Clinical trial NCT04660539: A Study to Evaluate the Safety and Efficacy of Satralizumab in Participants With Neuromyelitis Optica Spectrum Disorder (NMOSD)
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Neuromyelitis Optica Spectrum Disorder
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2020-12-09
Enrollment: 119
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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

Primary outcomes:
measure: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline up to 523 weeks
measure: Number of Participants With Adverse Events of Special Interest (AESIs) and Selected AEs
timeFrame: Baseline up to 523 weeks
Secondary outcomes:
measure: Number of Participants With Suicidality Assessed Using Columbia-Suicide Severity Rating Scale (C-SSRS)
timeFrame: Baseline up to 523 weeks
measure: Number of Participants With Serious Infections and Hepatotoxicity
timeFrame: Baseline up to 523 weeks
measure: Time to First Protocol-defined Relapse (PDR) as Assessed by Investigator (iPDR)
timeFrame: Baseline up to 528 weeks
measure: iPDR-free Rate up to Week 456
timeFrame: Baseline up to Week 456
measure: Percentage of Relapse-Free Participants
timeFrame: Baseline up to 528 weeks
measure: Annualized Relapse Rate (ARR)
timeFrame: Baseline up to 528 weeks
measure: Change in Expanded Disability Status Scale (EDSS) Score
timeFrame: Baseline and every 24 weeks (up to 528 weeks)
measure: Time to First EDSS Scores Worsening
timeFrame: Baseline up to 528 weeks
measure: Event-free Rate for EDSS Score Worsening up to Week 456
timeFrame: Baseline up to Week 456
measure: Percentage of Participants Without EDSS Worsening
timeFrame: Baseline up to 528 weeks
measure: Change in Visual Acuity (VA) Assessed by a Snellen 20-Foot Wall Chart
timeFrame: Baseline and every 24 weeks (up to 528 weeks)
measure: Concentrations of Interleukin-6 (IL-6) in Blood
timeFrame: 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
measure: Concentrations of Soluble IL-6 Receptor (sIL-6R) in Blood
timeFrame: 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
measure: Concentration of C-Reactive Protein (CRP) in Blood
timeFrame: 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
measure: Serum Concentration of Satralizumab at Specified Timepoints
timeFrame: 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
measure: Number of Participants With Anti-Drug Antibodies (ADAs) From the First Dose of Satralizumab in Studies NCT02028884 or NCT02073279
timeFrame: First dose of satralizumab in parent studies up to end of study WN42349 (up to 523 weeks)
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Children's Hospital of Alabama
state: Alabama
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Columbus
country: United States
facility: Columbus Research and Wellness
state: Georgia
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Northbrook
country: United States
facility: Consultants in Neurology Ltd
state: Illinois
city: Peoria
country: United States
facility: OSF Saint Francis Medical Center
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Detroit
country: United States
facility: Wayne State University; UHC-4H
state: Michigan
city: Charlotte
country: United States
facility: The Neurological Institute PA
state: North Carolina
city: Columbus
country: United States
facility: OhioHealth Research Institute
state: Ohio
city: Philadelphia
country: United States
facility: Jefferson Hospital For Neuroscience; Jefferson Neurology Associates
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Round Rock
country: United States
facility: Central Texas Neurology Consultants
state: Texas
city: Pleven
country: Bulgaria
facility: UMHAT 'Dr. Georgi Stranski', EAD
city: Sofia
country: Bulgaria
facility: Multiprofile Hospital for Active Treatment of Neurology and Psychiatry Sv. Naum EAD
city: Sofia
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
city: Vancouver
country: Canada
facility: MS Clinical Trials Group
state: British Columbia
city: Montreal
country: Canada
facility: Centre hospitalier de l'Universite de Montreal (CHUM)
state: Quebec
city: Osijek
country: Croatia
facility: Clinical Hospital Centre Osijek
city: Bochum
country: Germany
facility: Ruhr Universitat Bochum
city: Budapest
country: Hungary
facility: Jahn Ferenc Del-Pesti Korhaz es Rendelointezet
city: Roma
country: Italy
facility: Azienda Ospedaliera Sant'Andrea
state: Lazio
city: Catania
country: Italy
facility: Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele
state: Sicilia
city: Fukuoka
country: Japan
facility: Kyushu University Hospital
city: Miyagi
country: Japan
facility: Tohoku University Hospital
city: Niigata
country: Japan
facility: Niigata University Medical & Dental Hospital
city: Osaka
country: Japan
facility: Osaka University Hospital
city: Osaka
country: Japan
facility: Kindai University Hospital
city: Tokyo
country: Japan
facility: Tokyo Women's Medical University Hospital
city: Tokyo
country: Japan
facility: National Center of Neurology and Psychiatry
city: Seoul
country: Korea, Republic of
facility: Korea University Anam Hospital
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center - PPDS
city: Kuala Lumpur
country: Malaysia
facility: Hospital Kuala Lumpur
state: FED. Territory OF Kuala Lumpur
city: Katowice
country: Poland
facility: NZOZ Wielospecjalistyczna Poradnia Lekarska SYNAPSIS
city: Katowice
country: Poland
facility: M.A. - LEK A. M. Maciejowscy SC. Centrum Terapii SM
city: Lublin
country: Poland
facility: Samodzielny Publiczny Szpital Kliniczny Nr 4 w Lublinie; Klinika Neurologii
city: Warszawa
country: Poland
facility: Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny; Klinika Neurologii
city: Warszawa
country: Poland
facility: Instytut Psychiatrii i Neurologii
city: Warszawa
country: Poland
facility: Miedzyleski Szpital Specjalistyczny w Warszawie
city: Guaynabo
country: Puerto Rico
facility: San Juan MS Center
city: Campulung
country: Romania
facility: SC Clubul Sanatatii SRL
city: Barcelona
country: Spain
facility: Hospital Clinic de Barcelona
city: Madrid
country: Spain
facility: Hosp. Clinico San Carlos
city: North Dist.
country: Taiwan
facility: China Medical University Hospital
city: Tainan
country: Taiwan
facility: National Cheng Kung University Hospital
city: Taipei City
country: Taiwan
facility: Taipei Veterans General Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Istanbul
country: Turkey
facility: Bilim University Medical Faculty Florence Nightingale Hospital
city: Dnipro
country: Ukraine
facility: Municipal Non-Profit Enterprise City Clinical Hospital #16 of Dnipro City Council
state: Katerynoslav Governorate
city: Odesa
country: Ukraine
facility: Municipal Non-Commercial Enterprise Odesa RMC for Mental Health of Odessa Regional Council
state: Kherson Governorate
city: Vinnytsia
country: Ukraine
facility: Communal NPE Vinnytsia Reg. Clin. Psychoneurolog. Hosp. n.a. O.I. Yushchenko of Vinnytsia RC
state: Podolia Governorate
city: Ternopil
country: Ukraine
facility: Communal Nonprofit enterprise Ternopil Regional Clinical Psychoneurological Hospital of TRC
state: Volhynian Governorate
city: London
country: United Kingdom
facility: National Hospital For Neurology and Neurosurgery
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: