Neuromyelitis Optica Spectrum DisorderPhase 3NCT04660539
A Study to Evaluate the Safety and Efficacy of Satralizumab in Participants With Neuromyelitis Optica Spectrum Disorder (NMOSD)
A Phase 3 study testing satralizumab, azathioprine (AZA) and mycophenolate mofetil (MMF) in people with Neuromyelitis Optica Spectrum Disorder, run by Hoffmann-La Roche at 53 sites in 18 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorder
- What is being tested?
- satralizumab, azathioprine (AZA) and mycophenolate mofetil (MMF)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 119 participants
- Where
- 53 sitesin 18 countries
- Study dates
- 2 Mar 2021to 28 May 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
53 sites in 18 countries.
53 sites in 18 countries
United States13 sites
Children's Hospital of Alabama
Birmingham, Alabama, 35233
The Neurological Institute PA
Charlotte, North Carolina, 28204
University of Chicago
Chicago, Illinois, 60637
Columbus Research and Wellness
Columbus, Georgia, 31909
OhioHealth Research Institute
Columbus, Ohio, 43214
University of Texas Southwestern Medical Center
Dallas, Texas, 75390-0001
Wayne State University; UHC-4H
Detroit, Michigan, 48201
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of Miami
Miami, Florida, 33136
Consultants in Neurology Ltd
Northbrook, Illinois, 60062
OSF Saint Francis Medical Center
Peoria, Illinois, 64637
Jefferson Hospital For Neuroscience; Jefferson Neurology Associates
Philadelphia, Pennsylvania, 19107
Central Texas Neurology Consultants
Round Rock, Texas, 78681
Japan7 sites
Kyushu University Hospital
Fukuoka, 812-8582
Tohoku University Hospital
Miyagi, 980-8574
Niigata University Medical & Dental Hospital
Niigata, 951-8520
Osaka University Hospital
Osaka, 565-0871
Kindai University Hospital
Osaka, 589-8511
Tokyo Women's Medical University Hospital
Tokyo, 162-8666
National Center of Neurology and Psychiatry
Tokyo, 187-8551
Poland6 sites
NZOZ Wielospecjalistyczna Poradnia Lekarska SYNAPSIS
Katowice, 40-123
M.A. - LEK A. M. Maciejowscy SC. Centrum Terapii SM
Katowice, 40-571
Samodzielny Publiczny Szpital Kliniczny Nr 4 w Lublinie; Klinika Neurologii
Lublin, 20-954
Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny; Klinika Neurologii
Warsaw, 02-097
Instytut Psychiatrii i Neurologii
Warsaw, 02-957
Miedzyleski Szpital Specjalistyczny w Warszawie
Warsaw, 04-749
Taiwan4 sites
China Medical University Hospital
North Dist., 40402
National Cheng Kung University Hospital
Tainan, 70457
National Taiwan University Hospital
Taipei, 10002
Taipei Veterans General Hospital
Taipei, 112
Ukraine4 sites
Municipal Non-Profit Enterprise City Clinical Hospital #16 of Dnipro City Council
Dnipro, Katerynoslav Governorate, 49069
Municipal Non-Commercial Enterprise Odesa RMC for Mental Health of Odessa Regional Council
Odesa, Kherson Governorate, 65006
Communal Nonprofit enterprise Ternopil Regional Clinical Psychoneurological Hospital of TRC
Ternopil, Volhynian Governorate, 46027
Communal NPE Vinnytsia Reg. Clin. Psychoneurolog. Hosp. n.a. O.I. Yushchenko of Vinnytsia RC
Vinnytsia, Podolia Governorate, 21037
Bulgaria3 sites
UMHAT 'Dr. Georgi Stranski', EAD
Pleven, 5800
Multiprofile Hospital for Active Treatment of Neurology and Psychiatry Sv. Naum EAD
Sofia, 1113
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
Sofia, 1431
Canada2 sites
Centre hospitalier de l'Universite de Montreal (CHUM)
Montreal, Quebec, H2X 0A9
MS Clinical Trials Group
Vancouver, British Columbia, V6T 1Z3
Italy2 sites
Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele
Catania, Sicily, 95123
Azienda Ospedaliera Sant'Andrea
Rome, Lazio, 00189
South Korea2 sites
Korea University Anam Hospital
Seoul, 02841
Asan Medical Center - PPDS
Seoul, 05505
Spain2 sites
Hospital Clinic de Barcelona
Barcelona, 08036
Hosp. Clinico San Carlos
Madrid, 28040
Malaysia1 site
Hospital Kuala Lumpur
Kuala Lumpur, FED. Territory of Kuala Lumpur, 50586
Turkey (Türkiye)1 site
Bilim University Medical Faculty Florence Nightingale Hospital
Istanbul, 34333
United Kingdom1 site
National Hospital For Neurology and Neurosurgery
London, WC1N 3BG
About this study
Official title: A Multicenter, Single Arm, Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Satralizumab in Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)
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
- Being tested
- satralizumab, azathioprine (AZA), mycophenolate mofetil (MMF), oral corticosteroids
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
satralizumabDrug
Also called Enspryng
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
azathioprine (AZA)Drug
Also called non-investigational medicinal product (NIMP)
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)
mycophenolate mofetil (MMF)Drug
Also called NIMP
Participants are permitted to use MMF during the study as background immunosuppressive treatment at a maximum dose of 3000 mg/day
oral corticosteroidsDrug
Also called NIMP
Participants are permitted to use oral corticosteroids (prednisolone equivalent) during the study as background immunosuppressive treatment at a maximum dose of 15 mg/day
| Study group | Role | What they receive |
|---|---|---|
| Satralizumab Treatment | Experimental | Participants will receive satralizumab subcutaneously (SC) every 4 weeks (Q4W) Receives: Drug: satralizumab; Drug: azathioprine (AZA); Drug: mycophenolate mofetil (MMF); Drug: oral corticosteroids |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Baseline up to 523 weeks
Secondary outcomes (16)
Number of Participants With Suicidality Assessed Using Columbia-Suicide Severity Rating Scale (C-SSRS)
C-SSRS=assessment tool used to assess lifetime suicidality of a participant (at baseline) as well as any new instances of suicidality (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 a-Wish to be Dead; b-Non-specific Active Suicidal Thoughts; c-Active Suicidal Ideation with Any Methods(Not Plan) without Intent to Act; d-Active Suicidal Ideation with Some Intent to Act, without Specific Plan; e-Active Suicidal Ideation with Specific Plan \& Intent, f-Preparatory Acts \& Behavior; g-Aborted Attempt; h-Interrupted Attempt; i-Actual Attempt(non-fatal); j-Completed Suicide. Suicidal ideation/behavior is indicated by a "yes" answer to any of listed categories. Score of 0 is assigned if no suicide risk is present. Score of 1 or higher= suicidal ideation or behavior. Categories with non-zero values are only reported here.
Measured: Baseline up to 523 weeks
Number of Participants With Serious Infections and Hepatotoxicity
Hepatotoxicity was defined using the following Medical Dictionary for Regulatory Activities Standardised MedDRA Queries (MedDRA SMQs) - Cholestasis and jaundice of hepatic origin (SMQ narrow) and Drug related hepatic disorders - severe events only (SMQ narrow). 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. Data for all serious infections and hepatotoxicity from the time of randomization in the parent studies for satralizumab-treated participants are reported here.
Measured: Baseline up to 523 weeks
Time to First Protocol-defined Relapse (PDR) as Assessed by Investigator (iPDR)
Time to first relapse (TFR) was defined as the time from randomization in parent studies to the first occurrence of the first iPDR. PDR=occurrence of new or worsening neurological symptoms attributable to neurological neuromyelitis optica (NMO) or NMOSD. New or worsening neurological symptoms that occur < 31 days following onset of PDR were considered part of same relapse. The time point of relapse onset=time at which the participant experienced any new or worsening neurological symptoms representing NMOSD clinical relapse(s). For participants who did not relapse at the time of analysis, TFR was censored at the clinical cutoff date (CCOD) or at the time of withdrawal from study. The first dosing visit in current study or randomization visit in the parent studies (NCT02028884/NCT02073279) was considered as baseline. All TFR data from the time of randomization in the parent studies up to the end of study WN42349 for satralizumab-treated participants are reported here.
Measured: Baseline up to 528 weeks
iPDR-free Rate up to Week 456
iPDR free rate was defined as the percentage of participants who did not experience a protocol-defined relapse as assessed by the investigator. Protocol-defined relapse was the occurrence of new or worsening neurological symptoms attributable to NMOSD. New or worsening neurological symptoms that occur < 31 days following the onset of a PDR were considered part of the same relapse. The first dosing visit in the current study or randomization visit in the parent studies (NCT02028884/NCT0207327) was considered as baseline for this outcome measure. All data from the time of randomization in the parent studies up to Week 456 for satralizumab-treated participants are reported here. Percentages have been rounded off to the nearest decimal point. Kaplan-Meier method was used to estimate the iPDR-free rates.
Measured: Baseline up to Week 456
Percentage of Relapse-Free Participants
Protocol-defined relapse was the occurrence of new or worsening neurological symptoms attributable to NMOSD. New or worsening neurological symptoms that occur < 31 days following the onset of a PDR were considered part of the same relapse. The first dosing visit in the current study or randomization visit in the parent studies (NCT02028884/NCT0207327) was considered as baseline for this outcome measure. All data from the time of randomization in the parent studies up to end of study WN42349 for satralizumab-treated participants are reported here. Participants who did not experience any relapse events are reported here. Percentages have been rounded off to the nearest decimal point.
Measured: Baseline up to 528 weeks
Annualized Relapse Rate (ARR)
ARR was calculated as total number of relapses experienced divided by the participant-years of the whole study period. Adjusted ARR was calculated using Poisson regression model adjusted by study identifier (BN40898, BN40900). ARR was assessed from randomization in parent studies to the first occurrence of the iPDR. PDR=occurrence of new/worsening neurological symptoms attributable to NMO/NMOSD. New or worsening neurological symptoms that occur < 31 days following onset of a PDR were considered part of the same relapse. Time point of relapse onset=the time at which the participant experienced any new or worsening neurological symptoms representing NMOSD clinical relapse(s). 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 ARR data from time of randomization in the parent studies up to the end of study WN42349 for satralizumab-treated participants are reported here.
Measured: Baseline up to 528 weeks
Change in Expanded Disability Status Scale (EDSS) Score
EDSS was a quantitative measure of disability and for assessment of severity of relapse for participants with NMOSD. Values from 0 points (normal neurological examination) up to 10 points (death), increasing in increments of 0.5 points. Higher scores represent increased disability. Baseline is the last observation on or before the day of first study drug administration in the current study or the parent studies (NCT02028884/NCT02073279). All EDSS data from the time of randomization in the parent studies up to the end of study WN42349 for satralizumab-treated participants are reported here.
Measured: Baseline and every 24 weeks (up to 528 weeks)
Time to First EDSS Scores Worsening
EDSS=quantitative measure of disability \& for assessment of severity of relapse for participants with NMOSD. Values from 0 points (normal neurological examination) up to 10 points (death), increasing in increments of 0.5 points. Higher scores=increased disability. EDSS worsening=(a) worsening of 2/more points in EDSS score for participants with a baseline score (BS) of 0, (b) worsening of 1/more points in EDSS score for participants with a BS of 1 to 5, or (c) worsening of 0.5 points/more in EDSS score for participants with a BS of 5.5/more. Participants were censored at date of last EDSS assessment or if no EDSS assessment was performed at randomization date. Baseline=last observation on/before day of first study drug administration in current study/parent studies (NCT02028884/NCT02073279). All EDSS data from time of randomization in parent studies up to end of study WN42349 for satralizumab-treated participants are reported here.
Measured: Baseline up to 528 weeks
Event-free Rate for EDSS Score Worsening up to Week 456
Event-free rate for EDSS score worsening was defined as the percentage of participants who did not experience worsening in their EDSS score from baseline. EDSS=quantitative measure of disability \& for assessment of severity of relapse for participants with NMOSD. Values from 0 points (normal neurological examination) up to 10 points (death), increasing in increments of 0.5 points. Higher scores=increased disability. Participants were censored at the date of the last EDSS assessment or if no EDSS assessment was performed at the randomization date. Baseline is defined as the last observation on/before the day of the first study drug administration in current study/parent studies (NCT02028884/NCT02073279). All EDSS data from the time of randomization in the parent studies up to Week 456 for satralizumab-treated participants are reported here. Kaplan-Meier method was used to estimate the event-free rates.
Measured: Baseline up to Week 456
Percentage of Participants Without EDSS Worsening
EDSS=quantitative measure of disability \& for assessment of severity of relapse for participants with NMOSD. Values from 0 points (normal neurological examination) up to 10 points (death), increasing in increments of 0.5 points. Higher scores=increased disability. EDSS worsening=(a) worsening of 2/more points in EDSS score for participants with a BS of 0, (b) worsening of 1/more points in EDSS score for participants with a BS of 1 to 5, or (c) worsening of 0.5 points/more in EDSS score for participants with a BS of 5.5/more. Baseline=last observation on/before day of first study drug administration in current study/parent studies (NCT02028884/NCT02073279). All EDSS data from time of randomization in parent studies up to end of study WN42349 for satralizumab-treated participants are reported here. Participants who did not experience any EDDS worsening events are reported here. Percentages have been rounded off to the nearest decimal point.
Measured: Baseline up to 528 weeks
Change in Visual Acuity (VA) Assessed by a Snellen 20-Foot Wall Chart
Visual acuity is measured using Snellen 20-foot wall chart and then converted to logMAR visual acuity scoring. Lower values indicate better visual acuity. Data are reported for right eye (OD) and left eye (OS). Visual acuity scores (Snellen chart) worse than 20/200 [i.e. CF (counting fingers), HM (hand movement), LP (light perception), or NLP (no LP)] are converted to logMAR 1.85, logMAR 2.00, logMAR 2.70, and logMAR 3.00 respectively. LogMAR >= 1 is equivalent to Physically blind. A negative change from baseline indicates an improvement. 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 VA data from the time of randomization in the parent studies up to the end of study WN42349 for satralizumab-treated participants are reported here.
Measured: Baseline and every 24 weeks (up to 528 weeks)
Concentrations of Interleukin-6 (IL-6) in Blood
Baseline was defined as last observation collected on or before day of first study drug administration in parent studies (NCT02028884/NCT02073279). All data from the time of randomization in the parent studies up to the end of study WN42349 for satralizumab-treated participants are reported here.
Measured: Baseline, every two weeks from Weeks 2 to 8; every 4 weeks from Weeks 12 to 192; every 24 weeks from Weeks 216 to 528
Concentrations of Soluble IL-6 Receptor (sIL-6R) in Blood
Baseline was defined as last observation collected on or before day of first study drug administration in parent studies (NCT02028884/NCT02073279). All data from the time of randomization in the parent studies up to the end of study WN42349 for satralizumab-treated participants are reported here.
Measured: Baseline, every two weeks from Weeks 2 to 8; every 4 weeks from Weeks 12 to 192; every 24 weeks from Weeks 216 to 528
Concentration of C-Reactive Protein (CRP) in Blood
Baseline was defined as last observation collected on or before day of first study drug administration in parent studies (NCT02028884/NCT02073279). All data from the time of randomization in the parent studies up to the end of study WN42349 for satralizumab-treated participants are reported here.
Measured: Baseline, every two weeks from Weeks 2 to 8; every 4 weeks from Weeks 12 to 192; every 24 weeks from Weeks 216 to 528
Serum Concentration of Satralizumab at Specified Timepoints
Baseline was defined as last observation collected on or before day of first study drug administration in parent studies (NCT02028884/NCT02073279). All data from the time of randomization in the parent studies up to the end of study WN42349 for satralizumab-treated participants are reported here.
Measured: Baseline, Week 2, Week 4, Week 5, Week 6; every 4 weeks from Weeks 8 to 192; every 24 weeks from Weeks 216 to 528
Number of Participants With Anti-Drug Antibodies (ADAs) From the First Dose of Satralizumab in Studies NCT02028884 or NCT02073279
The number and percentage of ADA-positive participants and ADA-negative participants at baseline (baseline prevalence) and after drug administration (post-baseline incidence) were summarized. Baseline evaluable participants were participants with an ADA assay result at baseline. Post-baseline evaluable participants were participants with an ADA assay from at least one post-baseline sample. Participants positive for ADA= number of participants with positive ADA result. Participants negative for ADA=number of participants with negative or missing baseline ADA result(s) and all negative post-baseline results. Baseline was defined as last observation collected on or before day of first study drug administration in parent studies. All data from the time of randomization in the parent studies up to the end of study WN42349 for satralizumab-treated participants are reported here.
Measured: First dose of satralizumab in parent studies up to end of study WN42349 (up to 523 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2020
- Study start2 Mar 2021
- Primary completion28 May 2024
- Study completion28 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Neuromyelitis Optica Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 119 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04660539
- Sponsor's protocol ID
- WN42349
- Other IDs
- 2020-003413-35
- Status last verified
- Nov 2024
- First submitted
- 24 Nov 2020
- Last updated
- 27 Dec 2024
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research\_and\_development/who\_we\_are\_how\_we\_work/clinical\_trials/our\_commitment\_to\_data\_sharing.htm).
Publications
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.
PubMed 40743487
Study at a glance
From the registry record, last updated by the sponsor on 27 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Single Arm, Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Satralizumab in Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)
- ClinicalTrials.gov ID
- NCT04660539
- Acronym / sponsor ID
- WN42349
- Conditions
- Neuromyelitis Optica Spectrum Disorder
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 119 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 2 Mar 2021 (actual)
- Primary completion
- 28 May 2024 (actual)
- Study completion
- 28 May 2024 (actual)
- First posted
- 9 Dec 2020 (actual)
- Results
- Posted 27 Dec 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Satralizumab Treatment | Experimental | Drug: satralizumab, Drug: azathioprine (AZA), Drug: mycophenolate mofetil (MMF), Drug: oral corticosteroidsParticipants will receive satralizumab subcutaneously (SC) every 4 weeks (Q4W) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| satralizumabAlso: EnspryngSatralizumab 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 | Drug | Satralizumab Treatment |
| azathioprine (AZA)Also: non-investigational medicinal product (NIMP)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) | Drug | Satralizumab Treatment |
| mycophenolate mofetil (MMF)Also: NIMPParticipants are permitted to use MMF during the study as background immunosuppressive treatment at a maximum dose of 3000 mg/day | Drug | Satralizumab Treatment |
| oral corticosteroidsAlso: NIMPParticipants are permitted to use oral corticosteroids (prednisolone equivalent) during the study as background immunosuppressive treatment at a maximum dose of 15 mg/day | Drug | Satralizumab Treatment |
Primary outcome
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Baseline up to 523 weeks
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.
Number of Participants With Adverse Events of Special Interest (AESIs) and Selected AEs
Time frame: Baseline up to 523 weeks
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.
16 secondary outcomes
Number of Participants With Suicidality Assessed Using Columbia-Suicide Severity Rating Scale (C-SSRS)
Time frame: Baseline up to 523 weeks
Number of Participants With Serious Infections and Hepatotoxicity
Time frame: Baseline up to 523 weeks
Time to First Protocol-defined Relapse (PDR) as Assessed by Investigator (iPDR)
Time frame: Baseline up to 528 weeks
iPDR-free Rate up to Week 456
Time frame: Baseline up to Week 456
Percentage of Relapse-Free Participants
Time frame: Baseline up to 528 weeks
Annualized Relapse Rate (ARR)
Time frame: Baseline up to 528 weeks
Change in Expanded Disability Status Scale (EDSS) Score
Time frame: Baseline and every 24 weeks (up to 528 weeks)
Time to First EDSS Scores Worsening
Time frame: Baseline up to 528 weeks
Event-free Rate for EDSS Score Worsening up to Week 456
Time frame: Baseline up to Week 456
Percentage of Participants Without EDSS Worsening
Time frame: Baseline up to 528 weeks
Change in Visual Acuity (VA) Assessed by a Snellen 20-Foot Wall Chart
Time frame: Baseline and every 24 weeks (up to 528 weeks)
Concentrations of Interleukin-6 (IL-6) in Blood
Time frame: Baseline, every two weeks from Weeks 2 to 8; every 4 weeks from Weeks 12 to 192; every 24 weeks from Weeks 216 to 528
Concentrations of Soluble IL-6 Receptor (sIL-6R) in Blood
Time frame: Baseline, every two weeks from Weeks 2 to 8; every 4 weeks from Weeks 12 to 192; every 24 weeks from Weeks 216 to 528
Concentration of C-Reactive Protein (CRP) in Blood
Time frame: Baseline, every two weeks from Weeks 2 to 8; every 4 weeks from Weeks 12 to 192; every 24 weeks from Weeks 216 to 528
Serum Concentration of Satralizumab at Specified Timepoints
Time frame: Baseline, Week 2, Week 4, Week 5, Week 6; every 4 weeks from Weeks 8 to 192; every 24 weeks from Weeks 216 to 528
Number of Participants With Anti-Drug Antibodies (ADAs) From the First Dose of Satralizumab in Studies NCT02028884 or NCT02073279
Time frame: First dose of satralizumab in parent studies up to end of study WN42349 (up to 523 weeks)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Dec 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40743487Bennett 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.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
53 sites in 18 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing satralizumab, azathioprine (AZA) and mycophenolate mofetil (MMF) in people with Neuromyelitis Optica Spectrum Disorder, run by Hoffmann-La Roche at 53 sites in 18 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 53 sites in United States, Japan, Poland, Taiwan, Ukraine and 13 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Dec 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Baseline up to 523 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04660539, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Dec 2024.