 24 months (but not within 30 days prior to Baseline [Day 1]) and ≥1 new brain MRI lesion (Gd-positive and/or new or enlarging T2 hyperintense lesion) within the past 12 months prior to randomization. The screening MRI could be used to satisfy this criterion (if needed for inclusion, local reading is required). For new or enlarging T2 hyperintense lesions, the reference scan cannot be >12 months prior to randomization.
    • ≥1 GdE lesion on brain MRI within 6 months prior to randomization.

Key

Exclusion Criteria
  1. Diagnosis of primary progressive multiple sclerosis (PPMS) in accordance with the 2017 Revised McDonald criteria.

  2. An MS relapse that has occurred within 30 days prior to Baseline (Day 1) or the participant has not stabilized from a previous relapse at the time of screening.

  3. History of severe allergic, anaphylactic reactions or hypersensitivity reaction to BIIB091 or DRF, the excipients contained in the formulation, and if appropriate, any diagnostic agents to be administered during the study, including the following:

    • Known hypersensitivity to any components of the study treatment
    • Known hypersensitivity to previous fumarate or bruton's tyrosine kinase (BTK) inhibitor treatments
    • History of hypersensitivity to parenteral administration of Gd-based contrast agents
  4. Evidence of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection within the past 4 weeks prior to Baseline.

  5. History or positive test result at screening for human immunodeficiency virus (HIV).

  6. Current or history of hepatitis C infection regardless of viral load.

  7. Current or possible hepatitis B.

  8. Current enrollment or plan to enroll in any other drug, biological, device, clinical study, or treatment with an investigational drug or approved therapy for investigational use within 90 days prior to randomization or 5 half-lives of the drug or therapy, whichever is longer.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Part 1: BIIB091 Low Dose + Matching Placebo for DRFPlaceboParticipants will receive BIIB091 low dose and matching placebo for DRF, orally, for up to 48 weeks.
Part 1: BIIB091 High Dose + Matching Placebo for DRFPlaceboParticipants will receive BIIB091 high dose and matching placebo for DRF, orally, for up to 48 weeks.
Part 1: DRF + Matching Placebo for BIIB091DRFParticipants will receive DRF standard dose and matching placebo for BIIB091, orally, for up to 48 weeks.
Part 2: DRF + Matching Placebo for BIIB091DRFParticipants will receive DRF standard dose and matching placebo for BIIB091, orally, for up to 48 weeks.
Part 1: BIIB091 Low Dose + Matching Placebo for DRFBIIB091Participants will receive BIIB091 low dose and matching placebo for DRF, orally, for up to 48 weeks.
Part 2: BIIB091 + DRF Standard DoseDRFParticipants will receive selected dose of BIIB091 (based on Part 1 data) and DRF standard dose, orally, for up to 48 weeks.
Part 1: BIIB091 High Dose + Matching Placebo for DRFBIIB091Participants will receive BIIB091 high dose and matching placebo for DRF, orally, for up to 48 weeks.
Part 1: DRF + Matching Placebo for BIIB091PlaceboParticipants will receive DRF standard dose and matching placebo for BIIB091, orally, for up to 48 weeks.
Part 2: DRF + Matching Placebo for BIIB091PlaceboParticipants will receive DRF standard dose and matching placebo for BIIB091, orally, for up to 48 weeks.
Part 2: BIIB091 + DRF Low DoseDRFParticipants will receive selected dose of BIIB091 (based on Part 1 data) and DRF low dose, orally, for up to 48 weeks.
Part 2: BIIB091 + DRF Standard DoseBIIB091Participants will receive selected dose of BIIB091 (based on Part 1 data) and DRF standard dose, orally, for up to 48 weeks.
Part 2: BIIB091 + DRF Low DoseBIIB091Participants will receive selected dose of BIIB091 (based on Part 1 data) and DRF low dose, orally, for up to 48 weeks.
Primary Outcome Measures
NameTimeMethod
Part 2: Cumulative Number of New T1 Gadolinium-Enhancing (GdE) LesionsWeek 8 to Week 16
Part 1: Number of Participants With Adverse Events (AEs)Day 1 up to Week 50

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 or auxiliary medicinal product, whether or not related to the medicinal (investigational) product or auxiliary medicinal product.

Part 1: Number of Participants With Serious Adverse Events (SAEs)From signing the informed consent form (ICF) to Week 50

SAE is any untoward medical occurrence that at any dose results in death, in the view of the investigator, places the participant at immediate risk of death (a life-threatening event), requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect or is a medically important event.

Secondary Outcome Measures
NameTimeMethod
Part 1: Cumulative Number of New T1 Gadolinium-Enhancing (GdE) LesionsWeek 8 to Week 16
Part 1: Number of Participants With Change From Baseline in Heart RateUp to Week 50
Part 2: Number of Participants With AEsDay 1 up to Week 50

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 or auxiliary medicinal product, whether or not related to the medicinal (investigational) product or auxiliary medicinal product.

Part 1: Cumulative Number of New or Enlarging T2 Hyperintense LesionsWeek 8 to Week 16
Part 1: Cumulative Volume of New or Enlarging T2 Hyperintense LesionsWeek 8 to Week 16
Part 1: Mean Change From Baseline in QT Interval Corrected for Heart Rate Using Fridericia's Formula (QTcF), RR, PR, QRS, and QT IntervalsUp to Week 50
Part 2: Cumulative Volume of New or Enlarging T2 Hyperintense LesionsWeek 8 to Week 16
Part 2: Number of Participants With SAEsFrom signing of ICF up to Week 50

SAE is any untoward medical occurrence that at any dose results in death, in the view of the investigator, places the participant at immediate risk of death (a life-threatening event), requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect or is a medically important event.

Part 2: Number of Participants With Change From Baseline in QTcF, RR, PR, QRS, and QT intervalsUp to Week 50
Part 2: Number of Participants With Change From Baseline in Heart RateUp to Week 50
Part 2: Number of Participants With ECG Abnormalities as Assessed by 12-Lead ECG MeasurementsUp to Week 50
Part 1: Number of Participants With Clinically Significant Electrocardiogram (ECG) AbnormalitiesUp to Week 50
Part 2: Cumulative Number of New or Enlarging T2 Hyperintense LesionsWeek 8 to Week 16

Trial Locations

Locations (77)

Vanderbilt MS Center

🇺🇸

Nashville, Tennessee, United States

HonorHealth Neurology

🇺🇸

Scottsdale, Arizona, United States

Alta Bates Summit Medical Center

🇺🇸

Berkeley, California, United States

University of California at Irvine Medical Center

🇺🇸

Orange, California, United States

University of Colorado School of Medic

🇺🇸

Aurora, Colorado, United States

University of Miami Miller School of Medicine

🇺🇸

Miami, Florida, United States

University of South Florida

🇺🇸

Tampa, Florida, United States

Vero Beach Neurology and Research Institute

🇺🇸

Vero Beach, Florida, United States

Fort Wayne Neurological Center

🇺🇸

Fort Wayne, Indiana, United States

University of Kansas Medical Center Research Institute, Inc.

🇺🇸

Kansas City, Kansas, United States

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Vanderbilt MS Center
🇺🇸Nashville, Tennessee, United States
Siddharama S Pawate
Principal Investigator
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