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Home › Conditions › Neuromyelitis Optica

Neuromyelitis Optica

Part of Demyelinating Autoimmune Diseases, CNS

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for Neuromyelitis Optica

10 trials tracked for Neuromyelitis Optica: 2 in phase 3 or 4 and 2 with published results. The most-cited published study has 78 citations.

10Trials tracked
2Phase 3 & 4
0Recruiting
2With published results
Phase distribution
Phase 3 2 Phase 2 4 Phase 1 3 Other / NA 1
  1. Phase 3 An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients Completed · 78 cited
  2. Phase 3 An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD Completed · 9 cited
  3. Phase 2 An Open Label Study of the Effects of Eculizumab in Neuromyelitis Optica Completed
  4. Phase 2 Neuromyelitis Optica (NMO) & Cetirizine Completed
  5. Phase 2 Safety and Efficacy of Sustained Release Dalfampridine in Transverse Myelitis (Re-Launch) Completed
  6. Phase 2 High Frequency Impulse Therapy for Neuropathic Pain in NMOSD Completed
Show 4 more trials
  1. Phase 1 C1-esterase Inhibitor (Cinryze) for Acute Treatment of Neuromyelitis Optica Exacerbation Completed
  2. Phase 1 Ublituximab for Acute Neuromyelitis Optica (NMO) Relapses Completed
  3. Phase 1 Efficacy of Bevacizumab (Avastin) in Treatment of Acute NMO Exacerbations Completed
  4. N/A Scrambler Trial for Pain in NMOSD Completed

Showing the 10 most-cited and recently-updated of 10 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: what the trials found

Eculizumab has demonstrated significant efficacy in reducing the frequency of Neuromyelitis Optica (NMO) attacks, with a median of 0 attacks per year compared to 3 in the control group (p<0.0001) 6. Treatment with eculizumab was also associated with a statistically significant improvement in Expanded Disability Status Scale (EDSS) scores (p=0.0078) 6.

Ravulizumab significantly reduced the number of adjudicated on-trial relapses during the primary treatment period compared to the control group (p<0.0001) 2. Additionally, ravulizumab was associated with a clinically important change from baseline in Hauser Ambulation Index (HAI) scores at the end of the primary treatment period (p=0.0122) 2.

Recent results — preliminary, needs further review

  • High Frequency Impulse Therapy showed a reduction in pain scores on the Numerical Rating Scale (NRS) and a decrease in 'worst' pain scores 3.
  • Ublituximab was evaluated for changes in neurological disability (EDSS), though results are not yet corroborated 7.
  • Bevacizumab was assessed in patients with an initial EDSS of 3.5, showing a follow-up score of 3.0 8.
  • C1-esterase inhibitor (Cinryze) showed no subjects withdrawing due to adverse events in a small cohort 9.

For the clinician treating this condition

  • Eculizumab is effective in reducing the annual number of NMO attacks and improving EDSS scores 6.
  • Ravulizumab significantly reduces on-trial relapses and improves Hauser Ambulation Index (HAI) scores 2.

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.
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

Research across Demyelinating Autoimmune Diseases, CNS

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