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Home › Conditions › neuromyelitis optica spectrum disorder

Neuromyelitis optica spectrum disorder

Part of Demyelinating Autoimmune Diseases, CNS

6 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for neuromyelitis optica spectrum disorder

6 trials tracked for neuromyelitis optica spectrum disorder: 4 in phase 3 or 4 and 4 with published results. The most-cited published study has 191 citations.

6Trials tracked
4Phase 3 & 4
0Recruiting
4With published results
Phase distribution
Phase 3 4 Phase 1 2
  1. Phase 3 Comparison of Clinical Effects of Azathioprine and Rituximab NMO-SD Patients Completed · 191 cited
  2. Phase 3 An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients Completed · 78 cited
  3. Phase 3 An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD Completed · 9 cited
  4. Phase 3 A Study to Evaluate the Safety and Efficacy of Satralizumab in Participants With Neuromyelitis Optica Spectrum Disorder (NMOSD) Completed · 5 cited
  5. Phase 1 Ublituximab for Acute Neuromyelitis Optica (NMO) Relapses Completed
  6. Phase 1 Efficacy of Bevacizumab (Avastin) in Treatment of Acute NMO Exacerbations Completed

Showing the 6 most-cited and recently-updated of 6 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Neuromyelitis optica spectrum disorder: what the trials found

Clinical evidence for neuromyelitis optica spectrum disorder (NMOSD) includes several targeted therapies with varying outcomes in disability and relapse prevention. Ravulizumab demonstrated significant efficacy in preventing relapses, with zero participants experiencing an adjudicated on-trial relapse compared to 20 in the control group (p<0.0001), while also showing a statistically significant reduction in clinically important changes in Hauser Ambulation Index (HAI) scores (p=0.0122) 3.

Satralizumab showed an event-free rate for EDSS score worsening of 57.03% up to week 456, with a reported relapse-free participant percentage of 70.5% 4. Eculizumab was evaluated for its impact on disability and quality of life; however, the data provided for mean changes in EDSS, mRS, and EQ-5D VAS scores across treatment arms were not statistically significant or showed minimal variance from baseline 2.

Azathiopurine has been investigated as a treatment option within Phase 3 clinical trials for this condition 1.

Recent results — preliminary, needs further review

  • Ublituximab: Preliminary Phase 1 data observed changes in EDSS scores (4.0, 6.5, 6.5, 4.0) but lack large-scale confirmation 5.
  • Bevacizumab: Phase 1 data showed a mean baseline EDSS of 3.5 with a follow-up EDSS of 3.0 in a small cohort 6.

For the clinician treating this condition

  • Ravulizumab is associated with high efficacy in preventing relapses and maintaining ambulatory function (HAI scores) in NMOSD patients.
  • Satralizumab demonstrates a stable profile regarding EDSS progression over an extended period (up to 456 weeks).
  • Eculizumab and Azathiopurine are established interventions, though specific magnitude of improvement for eculizumab relative to control was not statistically significant in the reported data.

AI synthesis of 6 cited trials, updated Jun 20, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Optical coherence tomography shows significantly reduced retinal measurements in neuromyelitis optica spectrum disorder compared to multiple sclerosis
Neurology Meta-analysis
Optical coherence tomography shows significantly reduced retinal measurements in neuromyelitis optica spectrum disorder compared to multiple sclerosis Retinal Scans Show Distinct Differences Between MS and NMOSD
This systematic review and meta-analysis analyzed OCT measurements in patients with multiple sclerosis (PwMS) and neuromyelitis optica spect…
New analysis shows that eye scans reveal significant differences in retinal thickness and volume between people with MS and those with NMOSD…
Jul 1, 2026
Diabetes mellitus acts as a context-dependent modifier of risk and phenotype in neurological disorders
Neurology Sys. Review
Diabetes mellitus acts as a context-dependent modifier of risk and phenotype in neurological disorders Diabetes May Influence Risk and Progress of Neurological Disorders
This narrative review synthesizes evidence regarding the role of diabetes mellitus (DM) as a disease modifier across various neurological co…
New research suggests that diabetes can change how certain neurological conditions progress, potentially opening doors for new treatment opt…
Frontiers Jun 26, 2026
Case report and literature review of severe adverse reactions following pucotenlimab treatment
Allergy & Immunology Sys. Review
Case report and literature review of severe adverse reactions following pucotenlimab treatment New drug treatment linked to severe neurological reactions
This case report and literature review describes severe adverse reactions following treatment with pucotenlimab in a patient with advanced b…
A single patient facing advanced breast cancer experienced life-threatening neurological symptoms after receiving the drug pucotenlimab.
Frontiers Apr 30, 2026
Switching therapy after rituximab resistance in AQP4-IgG+ NMOSD shows variable relapse outcomes
Oncology Cohort
Switching therapy after rituximab resistance in AQP4-IgG+ NMOSD shows variable relapse outcomes New Hope for Tough-to-Treat Nerve Disorder
This single-center, retrospective case series of 10 adults with AQP4-IgG+ NMOSD who were resistant to rituximab evaluated switching therapy.
Patients with a rare nerve disease who stopped responding to standard treatment found relief after switching to new therapies that prevented…
Frontiers Apr 19, 2026
Narrative review of animal models in neuromyelitis optica spectrum disorder pathogenesis
Rheumatology Sys. Review
Narrative review of animal models in neuromyelitis optica spectrum disorder pathogenesis Animal Models Are Unlocking New Treatments for Rare Brain Disease
This narrative review examines animal models used to study neuromyelitis optica spectrum disorder pathogenesis.
Better animal models are helping scientists understand how NMOSD damages the brain, offering hope for new treatments for patients with few o…
Frontiers Apr 18, 2026
Phase 1b trial of intravenous B001 shows favorable safety in aquaporin-4-positive NMOSD patients
Allergy & Immunology Phase II
Phase 1b trial of intravenous B001 shows favorable safety in aquaporin-4-positive NMOSD patients A New MS-Like Disease Drug Passes Its First Human Safety Test
This Phase 1b randomized, double-blind, placebo-controlled trial evaluated intravenous B001 in 22 patients with aquaporin-4 immunoglobulin G…
A new drug for a rare nerve disease that can cause blindness and paralysis passed its first human safety test with no severe side effects se…
Frontiers Apr 18, 2026

Research across Demyelinating Autoimmune Diseases, CNS

Related studies from across the Demyelinating Autoimmune Diseases, CNS family.

Observational study abstract reports AI RNA sequencing diagnostic accuracy in multiple sclerosis and neuromyelitis optica
Genetics & Precision Medicine
Observational study abstract reports AI RNA sequencing diagnostic accuracy in multiple sclerosis and neuromyelitis optica Artificial Intelligence identifies early Multiple Sclerosis and separates it from others
This abstract describes an observational diagnostic accuracy study involving 997 participants.
Artificial Intelligence analyzes blood cells to tell early Multiple Sclerosis apart from healthy people and similar conditions like Neuromye…
medRxiv Apr 22, 2026

Questions about neuromyelitis optica spectrum disorder

What animal models help researchers understand NMOSD pathogenesis?

Animal models for NMOSD include AQP4-peptide immunized mice, passive transfer models, and T-cell-based models, each capturing different aspects of pathogenesis.

Full answer →
Can pucotenlimab cause severe adverse reactions in patients?

Yes, pucotenlimab has caused severe adverse reactions, including a fatal case of neuromyelitis optica spectrum disorder (NMOSD) in a patient with cancer.

Full answer →