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

• ClinicalTrials.gov Identifier: NCT04660539
• Recruitment Status: Recruiting
• First Posted: December 9, 2020
• Last Update Posted: January 11, 2022
• Sponsor: Hoffmann-La Roche
• Information provided by (Responsible Party): Hoffmann-La Roche

Tracking Information

• First Submitted Date: November 24, 2020
• First Posted Date: December 9, 2020
• Last Update Posted Date: January 11, 2022
• Actual Study Start Date: March 2, 2021
• Estimated Primary Completion Date: April 30, 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 7, 2020)

• Percentage of Participants with Adverse Events (AE) AEs of Special Interest (AESI), Serious AEs (SAE), and Selected AEs [ Time Frame: Up to 39 Months ]
• Columbia-Suicide Severity Rating Scale (C-SSRS) Scores [ Time Frame: Up to 39 Months ]

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: December 7, 2020)

• Percentage of Participants with Serious Infections and Hepatotoxicity [ Time Frame: Up to 39 Months ]
  Participants with NMOSD who are treated with satralizumab
• Time to First Relapse (TFR) [ Time Frame: Up to 39 Months ]
• Percentage of Relapse-Free Participants [ Time Frame: Up to 39 Months ]
• Annualized Relapse Rate (ARR) [ Time Frame: Up to 39 Months ]
• Change in Expanded Disability Status Scale (EDSS) Score [ Time Frame: Up to 39 Months ]
• Time to EDSS Worsening [ Time Frame: Up to 39 Months ]
• Percentage of Participants without EDSS Worsening [ Time Frame: Up to 39 Months ]
• Change in Visual Acuity Assessed by a Snellen 20-Foot Wall Chart [ Time Frame: Up to 39 Months ]
  The test will be performed monocularly and participants may use their habitual distance glasses or contact lenses
• Concentrations of Interleukin-6 (IL-6) and Soluble IL-6 Receptor (sIL-6R) in Blood [ Time Frame: Up to 39 Months ]
  Measured in picogram per milliliter (pg/mL)
• Concentration of C-Reactive Protein (CRP) in Blood [ Time Frame: Up to 39 Months ]
  Measured in milligram per liter (mg/L)
• Serum Concentration of Satralizumab at Specified Timepoints [ Time Frame: 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) [ Time Frame: Up to 39 Months ]
• Percentage of Participants with ADA to Satralizumab [ Time Frame: Up to 39 Months ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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)
• Brief Summary: This multicenter, single-arm, open-label study will evaluate the long-term safety and efficacy of satralizumab in patrticipants 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Neuromyelitis Optica Spectrum Disorder

Intervention

• Drug: satralizumab
  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
  Other Name: Enspryng
• Drug: azathioprine (AZA)
  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)
  Other Name: non-investigational medicinal product (NIMP)
• Drug: mycophenolate mofetil (MMF)
  Participants are permitted to use MMF during the study as background immunosuppressive treatment at a maximum dose of 3000 mg/day
  Other Name: NIMP
• Drug: oral corticosteroids
  Participants are permitted to use oral corticosteroids (prednisolone equivalent) during the study as background immunosuppressive treatment at a maximum dose of 15 mg/day
  Other Name: NIMP

Study Arms

Experimental: Satralizumab Treatment

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

Interventions:

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

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: December 7, 2020): 127
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: April 30, 2024
• Estimated Primary Completion Date: April 30, 2024 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Reference Study ID Number: WN42349 www.roche.com/about_roche/roche_worldwide.htm; 888-662-6728 (U.S. and Canada); global-roche-genentech-trials@gene.com

• Listed Location Countries: Bulgaria, Canada, Croatia, Germany, Hungary, Italy, Japan, Korea, Republic of, Malaysia, Poland, Puerto Rico, Romania, Spain, Taiwan, Ukraine, United Kingdom, United States
• Removed Location Countries: Turkey

Administrative Information

• NCT Number: NCT04660539
• Other Study ID Numbers: WN42349; 2020-003413-35 ( EudraCT Number )
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Yes
• Plan Description: 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).

• Responsible Party: Hoffmann-La Roche
• Study Sponsor: Hoffmann-La Roche
• Collaborators: Not Provided

Investigators

• Study Director: Clinical Trials; Hoffmann-La Roche

• PRS Account: Hoffmann-La Roche
• Verification Date: January 2022