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

Study Overview
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Brief Summary
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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.

Official Title
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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)

Conditions
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Neuromyelitis Optica Spectrum Disorder

Intervention / Treatment
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* Drug: satralizumab
* Drug: azathioprine (AZA)
* Drug: mycophenolate mofetil (MMF)
* Drug: oral corticosteroids


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Satralizumab Treatment<br>Participants will receive satralizumab subcutaneously (SC) every 4 weeks (Q4W) | Drug: satralizumab<br>* 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<br>* Other names: Enspryng;Drug: azathioprine (AZA)<br>* 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)<br>* Other names: non-investigational medicinal product (NIMP);Drug: mycophenolate mofetil (MMF)<br>* Participants are permitted to use MMF during the study as background immunosuppressive treatment at a maximum dose of 3000 mg/day<br>* Other names: NIMP;Drug: oral corticosteroids<br>* Participants are permitted to use oral corticosteroids (prednisolone equivalent) during the study as background immunosuppressive treatment at a maximum dose of 15 mg/day<br>* Other names: NIMP;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants with Adverse Events (AE) AEs of Special Interest (AESI), Serious AEs (SAE), and Selected AEs |  | Up to 39 Months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Columbia-Suicide Severity Rating Scale (C-SSRS) Scores |  | Up to39 months | 
| Percentage of Participants with Serious Infections and Hepatotoxicity | Participants with NMOSD who are treated with satralizumab | Up to 39 Months | 
| Time to First Relapse (TFR) |  | Up to 39 Months | 
| Percentage of Relapse-Free Participants |  | Up to 39 Months | 
| Annualized Relapse Rate (ARR) |  | Up to 39 Months | 
| Change in Expanded Disability Status Scale (EDSS) Score |  | Up to 39 Months | 
| Time to EDSS Worsening |  | Up to 39 Months | 
| Percentage of Participants without EDSS Worsening |  | Up to 39 Months | 
| Change in Visual Acuity Assessed by a Snellen 20-Foot Wall Chart | The test will be performed monocularly and participants may use their habitual distance glasses or contact lenses | Up to 39 Months | 
| Concentrations of Interleukin-6 (IL-6) and Soluble IL-6 Receptor (sIL-6R) in Blood | Measured in picogram per milliliter (pg/mL) | Up to 39 Months | 
| Concentration of C-Reactive Protein (CRP) in Blood | Measured in milligram per liter (mg/L) | Up to 39 Months | 
| Serum Concentration of Satralizumab at Specified Timepoints |  | Up to 39 Months | 
| Percentage of Participants with Anti-Drug Antibodies (ADAs) from the First Dose of Satralizumab in Studies BN40898 or BN40900 (parent studies) |  | Up to 39 Months |