Inebilizumab in Acute Neuromyelitis Optica Spectrum Disorders

Study Overview
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Brief Summary
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This study is aimed to observe the effectiveness and safety of inebilizumab in the acute phase of neuromyelitis optica spectrum disorders.

Detailed Description
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This is a a multicentric, prospective, real word study of inebilizumab in NMOSD acute attack compared with oral immunosuppressant. A total of 50 patients will be enrolled at approximately 10 centers around China.

Official Title
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Effectiveness and Safety of Inebilizumab in the Acute Phase of Neuromyelitis Optica Spectrum Disorders-a Multicentric, Prospective, Real Word Study

Conditions
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Neuromyelitis Optica Spectrum Disorder

Intervention / Treatment
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* Drug: Inebilizumab
* Drug: oral immunosuppressant


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 1. Age ≥ 18 years with anti-AQP4-IgG seropositive NMOSD as defined by 2015 NMOSD diagnostic criteria by IPND (International Panel for NMO Diagnosis); 2. In the acute phase of NMOSD (definition of acute phase: new neurological symptoms or aggravation of existing symptoms within 30 days before screening, lasting at least 24 hours without with fever); 3. Patients who plan to receive or are receiving intravenous methylprednisolone therapy; 4. Expanded disability status scale (EDSS) score ≤ 8 and ≥ 2.5 during the screening period; 5. Able and willing to give written informed consent; 6. Women of childbearing potential who agree to use adequate contraception during the study. Exclusion Criteria: 1. Lactating and pregnant females; 2. Participate in other interventional studies within 30 days before screening or within 5 half-lives of the investigational agent before enrollment; 3. Combined with severe mental disorders and other conditions and unable to cooperate with follow-up; 4. History of malignancies; 5. Received plasma exchange, immunoadsorption or intravenous immunoglobulin therapy within 1 month before screening; 6. Patients with negative hepatitis B surface antibody (HBsAg) and positive hepatitis B core antibody (HBcAb), or HBsAg-positive virus carriers, or patients with active tuberculosis or positive tuberculosis screening without appropriate treatment history; 7. Other situations that researchers think are not suitable to participate in this study.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Exposed group<br>intravenous Intravenous methylprednisolone (IVMP) plus intravenous inebilizumab | Drug: Inebilizumab<br>* Inebilizumab: 300mg IV on Day1 and Day 15. The first dose of inelizumab is given during IVMP.<br>|
| Non-exposed group<br>IVMP plus oral immunosuppressant (Mycophenolate Mofetil or Azathioprine) | Drug: oral immunosuppressant<br>* Oral immunosuppressants (azathioprine or mycolate mofetil) are initiated during IVMP.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Expanded Disability Status Scale (EDSS) score from baseline | Change in Expanded Disability Status Scale (EDSS) score from baseline at the last visit(EDSS : Minimum Score 1, Maximum score 10, higher scores mean a worse outcome). | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Expanded Disability Status Scale (EDSS) score from baseline | Change in Expanded Disability Status Scale (EDSS) score from baseline at month 1, month 3(EDSS : Minimum Score 1, Maximum score 10, higher scores mean a worse outcome). | 1 months, 3 months | 
| Percentage of Participants With Disability Improvement | Disability improvement is defined as a reduction in EDSS score of: A) >=1.0 from the baseline EDSS score when the baseline score was <=5.5 B) >= 0.5 when the baseline EDSS score > 5.5(EDSS : Minimum Score 1, Maximum score 10, higher scores mean a worse outcome). | 6 months | 
| Change in modified Rankin score (mRS) from baseline | Change in modified Rankin score (mRS) from baseline at month 1, month 3, month 6(mRS : Minimum Score 0, Maximum score 6, higher scores mean a worse outcome). | 1 months, 3 months, 6 months | 
| Time to first relapse |  | 6 months | 
| Number of New, and/or Enlarging T2 Hyperintense Lesions Detected by Magnetic Resonance Imaging (MRI) | Number of New, and/or Enlarging T2 Hyperintense Lesions Detected by Magnetic Resonance Imaging (MRI) at the last visit | 6 months | 
| Change in Timed 25 Foot Walk Test from baseline | Change in time taken to complete the timed 25 foot walk test from baseline | 1 months, 3 months , 6 months | 
| Number of NMOSD attacked related rescue treatment |  | 6 months | 
| Change in serum GFAP levels from baseline | Change in serum GFAP levels from baseline at the last visit | 6 months | 
| Change in AQP4-ab titers from baseline | Change in AQP4-ab titers from baseline at the last visit | 6 months | 
| Change in Low-contrast Visual Acuity (LCVA) from baseline | Change in Low-contrast Visual Acuity (LCVA) at month 3, month 6)(The LCVA test is used to determine the number of letters that can be read on a standardized low-contrast Landolt C Broken Rings Chart held at a distance of 3 meters). | 3 months, 6 months | 
| Changes in EQ-5D-5L scores from baseline | Changes in EQ-5D scores from baseline at month 1 month, month 3 , month 6(EQ-5D-5L: Minimum Score 5, Maximum score 25, lower scores mean a better quality of life). | 1 month, 3 months ,6 months | 
| Change in retinal nerve fiber layer (RNFL) loss from baseline | Change in retinal nerve fiber layer (RNFL) loss measured by optical coherence tomography (OCT) from baseline at month 1, month 3，month 6. | 3 months ,6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Neuromyelitis Optica Spectrum Disorder, inebilizumab, observational study