Efficacy and Safety of Monoclonal Antibody in Acute Phase of Neuromyelitis Optica Spectrum Disorder

NCT07182409 | Not Yet Recruiting

• 1 other identifier: MAAP-NMO-001
• Study Type: observational
• Target: 40
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study aims to evaluate the efficacy and safety of different monoclonal antibody in the acute phase of neuromyelitis optica spectrum disorder (MAAP-NMO). It will also examine immune-related biomarkers and their relationship with treatment response to provide evidence for optimizing acute-phase therapeutic strategies.

Conditions

NMOSD

Keywords

NMOSD; Intravenous methylprednisolone; Efgartigimod; Eculizumab; Plasma exchange

Outcome Measures

Primary Outcomes (1)

• Change in Expanded Disability Status Scale (EDSS) score

  Change in Expanded Disability Status Scale (EDSS) score from baseline to 1 months after treatment (EDSS: Minimum Score 1, Maximum score 10, higher scores mean a worse outcome).

  1 months

Secondary Outcomes (13)

• Change in Expanded Disability Status Scale (EDSS) score

  3 and 6 months

• Percentage of Participants with Improvement

  1, 3 and 6 months

• Change in Low-Contrast Visual Acuity (LCVA)

  1, 3 and 6 months

• Percentage of Participants without relapse

  1, 3 and 6 months

• MRI changes after immunotherapy

  1 and 6 months

Study Arms (4)

IVMP group

Methylprednisolone, intravenous infusion, 1000 mg/day for 5 consecutive days.

IVMP + Plasma Exchange (PE) group

IVMP combined with plasma exchange, 2000-3000 mL per session, once every 1-2 days, for a total of 3-5 sessions.

IVMP + Efgartigimod group

IVMP combined with efgartigimod, intravenous infusion, 10 mg/kg once weekly for 4 weeks.

IVMP + Eculizumab group

IVMP combined with eculizumab, intravenous infusion of 900 mg once weekly for 4 weeks.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

NMOSD patients with acute attacks

You may qualify if:

• Age at onset ≥18 years, any gender.
• Patients meeting the 2015 International Panel for NMO Diagnosis (IPND) criteria for NMOSD and currently in the acute phase, defined as new or significantly worsened neurological deficits lasting \>24 hours, with onset within \<14 days, excluding pseudo-relapses caused by fever, infection, or metabolic disturbances. The acute relapse must meet at least one of the following clinical phenotypes: a) Optic neuritis (ON): EDSS visual function score ≥3; b) Transverse myelitis (TM): EDSS pyramidal function score ≥2. NMOSD-related syndromes (e.g., area postrema syndrome, acute brainstem syndrome, acute diencephalic syndrome, cerebral syndrome) may be present as concomitant features but cannot be the sole or primary manifestation.
• Serum AQP4-IgG positive by cell-based assay (CBA) with a titer ≥1:32, and negative for MOG-IgG (CBA or LCBA) and GFAP-IgG.
• Expanded Disability Status Scale (EDSS) score at enrollment ≥3 and ≤8 points.
• Planned to receive or currently receiving intravenous methylprednisolone (IVMP) treatment, and not on or only using conventional immunosuppressive maintenance therapy.
• Able to comply with standardized follow-up, with an expected minimum follow-up of 12 months during the study period.
• Signed informed consent by the patient or legal guardian (if applicable).

You may not qualify if:

• Participation in a randomized clinical trial with blinded treatment allocation.
• Received treatment with monoclonal antibody (including rituximab, satralizumab, inebilizumab, eculizumab, efgartigimod, etc.) within 3 months prior to screening.
• Received treatment with IVIg, plasma exchange (PE), IVMP, or oral corticosteroids \>30 mg/day within 1 month prior to screening.
• Incomplete or unavailable follow-up data, expected inability to complete follow-up, or poor compliance.
• Patients who have independently discontinued immunotherapy or demonstrate poor adherence.
• Presence of severe underlying diseases or other conditions that may affect the safety of immunotherapy or the interpretation of study results, including but not limited to: a) Chronic or active infections requiring long-term systemic treatment (e.g., progressive multifocal leukoencephalopathy, chronic renal infection, chronic respiratory infection with bronchiectasis, active tuberculosis, active hepatitis C, etc.); b) Positive hepatitis B serology (except in individuals with prior vaccination); c) History or suspicion of tuberculosis; d) Positive HIV serology; e) History or current clinically significant adverse reactions (including severe allergic reactions) related to corticosteroids, FcRn antagonists, or complement inhibitors.
• Pregnant or breastfeeding women, or women planning pregnancy in the near future (contraception required during treatment).
• Any other condition deemed by the investigators to make participation in the study inappropriate.

Sponsors & Collaborators

• First Affiliated Hospital of Chongqing Medical University: lead

Biospecimen

Retention: SAMPLES WITHOUT DNA

Peripheral blood samples will be collected at four time points (baseline, month 1, month 3, and month 6).

Study Officials

• Jinzhou Feng, Ph.D

  First Affiliated Hospital of Chongqing Medical University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jinzhou Feng, Ph.D

CONTACT

023-89012487; 203756@cqmu.edu.cn

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate professor

Study Record Dates

• First Submitted: September 4, 2025
• First Posted: September 19, 2025
• Study Start: October 1, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): June 30, 2027
• Last Updated: September 19, 2025

Record last verified: 2025-09