A Phase 3 interventional study of Eculizumab administration and High-dose corticosteroid pulse therapy in Neuromyelitis Optica Spectrum Disorders (NMOSD), sponsored by Tianjin Medical University General Hospital. Recruiting at 1 site in China. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2026-05-14.
Sponsored by Tianjin Medical University General Hospital · Phase 3, Interventional, and Treatment
Study Title: A National, Multicenter, Randomized Controlled Trial of the Modified Zipper Therapy in AQP4 Antibody-Positive Neuromyelitis Optica Spectrum Disorder (ELITE Study)
Brief Summary:
The goal of this clinical trial is to evaluate the efficacy and safety of a novel sequential immunomodulation strategy, termed "Modified Zipper Therapy," in patients with acute attacks of Aquaporin-4 antibody-positive Neuromyelitis Optica Spectrum Disorder (AQP4-IgG+ NMOSD). The therapy aims to enhance neurological recovery by combining plasma exchange (PE) with immediate complement inhibition using eculizumab, following high-dose corticosteroid pulse therapy.
The main questions this trial aims to answer are:
Efficacy: Does the Modified Zipper Therapy (high-dose corticosteroids + plasma exchange + eculizumab) lead to a higher rate of neurological improvement at Week 12 compared to standard therapy (high-dose corticosteroids + plasma exchange alone)?
For patients with NMOSD-related optic neuritis (NMOSD-ON), improvement is defined as a gain of ≥10 letters on the ETDRS chart or a decrease of ≥0.2 LogMAR in best-corrected visual acuity (BCVA).
For patients with NMOSD-related longitudinally extensive transverse myelitis (NMOSD-LETM), improvement is defined as a reduction of ≥2 points on the Expanded Disability Status Scale (EDSS).
Safety: What is the nature and frequency of adverse events experienced by participants receiving the Modified Zipper Therapy compared to those receiving standard therapy?
Researchers will compare the Modified Zipper Therapy group to the Standard Therapy group to see if the novel combination is more effective in improving visual and functional outcomes in acute AQP4-IgG+ NMOSD.
Participants will:
Be randomly assigned (like a coin toss) to receive either the Modified Zipper Therapy or the Standard Therapy.
Undergo a treatment period involving intravenous corticosteroids and a series of plasma exchange sessions. The Modified Zipper Therapy group will also receive intravenous eculizumab infusions timed around the plasma exchange procedures.
Be followed for 24 weeks after treatment completion.
Attend scheduled clinic visits for comprehensive assessments including:
Visual acuity testing (using ETDRS, Snellen, and low-contrast charts).
Neurological function evaluations (EDSS and OSIS scores).
Optical coherence tomography (OCT) and visual evoked potential (VEP) tests.
Magnetic resonance imaging (MRI) scans of the optic nerves.
Safety monitoring (physical exams, lab tests, ECGs).
178 studies on the registry are indexed under Neuromyelitis Optica; 78 are open to participants now.
This study's planned enrollment of 198 is above the median of 29 across 108 interventional studies indexed under Neuromyelitis Optica.
Browse Neuromyelitis Optica studies →Tianjin Medical University General Hospital is the lead sponsor of 75 studies on the registry; 31 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biological: Eculizumab administration · Drug: High-dose corticosteroid pulse therapy · Procedure: Plasma exchange (PE)
High-dose corticosteroid pulse therapy+ plasma exchange
Drug: High-dose corticosteroid pulse therapy · Procedure: Plasma exchange (PE)
Complement Inhibitor Therapy (Eculizumab): Initial Dose: Administer 900 mg of eculizumab intravenously immediately after the first PE session. Supplemental Doses: Administer supplemental doses (300-600 mg) after the 2nd, 3rd, and 4th PE sessions. The exact dose will be adjusted in real-time based on drug clearance post-PE. Maintenance Therapy: Administer three additional 900 mg intravenous infusions on the day of the last PE session, and at the 1st and 2nd weeks after its completion, to consolidate efficacy and maintain stable complement inhibition.
High-Dose Corticosteroid Pulse: Intravenous methylprednisolone (IVMP) 1000 mg daily for 3 days, then 500 mg daily for 3 days, followed by 250 mg daily for 3 days, and finally 125 mg daily for 3 days. Subsequently, switch to oral prednisone at 1 mg/kg/day, tapered by 5 mg weekly until a maintenance dose of 10 mg/day is reached, which is continued for 6 months.
Plasma Exchange (PE) Therapy: Initiated concurrently with corticosteroids. Perform at least 5 PE sessions every other day. Each session exchanges 1.0-1.5 times the plasma volume. Treatment intervals may be adjusted based on fibrinogen levels and bleeding risk.
Best corrected visual acuity
Time frame: Baseline, Week 12
Expanded Disability Status Scale (EDSS)
The Expanded Disability Status Scale (EDSS) is an ordinal scale ranging from 0 to 10, with higher scores indicating worse neurological impairment and disability in patients with neuromyelitis optica spectrum disorder (NMOSD)
Time frame: Baseline, Week 12
Best corrected visual acuity
Time frame: Baseline, Week 4, Week 24,
Expanded Disability Status Scale (EDSS)
The Expanded Disability Status Scale (EDSS) is an ordinal scale ranging from 0 to 10, with higher scores indicating worse neurological impairment and disability in patients with neuromyelitis optica spectrum disorder (NMOSD)
Time frame: Baseline, Week 4, Week 24,
Optic-Spinal Impairment Score(OSIS)
The Optic-Spinal Impairment Score (OSIS) is a composite scale specifically designed for neuromyelitis optica spectrum disorder (NMOSD), assessing visual function (0-8), motor function (0-7), sensory function (0-5), and sphincter function (0-5). Higher scores reflect greater severity of optic and spinal cord dysfunction, indicating a worse outcome.
Time frame: Baseline, Week 4, Week 12, Week 24
Visual Evoked Potential P100 Wave Latency
Mean change from baseline in P100 wave latency, measured by visual evoked potential (VEP), at Week 12, and Week 24. P100 latency is expressed in milliseconds (ms). Longer latency indicates delayed neural conduction and worse visual pathway function; a greater reduction from baseline reflects a better outcome.
Time frame: Baseline, Week 12, Week 24
Visual Evoked Potential P100 Wave Amplitude
Mean change from baseline in P100 wave amplitude, measured by visual evoked potential (VEP), at Week 12, and Week 24. P100 amplitude is expressed in microvolts (μV). Higher amplitude indicates better neural signal transmission and visual pathway integrity; a smaller decrease or greater increase from baseline reflects a better outcome.
Time frame: Baseline, Week 12, Week 24
Retinal nerve fiber layer (RNFL) thickness
Mean change from baseline in peripapillary retinal nerve fiber layer (RNFL) thickness, measured by optical coherence tomography (OCT), at Week 12 and Week 24. RNFL thickness is expressed in micrometers (μm). Higher values indicate greater structural preservation of the optic nerve; a smaller decrease or greater increase from baseline reflects a better outcome.
Time frame: Baseline, Week 12, Week 24,
Ganglion Cell-Inner Plexiform Layer (GCIPL) Thickness
Mean change from baseline in macular ganglion cell-inner plexiform layer (GCIPL) thickness, measured by optical coherence tomography (OCT), at Week 12 and Week 24. GCIPL thickness is expressed in micrometers (μm). Higher values indicate greater structural integrity of the macular ganglion cells; a smaller decrease or greater increase from baseline reflects a better outcome.
Time frame: Baseline, Week 12, Week 24
Cross-sectional area of the optic nerve
Mean change from baseline in optic nerve cross-sectional area, measured by magnetic resonance imaging (MRI), at Week 12 and Week 24.
Time frame: Baseline, Week 12, Week 24
Length of Optic Nerve Lesion
Mean change from baseline in optic nerve lesion length, measured by magnetic resonance imaging (MRI), at Week 12 and Week 24.
Time frame: Baseline, Week 12, Week 24
Concentration of serum sC5b-9
Time frame: Baseline, Week 4, Week 12, Week 24
Level of serum CH50
Time frame: Baseline, Week 4, Week 12, Week 24
Plan to share: No — IPD sharing is not planned because the informed consent approved by the ethics committee does not include authorization for sharing individual participant data with external researchers.
No publications or documents are linked to this record.
Started Jan 2026 (actual). Primary completion is estimated for Dec 2026, 11 months after the start.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Tianjin Medical University General Hospital