A Study to Evaluate the Safety and Efficacy of Satralizumab in Participants With Neuromyelitis Optica Spectrum Disorder (NMOSD)

NCT04660539 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: WN423492020-003413-35
• Study Type: interventional
• Target: 119
• Locations: 18 countries
• Sites: 53

Brief Summary

This multicenter, single-arm, open-label study will evaluate the long-term safety and efficacy of satralizumab in participants with neuromyelitis optica spectrum disorder (NMOSD) who completed open-label extension (OLE) period of studies BN40898 and BN40900. Participants will receive satralizumab as monotherapy or in combination with one of the following background immunosuppressive treatments: azathioprine (AZA), mycophenolate mofetil (MMF), or oral corticosteroids.

Conditions

Neuromyelitis Optica Spectrum Disorder

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  AE=any untoward medical occurrence in a participant administered a pharmaceutical product, regardless of causal relationship with it. AE can therefore be any unfavorable \& unintended sign, symptoms/disease temporally associated with use of a medicinal (investigational) product, whether or not considered related to it. SAE is any significant hazard, contraindication, or side effect that is fatal or life-threatening, requires hospitalization or prolongation of an existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is medically significant or requires intervention. The first dosing visit in the current study or randomization visit in the parent studies (NCT02028884/NCT02073279) was considered as baseline for this outcome measure. All AEs from the time of randomization in the parent studies for satralizumab-treated participants are reported here.

  Baseline up to 523 weeks

• Number of Participants With Adverse Events of Special Interest (AESIs) and Selected AEs

  An AESIs included potential drug induced liver injury and suspected transmission of an infectious agent by study drug defined as any organism, virus, or infectious particle (e.g., prion protein transmitting transmissible spongiform encephalopathy), pathogenic or non-pathogenic causing clinical symptoms or laboratory findings that indicate an infection in a participant exposed to a medicinal product. Selected AEs included infections that required treatments with IV antibiotics, antifungals, or antivirals; opportunistic infections that required treatment with oral antibiotics, antifungals, or antivirals and injection related reaction. The first dosing visit in the current study or randomization visit in the parent studies (NCT02028884/NCT02073279) was considered as baseline for this outcome measure. All AEs from the time of randomization in the parent studies for satralizumab-treated participants are reported here.

  Baseline up to 523 weeks

Secondary Outcomes (16)

• Number of Participants With Suicidality Assessed Using Columbia-Suicide Severity Rating Scale (C-SSRS)

  Baseline up to 523 weeks

• Number of Participants With Serious Infections and Hepatotoxicity

  Baseline up to 523 weeks

• Time to First Protocol-defined Relapse (PDR) as Assessed by Investigator (iPDR)

  Baseline up to 528 weeks

• iPDR-free Rate up to Week 456

  Baseline up to Week 456

• Percentage of Relapse-Free Participants

  Baseline up to 528 weeks

Study Arms (1)

Satralizumab Treatment

EXPERIMENTAL

Participants will receive satralizumab subcutaneously (SC) every 4 weeks (Q4W)

Drug: satralizumab; Drug: azathioprine (AZA); Drug: mycophenolate mofetil (MMF); Drug: oral corticosteroids

Interventions

satralizumab DRUG

Satralizumab will be administered by SC injection in the abdominal or femoral region at a dose of 120 mg (fixed dose) Q4W for up to 3 years

Also known as: Enspryng

Satralizumab Treatment

azathioprine (AZA) DRUG

Participants are permitted to use AZA during the study as background immunosuppressive treatment at a maximum dose of 3 milligram per kilogram per day (mg/kg/day)

Also known as: non-investigational medicinal product (NIMP)

Satralizumab Treatment

mycophenolate mofetil (MMF) DRUG

Participants are permitted to use MMF during the study as background immunosuppressive treatment at a maximum dose of 3000 mg/day

Also known as: NIMP

Satralizumab Treatment

oral corticosteroids DRUG

Participants are permitted to use oral corticosteroids (prednisolone equivalent) during the study as background immunosuppressive treatment at a maximum dose of 15 mg/day

Also known as: NIMP

Satralizumab Treatment

Eligibility Criteria

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

You may qualify if:

• Participants aged less than 18 years at the time of informed consent for Study BN40898 can continue treatment with a combination of oral corticosteroids and either AZA or MMF
• Participated in Study BN40898 or Study BN40900 with satralizumab in NMOSD, are on ongoing satralizumab treatment and were anti-aquaporin-4 IgG antibody (AQP4-IgG) seropositive at screening in these studies. Participants with NMOSD who were AQP4-IgG seronegative at screening in Study BN40898 or Study BN40900 can be enrolled if the investigator considers the continued treatment with satralizumab to be beneficial for the participant
• For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use adequate contraception during the treatment period and for 3 months after the final dose of satralizumab.

You may not qualify if:

• Pregnant or breastfeeding, or intending to become pregnant during the study or within 3 months after the final dose of study drug. Women of childbearing potential must have a negative urine pregnancy test result on the baseline visit prior to initiation of study drug
• Evidence of any serious uncontrolled concomitant diseases that may preclude participation including 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 that requires delaying the next satralizumab dose at the time of enrollment
• NMOSD relapse at the time of enrollment
• Laboratory abnormalities at the last assessment in Study BN40898 or Study BN40900 that preclude re-treatment with satralizumab

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (53)

Children's Hospital of Alabama

Birmingham, Alabama, 35233, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Columbus Research and Wellness

Columbus, Georgia, 31909, United States

Location

University of Chicago

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

Wayne State University; UHC-4H

Detroit, Michigan, 48201, United States

Location

The Neurological Institute PA

Charlotte, North Carolina, 28204, United States

Location

OhioHealth Research Institute

Columbus, Ohio, 43214, United States

Location

Jefferson Hospital For Neuroscience; Jefferson Neurology Associates

Philadelphia, Pennsylvania, 19107, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75390-0001, United States

Location

Central Texas Neurology Consultants

Round Rock, Texas, 78681, United States

Location

UMHAT 'Dr. Georgi Stranski', EAD

Pleven, 5800, Bulgaria

Location

Multiprofile Hospital for Active Treatment of Neurology and Psychiatry Sv. Naum EAD

Sofia, 1113, Bulgaria

Location

University Multiprofile Hospital for Active Treatment Aleksandrovska EAD

Sofia, 1431, Bulgaria

Location

MS Clinical Trials Group

Vancouver, British Columbia, V6T 1Z3, Canada

Location

Centre hospitalier de l'Universite de Montreal (CHUM)

Montreal, Quebec, H2X 0A9, Canada

Location

Clinical Hospital Centre Osijek

Osijek, 31000, Croatia

Location

Ruhr Universitat Bochum

Bochum, 44791, Germany

Location

Jahn Ferenc Del-Pesti Korhaz es Rendelointezet

Budapest, 1204, Hungary

Location

Azienda Ospedaliera Sant'Andrea

Rome, Lazio, 00189, Italy

Location

Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele

Catania, Sicily, 95123, Italy

Location

Kyushu University Hospital

Fukuoka, 812-8582, Japan

Location

Tohoku University Hospital

Miyagi, 980-8574, Japan

Location

Niigata University Medical & Dental Hospital

Niigata, 951-8520, Japan

Location

Osaka University Hospital

Osaka, 565-0871, Japan

Location

Kindai University Hospital

Osaka, 589-8511, Japan

Location

Tokyo Women's Medical University Hospital

Tokyo, 162-8666, Japan

Location

National Center of Neurology and Psychiatry

Tokyo, 187-8551, Japan

Location

Hospital Kuala Lumpur

Kuala Lumpur, FED. Territory of Kuala Lumpur, 50586, Malaysia

Location

NZOZ Wielospecjalistyczna Poradnia Lekarska SYNAPSIS

Katowice, 40-123, Poland

Location

M.A. - LEK A. M. Maciejowscy SC. Centrum Terapii SM

Katowice, 40-571, Poland

Location

Samodzielny Publiczny Szpital Kliniczny Nr 4 w Lublinie; Klinika Neurologii

Lublin, 20-954, Poland

Location

Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny; Klinika Neurologii

Warsaw, 02-097, Poland

Location

Instytut Psychiatrii i Neurologii

Warsaw, 02-957, 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

Seoul, 02841, South Korea

Location

Asan Medical Center - PPDS

Seoul, 05505, South Korea

Location

Hospital Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hosp. Clinico San Carlos

Madrid, 28040, Spain

Location

China Medical University Hospital

North Dist., 40402, Taiwan

Location

National Cheng Kung University Hospital

Tainan, 70457, Taiwan

Location

National Taiwan University Hospital

Taipei, 10002, Taiwan

Location

Taipei Veterans General Hospital

Taipei, 112, Taiwan

Location

Bilim University Medical Faculty Florence Nightingale Hospital

Istanbul, 34333, Turkey (Türkiye)

Location

Municipal Non-Profit Enterprise City Clinical Hospital #16 of Dnipro City Council

Dnipro, Katerynoslav Governorate, 49069, Ukraine

Location

Municipal Non-Commercial Enterprise Odesa RMC for Mental Health of Odessa Regional Council

Odesa, Kherson Governorate, 65006, Ukraine

Location

Communal NPE Vinnytsia Reg. Clin. Psychoneurolog. Hosp. n.a. O.I. Yushchenko of Vinnytsia RC

Vinnytsia, Podolia Governorate, 21037, Ukraine

Location

Communal Nonprofit enterprise Ternopil Regional Clinical Psychoneurological Hospital of TRC

Ternopil, Volhynian Governorate, 46027, Ukraine

Location

National Hospital For Neurology and Neurosurgery

London, WC1N 3BG, United Kingdom

Location

Related Publications (1)

• 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

MeSH Terms

Conditions

Neuromyelitis Optica

Interventions

satralizumab; Azathioprine; Mycophenolic Acid; Adrenal Cortex Hormones

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

Intervention Hierarchy (Ancestors)

Thionucleosides; Sulfur Compounds; Organic Chemicals; Mercaptopurine; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists

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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2020
• First Posted: December 9, 2020
• Study Start: March 2, 2021
• Primary Completion: May 28, 2024
• Study Completion: May 28, 2024
• Last Updated: December 27, 2024
• Results First Posted: December 27, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will share

Locations

IT (2); TW (4); CR (1); JP (7); HU (1); TU (1); UA (4); GB (1); CA (2); PR (1); PL (6); US (13); KR (2); DE (1); BU (3); ES (2); MY (1); RO (1)