Eculizumab For the Acute Attack of Neuromyelitis Optica Spectrum Disorder (EASE-NMO)

Tianjin Medical University

Status and phase: Enrolling; Phase 2

Conditions: Neuromyelitis Optica Spectrum Disorder Attack

Treatments: Drug: Intravenous Methylprednisolone (IVMP); Drug: Complement protein C5 inhibitor

Study type: Interventional

Funder types: Other

Identifiers

NCT07184840

IRB2024-YX-456-01

Details and patient eligibility

About

Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing, inflammatory autoimmune disorder of the central nervous system characterized by the pathogenic anti-aquaporin 4 antibody (AQP4-IgG). The objectives of this study are to assess the efficacy and safety of eculizumab for treatment of patients with neuromyelitis optica spectrum disorders during acute phase who are anti-aquaporin-4 (AQP4) antibody-positive. Eculizumab, a humanized monoclonal antibody, inhibits the terminal complement protein C5 and prevents its cleavage into C5a and the formation of C5b-9 (MAC), has approved for preventive treatment of NMOSD. Given the high efficacy of C5 inhibition, eculizumab is proposed to potentially provide rapid relief from astrocyte destruction by reducing MAC formation, which could contribute to the fast alleviation of neurological deficit during NMO acute attack. The potential of eculizumab warrants further investigation as a treatment for acute neuromyelitis optica spectrum disorders attacks.

Enrollment: 110 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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110 participants in 2 patient groups

Intravenous Methylprednisolone (IVMP) arm

Other group

Description:

IVMP arm: 1000mg methylprednisolone x5d, oral prednisone 60mg, 5mg weekly decline + antibiotics

Treatment:

Drug: Intravenous Methylprednisolone (IVMP)

IVMP+Eculizumab arm

Experimental group

Description:

IVMP+Eculizumab arm: eculizumab (900 mg) will be administered intravenously once per week for a total of four doses (days 1, 8, 15, and 22) in conjunction with IVMP and oral prednisone (60mg, 5mg weekly decline). All enrolled patients will receive antibiotic prophylaxis against N meningitidis.

Treatment:

Drug: Complement protein C5 inhibitor

Drug: Intravenous Methylprednisolone (IVMP)