Efficacy and Safety Study of Satralizumab (SA237) as Monotherapy to Treat Participants With Neuromyelitis Optica (NMO) and Neuromyelitis Optica Spectrum Disorder (NMOSD)

NCT02073279 | Completed Phase 3 Results DMC FDA Drug

• 3 other identifiers: BN40900SA-309JG2015-005431-41
• Study Type: interventional
• Target: 95
• Locations: 15 countries
• Sites: 58

Brief Summary

The objectives of this study are to evaluate the efficacy, safety, pharmacodynamic, pharmacokinetic and immunogenic profiles of satralizumab in participants with NMO and NMOSD.

Conditions

Neuromyelitis Optica (NMO); NMO Spectrum Disorder (NMOSD)

Outcome Measures

Primary Outcomes (1)

• Time to First Protocol-Defined Relapse (TFR) During the Double-Blind (DB) Period

  TFR was defined as time from randomization to first occurrence of relapse in the DB period. Protocol-defined relapse was occurrence of new or worsening neurological symptoms attributable to neurological neuromyelitis optica (NMO) or neuromyelitis optica spectrum disorder (NMOSD). Symptoms had to persist for \>24 hours and not be attributable to confounding clinical factors (e.g., fever, infection, injury, change in mood, adverse reactions to medications). New or worsening neurological symptoms that occurred \< 31 days following onset of a protocol-defined relapse were considered part of same relapse (i.e., if 2 relapses had onset days that were 30 days of one another, they were counted only as 1 relapse), and onset date used in analysis was the date of first relapse.

  Up to Week 216

Secondary Outcomes (31)

• Change From Baseline to Week 24 in Visual Analogue Scale (VAS) for Pain During the DB Period

  Baseline, Week 24

• Change From Baseline to Week 24 in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale During the DB Period

  Baseline, Week 24

• Relapse-Free Rate During the DB Period

  Up to Week 216

• Annualized Relapse Rate (ARR) During the DB Period

  Up to Week 216

• Change From Baseline in Short Form Generic Health Survey (SF-36) Bodily Pain Domain Scores at 24 Week Intervals During the DB Period

  Baseline up to Week 216

Study Arms (2)

Satralizumab

EXPERIMENTAL

Participants randomized to this arm for the double-blind period will receive satralizumab monotherapy. The double-blind period for the participant ends either when the participant experiences a Clinical Endpoint Committee (CEC) confirmed protocol-defined relapse, the total number of CEC confirmed protocol-defined relapses reaches 44, or 1.5 years after the last patient was randomized. In the open-label extension period, the participant will receive satralizumab SC injection at Weeks 0, 2, and 4, and Q4W thereafter, up to the end of the study.

Drug: Satralizumab

Placebo

PLACEBO COMPARATOR

Participants randomized to this arm for the double-blind period will receive placebo monotherapy. The double-blind period for the participant ends either when the participant experiences a Clinical Endpoint Committee (CEC) confirmed protocol-defined relapse, the total number of CEC confirmed protocol-defined relapses reaches 44, or 1.5 years after the last patient was randomized.. In the open-label extension period, the participant will receive satralizumab SC injection at Weeks 0, 2, and 4, and Q4W thereafter, up to the end of the study.

Drug: Satralizumab; Drug: Placebo

Interventions

Satralizumab DRUG

Satralizumab will be administered subcutaneously (SC) at Weeks 0, 2, and 4, and thereafter once every 4 weeks (Q4W).

Also known as: SA237, RG6168

Placebo; Satralizumab

Placebo DRUG

Placebo will be administered subcutaneously (SC) at Weeks 0, 2, and 4, and thereafter once every 4 weeks (Q4W).

Placebo

Eligibility Criteria

• Age: 18 Years - 74 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants must be diagnosed as having either neuromyelitis optica (NMO) or NMO spectrum disorder (NMOSD), defined as the following:
• NMO as defined by Wingerchuk et al. 2006 criteria (requires all of the following 3 criteria: I. Optic neuritis, II. Acute myelitis, III. At least two of three supportive criteria: Contiguous spinal cord lesion identified on a magnetic resonance imaging \[MRI\] scan extending over 3 vertebral segments; Brain MRI not meeting diagnostic criteria for multiple sclerosis \[MS\]; NMO-IgG seropositive status)
• NMOSD as defined by either of following criteria with anti-aquaporin-4 (AQP4) antibody seropositive status at screening: i. Idiopathic single or recurrent events of longitudinally extensive myelitis (≥3 vertebral segment spinal cord MRI lesion); ii. Optic neuritis, single, recurrent or simultaneous bilateral
• Clinical evidence of at least 1 documented relapse (including first attack) in last 12 months prior to screening
• Expanded Disability Status Scale (EDSS) score from 0 to 6.5 inclusive at screening
• Age 18 to 74 years, inclusive at the time of informed consent
• Ability and willingness to provide written informed consent and to comply with the requirements of the protocol

You may not qualify if:

• Clinical relapse onset (including first attack) within 30 days prior to baseline
• Any previous treatment with interleukin 6 (IL-6) inhibitory therapy (e.g., tocilizumab), alemtuzumab, total body irradiation or bone marrow transplantation at any time
• Any previous treatment with anti-CD20, eculizumab, anti-BLyS monoclonal antibody (e.g., belimumab), any other treatment for prevention of multiple sclerosis (MS) relapse (e.g., interferon, natalizumab, glatiramer acetate, fingolimod, teriflunomide or dimethyl fumarate) within 6 months prior to baseline
• Any previous treatment with anti-CD4, cladribine, cyclophosphamide or mitoxantrone within 2 years prior to baseline
• Treatment with any investigational agent within 3 months prior to baseline
• Pregnancy or lactation.
• For participants of reproductive potential, a positive result from a serum pregnancy test at screening, or not willing to use reliable means of contraception (physical barrier \[participants or partner\] in conjunction with a spermicidal product, contraceptive pill, patch, injectables, intrauterine device or intrauterine system) during the treatment period and for at least 3 months after the last dose of study drug
• Any surgical procedure (except for minor surgeries) within 4 weeks prior to baseline
• Evidence of other demyelinating disease or progressive multifocal leukoencephalopathy (PML)
• Evidence of serious uncontrolled concomitant diseases that may preclude participant participation, as described; Other nervous system disease, cardiovascular disease, hematologic/hematopoiesis disease, respiratory disease, muscular disease, endocrine disease, renal/urologic disease, digestive system disease, congenital or acquired severe immunodeficiency
• Known active infection (excluding fungal infections of nail beds or caries dentium) within 4 weeks prior to baseline
• Evidence of chronic active hepatitis B or C
• History of drug or alcohol abuse within 1 year prior to baseline
• History of diverticulitis that, in the Investigator's opinion, may lead to increased risk of complications such as lower gastrointestinal perforation
• Evidence of active tuberculosis (excluding participants receiving chemoprophylaxis for latent tuberculosis infection)

Sponsors & Collaborators

• Hoffmann-La Roche: lead
• Chugai Pharmaceutical: collaborator

Study Sites (58)

University of Colorado Denver -; Neurology

Aurora, Colorado, 80045, United States

Location

University of Miami UHealth Professional Arts Center

Miami, Florida, 33136, United States

Location

The MS Center of Vero Beach

Vero Beach, Florida, 32962, United States

Location

Columbus Research and Wellness

Columbus, Georgia, 31909, United States

Location

University of Chicago; Neurology

Chicago, Illinois, 60637, United States

Location

Consultants in Neurology Ltd

Northbrook, Illinois, 60062, United States

Location

OSF Saint Francis Medical Center

Peoria, Illinois, 64637, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

MidAmerica Neuroscience Institute

Prairie Village, Kansas, 66206, United States

Location

University of Michigan Health System

Ann Arbor, Michigan, 48109-0666, United States

Location

Wayne State University - Comp Clinic and MS. Center

Detroit, Michigan, 48201, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

The Neurological Institute PA

Charlotte, North Carolina, 28204, United States

Location

OhioHealth Research Institute

Columbus, Ohio, 43214, United States

Location

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Central Texas Neurology Consultants

Round Rock, Texas, 78681, United States

Location

UT Medicine San Antonio

San Antonio, Texas, 78229, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23219-1901, United States

Location

Wheaton Franciscan Healthcare - St. Francis Outpatient Center; Center for Neurological Disorders

Milwaukee, Wisconsin, 53215, United States

Location

Medical Help Center EOOD

Dobrich, 9300, Bulgaria

Location

MMA-MHAT Pleven - Clinic for Neurology

Pleven, 5800, Bulgaria

Location

UMHAT 'Dr. Georgi Stranski', EAD

Pleven, 5800, Bulgaria

Location

MHATNP Sveti Naum EAD

Sofia, 1113, Bulgaria

Location

UMHAT Alexandrovska, EAD

Sofia, 1431, Bulgaria

Location

MS Clinical Trials Group

Vancouver, British Columbia, V6T 1Z3, Canada

Location

Recherche Sepmus Inc.

Greenfield Park, Quebec, J4V 2J2, Canada

Location

Centre hospitalier de l'Universite de Montreal (CHUM)

Montreal, Quebec, H2X 0A9, Canada

Location

Klinicki bolnicki centar Osijek

Osijek, 31000, Croatia

Location

LTD Helsicore

Tbilisi, 0112, Georgia

Location

Pineo Medical Ecosystem LTD

Tbilisi, 0114, Georgia

Location

S.Khechinashvili Tbilisi State Medical University Clinic Ne

Tbilisi, 0179, Georgia

Location

PO G.Rodolico, AOU Policlinico-Vittorio Emanuele Catania

Catania, Sicily, 95123, Italy

Location

Hospital Universiti Sains Malaysia [Neurology]

Kubang Kerian, Kelantan, 16150, Malaysia

Location

Hospital Kuala Lumpur

Kuala Lumpur, 50586, Malaysia

Location

Philippine General Hospital

Manila, 1000, Philippines

Location

NZOZ Wielospecjalistyczna Poradnia Lekarska SYNAPSIS

Katowice, 40-123, Poland

Location

Miedzyleski Szpital Specjalistyczny w Warszawie

Warsaw, 04-749, Poland

Location

San Juan MS Center

Guaynabo, 00968, Puerto Rico

Location

SC Clubul Sanatatii SRL

Campulung Muscel, 115100, Romania

Location

Korea University Anam Hospital - Neurology

Seoul, 02841, South Korea

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Asan Medical Center

Seoul, 05505, South Korea

Location

Severance Hospital - Yonsei University Health System - Neurology

Seoul, 3722, South Korea

Location

China Medical University Healthcare System

Taichung, 40447, Taiwan

Location

National Cheng Kung University Hospital; Neurology

Tainan, 704, Taiwan

Location

Taipei Veterans General Hospital-Neurology

Taipei, 11217, Taiwan

Location

Bilim University Medical Faculty Florence Nightingale Hospital

Istanbul, 34333, Turkey (Türkiye)

Location

Ondokuz Mayis Univ. Med. Fac.

Samsun, 55139, Turkey (Türkiye)

Location

Ivano-Frankivska oblasna klinichna likarnia

Ivano-Frankivsk, KIEV Governorate, 76008, Ukraine

Location

Ivano-Frankivska miska klinichna likarnia №1

Ivano-Frankivsk, KIEV Governorate, 76018, Ukraine

Location

Kiev National Medical University

Kiev, KIEV Governorate, 3110, Ukraine

Location

Municipal Foundation of Kyiv Regional Council " Kyiv Region

Kyiv, KIEV Governorate, 4107, Ukraine

Location

Reginal clinical psyconeurological hospital

Ternopil, KIEV Governorate, 46014, Ukraine

Location

Vinnytskyi natsionalnyi medychnyi universytet imeni M.I. Pyr

Vinnytsia, Poltava Governorate, 21005, Ukraine

Location

Municipal Establishment "City Clinical Hospital #2; Neurology

Zaporozhya, Poltava Governorate, 69068, Ukraine

Location

Miska Klinichna Likarnia №16

Dnipropetrovsk, Tavria Okruha, 49100, Ukraine

Location

KU "Odeskyi oblasnyi medychnyi tsentr psykhichnoho zdorovia"

Odesa, Tavria Okruha, 65006, Ukraine

Location

Related Publications (5)

• Bennett JL, Fujihara K, Saiz A, Traboulsee AL, Greenberg BM, Weinshenker BG, Patti F, Kleiter I, Palace J, De Seze J, Evans R, Blondeau K, Klingelschmitt G, Vodopivec I, Rahim M, Yamamura T. Long-Term Efficacy and Safety of Satralizumab in Patients With Neuromyelitis Optica Spectrum Disorder From the SAkuraMoon Open-Label Extension Study. Neurol Neuroimmunol Neuroinflamm. 2025 Sep;12(5):e200450. doi: 10.1212/NXI.0000000000200450. Epub 2025 Jul 31.

  PMID: 40743487 DERIVED

• Greenberg BM, Fujihara K, Weinshenker B, Patti F, Kleiter I, Bennett JL, Palace J, Blondeau K, Burdeska A, Ngwa I, Klingelschmitt G, Triyatni M, Yamamura T. Analysis of infection rates in neuromyelitis optica spectrum disorder: Comparing satralizumab treatment in SAkuraMoon, post-marketing, and US-based health claims data. Mult Scler Relat Disord. 2025 Jul;99:106444. doi: 10.1016/j.msard.2025.106444. Epub 2025 Apr 19.

  PMID: 40288333 DERIVED

• Kleiter I, Traboulsee A, Palace J, Yamamura T, Fujihara K, Saiz A, Javed A, Mayes D, Budingen HV, Klingelschmitt G, Stokmaier D, Bennett JL. Long-term Efficacy of Satralizumab in AQP4-IgG-Seropositive Neuromyelitis Optica Spectrum Disorder From SAkuraSky and SAkuraStar. Neurol Neuroimmunol Neuroinflamm. 2022 Dec 8;10(1):e200071. doi: 10.1212/NXI.0000000000200071. Print 2023 Jan.

  PMID: 36724181 DERIVED

• Yamamura T, Weinshenker B, Yeaman MR, De Seze J, Patti F, Lobo P, von Budingen HC, Kou X, Weber K, Greenberg B. Long-term safety of satralizumab in neuromyelitis optica spectrum disorder (NMOSD) from SAkuraSky and SAkuraStar. Mult Scler Relat Disord. 2022 Oct;66:104025. doi: 10.1016/j.msard.2022.104025. Epub 2022 Jul 5.

  PMID: 36007339 DERIVED

• Traboulsee A, Greenberg BM, Bennett JL, Szczechowski L, Fox E, Shkrobot S, Yamamura T, Terada Y, Kawata Y, Wright P, Gianella-Borradori A, Garren H, Weinshenker BG. Safety and efficacy of satralizumab monotherapy in neuromyelitis optica spectrum disorder: a randomised, double-blind, multicentre, placebo-controlled phase 3 trial. Lancet Neurol. 2020 May;19(5):402-412. doi: 10.1016/S1474-4422(20)30078-8.

  PMID: 32333898 DERIVED

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

satralizumab

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Results Point of Contact

• Title: Medical Communications
• Organization: Hoffmann-La Roche

genentech@druginfo.com

800 821-8590

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 25, 2014
• First Posted: February 27, 2014
• Study Start: August 5, 2014
• Primary Completion: October 12, 2018
• Study Completion: January 31, 2022
• Last Updated: March 1, 2023
• Results First Posted: December 31, 2020

Record last verified: 2023-01

Locations

CR (1); RO (1); IT (1); KR (4); GE (3); TW (3); PR (1); PH (1); UA (9); BU (5); CA (3); MY (2); TU (2); PL (2); US (20)