Eculizumab for acute AQP4‑positive neuromyelitis optica attacks

Eculizumab For Acute Attack of Neuromyelitis Optica Spectrum Disorder (NMOSD): a Multi-Center, Phase 2 Trial (EASE-NMO)

This trial will test if giving eculizumab together with high‑dose steroids helps adults with AQP4‑positive NMOSD recover faster from an acute optic neuritis or transverse myelitis attack.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 110 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Tianjin Medical University General Hospital (Academic / other)
• Drugs / interventions: eculizumab
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT07184840 on ClinicalTrials.gov

What this trial studies

This is a phase 2 interventional trial enrolling adults with anti‑AQP4 antibody‑positive NMOSD who present within 28 days of an acute optic neuritis and/or transverse myelitis attack. Participants receive eculizumab, a complement C5 inhibitor, in the acute phase in addition to standard high‑dose intravenous methylprednisolone to see whether blocking terminal complement reduces astrocyte injury and hastens neurological recovery. Key eligibility includes age ≥18, body weight ≥35 kg, and a documented worsening in neurological exam (OSIS increase ≥2) compared with the prior attack; major exclusions include prior eculizumab, recent IVIg, and unresolved significant infections. The trial is conducted at Tianjin Medical University General Hospital and focuses on safety and efficacy signals to inform larger studies.

Who should consider this trial

Good fit: Ideal candidates are adults (≥18 years, ≥35 kg) who are anti‑AQP4 antibody positive and present within 28 days of an acute optic neuritis or transverse myelitis attack with an OSIS increase of at least 2 points and who are not pregnant or breastfeeding.

Not a fit: Patients who are AQP4‑negative, present after 28 days from the attack, have active unresolved infections, recent IVIg, prior eculizumab exposure, or other exclusionary conditions are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, the approach could shorten attack severity and speed recovery, potentially reducing the risk of permanent disability from NMOSD attacks.

How similar studies have performed: Eculizumab has shown strong benefit as a preventive treatment for AQP4‑positive NMOSD in prior trials, but using C5 inhibition specifically during the acute attack is novel and has limited direct clinical evidence.

Eligibility criteria

Inclusion Criteria:

1. Anti-AQP4 antibody seropositive.
2. Male or female patients ≥18 years old
3. Body weight ≥ 35 kg
4. Acute optic neuritis and/or transverse myelitis enrolled within 28 days from the attack, with a change in neurological exam that meet an increase of OSIS at least 2 points of baseline compared to that of prior attack.
5. A female subject is eligible to enter the trial if she is:

   * Not pregnant or breastfeeding, not intending to conceive during the course of the trial

Exclusion Criteria:

1. Use of IVIg within 3 weeks prior to screening
2. Unresolved meningococcal infection
3. Any systemic bacterial or other infection which is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics
4. Participation in any other investigational drug study or was exposed to an investigational drug or device within 30 days of screening.
5. Has previously received treatment with eculizumab
6. Hypersensitivity to murine proteins or to one of the excipients of eculizumab
7. Any medical condition that, in the opinion of the Investigator, might interfere with thepatient's participation in the trial, poses any added risk for the patient, or confounds the assessment of the patients

Where this trial is running

Tianjin, Tianjin Municipality

• Tianjin Medical University General Hospital — Tianjin, Tianjin Municipality, China (Recruiting)

Study contacts

• Study coordinator: Fu-Dong Shi

Conditions Neuromyelitis Optica Spectrum Disorder Attack; Eculizumab; Acute Attack; Neuromyelitis Optica Spectrum Disorder