Phase 3
N=166
A Study to Evaluate the Safety and Efficacy of Satralizumab in Participants With Neuromyelitis Optica Spectrum Disorder (NMOSD)
Neuromyelitis Optica Spectrum Disorder
Bottom Line
View on ClinicalTrials.gov: NCT04660539 ↗Enrolled (actual)
166
Serious AEs
26.5%
Results posted
Dec 2024
Primary outcome: Primary: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 162; 44 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- satralizumab (Drug); azathioprine (AZA) (Drug); mycophenolate mofetil (MMF) (Drug); oral corticosteroids (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Hoffmann-La Roche
- Primary completion
- May 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
162; 44 | — |
| PRIMARY Number of Participants With Adverse Events of Special Interest (AESIs) and Selected AEs |
0; 0 | — |
| SECONDARY Number of Participants With Suicidality Assessed Using Columbia-Suicide Severity Rating Scale (C-SSRS) |
7; 5; 5; 2; 1; 0 | — |
| SECONDARY Number of Participants With Serious Infections and Hepatotoxicity |
19; 8 | — |
| SECONDARY Time to First Protocol-defined Relapse (PDR) as Assessed by Investigator (iPDR) |
NA | — |
| SECONDARY iPDR-free Rate up to Week 456 |
67.11 | — |
| SECONDARY Percentage of Relapse-Free Participants |
70.5 | — |
| SECONDARY Annualized Relapse Rate (ARR) |
0.0788 | — |
| SECONDARY Change in Expanded Disability Status Scale (EDSS) Score |
3.80; -0.08; -0.16; -0.14; -0.18; -0.14 | — |
| SECONDARY Time to First EDSS Scores Worsening |
NA | — |
| SECONDARY Event-free Rate for EDSS Score Worsening up to Week 456 |
57.03 | — |
| SECONDARY Percentage of Participants Without EDSS Worsening |
64.8 | — |
| SECONDARY Change in Visual Acuity (VA) Assessed by a Snellen 20-Foot Wall Chart |
0.438; 0.566; 0.028; 0.009; 0.014; 0.020 | — |
| SECONDARY Concentrations of Interleukin-6 (IL-6) in Blood |
2.07; 21.27; 21.90; 19.35; 18.84; 17.53 | — |
| SECONDARY Concentrations of Soluble IL-6 Receptor (sIL-6R) in Blood |
32.27; 404.20; 536.46; 566.91; 568.96; 561.69 | — |
| SECONDARY Concentration of C-Reactive Protein (CRP) in Blood |
1.27; 0.38; 0.36; 0.39; 0.42; 0.42 | — |
| SECONDARY Serum Concentration of Satralizumab at Specified Timepoints |
103.75; 7489.76; 14631.06; 20068.86; 17495.09; 12673.14 | — |
| SECONDARY Number of Participants With Anti-Drug Antibodies (ADAs) From the First Dose of Satralizumab in Studies NCT02028884 or NCT02073279 |
3; 161; 99; 67 | — |
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
Data sourced from ClinicalTrials.gov (NCT04660539). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.