NMOSDNCT07182409
Efficacy and Safety of Monoclonal Antibody in Acute Phase of Neuromyelitis Optica Spectrum Disorder
An observational study of NMOSD, run by First Affiliated Hospital of Chongqing Medical University.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- NMOSD
- Type of treatment
- Injection or infusion
- How many people
- 40 participants
- Where
- No sites listed yet
- Study dates
- 1 Oct 2025to 30 Jun 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: NMOSD patients with acute attacks
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Efficacy and Safety of Monoclonal Antibody in Acute Phase of Neuromyelitis Optica Spectrum Disorder（MAAP-NMO），A Prospective, Multicenter Cohort Study
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.
Read the detailed description
Neuromyelitis optica spectrum disorder (NMOSD) is an inflammatory demyelinating disease of the central nervous system characterized primarily by humoral immune dysfunction. The acute phase is highly disabling; therefore, improving the effectiveness of acute-phase interventions represents a critical challenge in the clinical management of NMOSD. Conventional acute treatments such as intravenous methylprednisolone (IVMP), plasma exchange (PE), and intravenous immunoglobulin (IVIg) provide only limited rates of remission. The advent of novel biologics has expanded therapeutic options for NMOSD, but consensus regarding the optimal treatment approach during the acute phase has not yet been established.
This project is a multicenter, prospective, real-world observational study. A total of 35-45 patients with acute-phase NMOSD from 12 centers across China will be enrolled and followed systematically for at least 6 months according to a standardized protocol. The study will evaluate the real-world efficacy and safety of different monoclonal antibodies, primarily focusing on efgartigimod and eculizumab, in the treatment of acute-phase NMOSD. It will further assess their impact on symptom and neurological disability improvement, as well as their effects on immunological parameters, biomarkers, and imaging outcomes, in order to explore the optimal acute-phase immunotherapy strategy in NMOSD.
- Conditions
- NMOSD
- Keywords
- NMOSD, Intravenous methylprednisolone, Efgartigimod, Eculizumab, Plasma exchange
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 1 months
Secondary outcomes (13)
Change in Expanded Disability Status Scale (EDSS) score
Change in Expanded Disability Status Scale (EDSS) score from baseline at 3 and 6 months (EDSS: Minimum Score 1, Maximum score 10, higher scores mean a worse outcome).
Measured: 3 and 6 months
Percentage of Participants with Improvement
Improvement is defined as:
- ≥1.0-point decrease from baseline if baseline EDSS ≤5.5;
- ≥0.5-point decrease from baseline if baseline EDSS >5.5.
Measured: 1, 3 and 6 months
Change in Low-Contrast Visual Acuity (LCVA)
Low-Contrast Visual Acuity (LCVA), using a standardized low-contrast ETDRS visual acuity chart, the number of letters that the patient can identify at a sitting position and a distance of 4 meters / 1 meter is measured to reflect the recovery of visual function.
Measured: 1, 3 and 6 months
Percentage of Participants without relapse
Relapse is defined as new neurological symptoms or significant worsening of existing symptoms lasting ≥24 hours during the follow-up period, excluding causes such as infection or fever, and confirmed by imaging or clinical evaluation.
Measured: 1, 3 and 6 months
MRI changes after immunotherapy
MRI changes including improvement of acute-phase pre-existing T2 hyperintense lesions and enhancing lesions (volume reduction, signal attenuation, or decreased enhancement), and the number of new and/or enlarged T2 hyperintense lesions or new enhancing lesions.
Measured: 1 and 6 months
Change in serum AQP4-IgG titer
Change in serum AQP4-IgG titer before treatment and at 1, 3, and 6 months after immunotherapy, measured by cell-based assay.
Measured: 1, 3 and 6 months
Change in serum GFAP level
Change in serum GFAP level before treatment and at 1, 3, and 6 months after immunotherapy, measured by Simoa immunoassay.
Measured: 1, 3, and 6 months
Change in serum NfL level
Change in serum NfL level before treatment and at 1, 3, and 6 months after immunotherapy, measured by Simoa immunoassay.
Measured: 1, 3, and 6 months
Changes in serum IL-6, IL-17, TNF-α, IFN-γ, and IL-10 levels
Changes in serum IL-6, IL-17, TNF-α, IFN-γ, and IL-10 levels before treatment and at 1, 3, and 6 months after immunotherapy, measured by multiplex bead-based immunoassay. Units: pg/mL.
Measured: 1, 3 and 6 months
Changes in T/B cell subsets
Changes in T/B cell subsets (immune function monitoring) before treatment and at 1, 3, and 6 months after immunotherapy.
Measured: 1, 3 and 6 months
Changes in serum lactate, pyruvate, and glucose levels
Energy metabolism will be assessed by quantifying serum lactate, pyruvate, and glucose concentrations at baseline, 1, 3, and 6 months using liquid chromatography-tandem mass spectrometry (LC-MS/MS). Units: mmol/L.
Measured: 1, 3 and 6 months
Changes in serum lipid metabolites
Lipid metabolism will be evaluated by measuring serum cholesterol at baseline, 1, 3 and 6 months using LC-MS/MS. Units: µmol/L.
Measured: 1, 3 and 6 months
Incidence of adverse events (AEs) and serious adverse events (SAEs)
AEs are any unfavorable medical occurrences during the study period, regardless of causality. SAEs include events resulting in death, life-threatening conditions, permanent or significant disability or loss of function, hospitalization or prolongation of hospitalization, congenital anomaly, or birth defect. Record the time of onset, type, severity, and outcome. Drug-related adverse effects of biologics (e.g., efgartigimod, inebilizumab) will be specifically monitored, including hypogammaglobulinemia (assessed at baseline, 1, 3, and 6 months) and occurrence of meningococcal infection.
Measured: 1, 3 and 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2025
- Study start1 Oct 2025 (estimated)
- Primary completion31 Dec 2026 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NMOSDStudies of the same condition, open and under way.
- More trials run by First Affiliated Hospital of Chongqing Medical UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Feng Jinzhou, First Affiliated Hospital of Chongqing Medical University
- Investigators
- Jinzhou Feng, Ph.D, Principal investigator, First Affiliated Hospital of Chongqing Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07182409
- Sponsor's protocol ID
- MAAP-NMO-001
- Status last verified
- Sep 2025
- First submitted
- 4 Sep 2025
- Last updated
- 19 Sep 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Monoclonal Antibody in Acute Phase of Neuromyelitis Optica Spectrum Disorder（MAAP-NMO），A Prospective, Multicenter Cohort Study
- ClinicalTrials.gov ID
- NCT07182409
- Acronym / sponsor ID
- MAAP-NMO-001
- Conditions
- NMOSD
- Keywords
- NMOSD, Intravenous methylprednisolone, Efgartigimod, Eculizumab, Plasma exchange
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- First Affiliated Hospital of Chongqing Medical UniversityUniversity, hospital or other
- Study start
- 1 Oct 2025 (estimated)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 19 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Neuromyelitis optica spectrum disorder (NMOSD) is an inflammatory demyelinating disease of the central nervous system characterized primarily by humoral immune dysfunction. The acute phase is highly disabling; therefore, improving the effectiveness of acute-phase interventions represents a critical challenge in the clinical management of NMOSD. Conventional acute treatments such as intravenous methylprednisolone (IVMP), plasma exchange (PE), and intravenous immunoglobulin (IVIg) provide only limited rates of remission. The advent of novel biologics has expanded therapeutic options for NMOSD, but consensus regarding the optimal treatment approach during the acute phase has not yet been established.
This project is a multicenter, prospective, real-world observational study. A total of 35-45 patients with acute-phase NMOSD from 12 centers across China will be enrolled and followed systematically for at least 6 months according to a standardized protocol. The study will evaluate the real-world efficacy and safety of different monoclonal antibodies, primarily focusing on efgartigimod and eculizumab, in the treatment of acute-phase NMOSD. It will further assess their impact on symptom and neurological disability improvement, as well as their effects on immunological parameters, biomarkers, and imaging outcomes, in order to explore the optimal acute-phase immunotherapy strategy in NMOSD.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- NMOSD patients with acute attacks
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change in Expanded Disability Status Scale (EDSS) score
Time frame: 1 months
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).
13 secondary outcomes
Change in Expanded Disability Status Scale (EDSS) score
Time frame: 3 and 6 months
Percentage of Participants with Improvement
Time frame: 1, 3 and 6 months
Change in Low-Contrast Visual Acuity (LCVA)
Time frame: 1, 3 and 6 months
Percentage of Participants without relapse
Time frame: 1, 3 and 6 months
MRI changes after immunotherapy
Time frame: 1 and 6 months
Change in serum AQP4-IgG titer
Time frame: 1, 3 and 6 months
Change in serum GFAP level
Time frame: 1, 3, and 6 months
Change in serum NfL level
Time frame: 1, 3, and 6 months
Changes in serum IL-6, IL-17, TNF-α, IFN-γ, and IL-10 levels
Time frame: 1, 3 and 6 months
Changes in T/B cell subsets
Time frame: 1, 3 and 6 months
Changes in serum lactate, pyruvate, and glucose levels
Time frame: 1, 3 and 6 months
Changes in serum lipid metabolites
Time frame: 1, 3 and 6 months
Incidence of adverse events (AEs) and serious adverse events (SAEs)
Time frame: 1, 3 and 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2025.
Related trials for NMOSD
Recruiting
Impact of a Structured Wellness Behavioral Intervention on Quality of Life in NMOSD
Neuromyelitis Optica Spectrum Disorder (NMOSD)
RecruitingObservational study
Observational Safety Study in Women With Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® During Pregnancy
Neuromyelitis Optica Spectrum Disorder, Pregnancy Related
RecruitingPhase 2
Efgartigimod for the Treatment of Acute Optic Neuritis
Optic Neuritis
Not yet recruitingPhase 1/2
A Safety and Efficacy Study Evaluating CTX112 in Subjects With Refractory Neurologic Autoimmune Disease
Progressive Multiple Sclerosis (PMS), Neuromyelitis Optica
RecruitingObservational study
Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies
Neuromyelitis Optica Spectrum Disorder, NMOSD
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jinzhou Feng, Ph.D principal investigator
First Affiliated Hospital of Chongqing Medical University
Questions about this trial
What is this clinical trial studying?
An observational study of NMOSD, run by First Affiliated Hospital of Chongqing Medical University.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 19 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jinzhou Feng, Ph.D (023-89012487, 203756@cqmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Expanded Disability Status Scale (EDSS) score (time frame: 1 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2025.