Inebilizumab in Acute Neuromyelitis Optica Spectrum Disorders

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

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

Study Overview

• Status: Not yet recruiting
• Conditions: Neuromyelitis Optica Spectrum Disorder
• Intervention / Treatment: Drug: Inebilizumab; Drug: oral immunosuppressant

Detailed Description

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.

• Study Type: Observational
• Enrollment (Estimated): 50

Contacts and Locations

• Study Contact: Name: Junwei Hao, MD; Phone Number: 01083198277; Email: haojunwei@vip.163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

NMOSD patients with acute attacks

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Xuanwu Hospital, Beijing
• Collaborators: Jiangsu Hansoh Pharmaceutical Co., Ltd.
• Investigators: Principal Investigator: Junwei Hao, MD, Xuanwu Hospital, Beijing

Study record dates

Study Major Dates

• Study Start (Estimated): July 20, 2023
• Primary Completion (Estimated): July 31, 2025
• Study Completion (Estimated): July 31, 2025

Study Registration Dates

• First Submitted: May 24, 2023
• First Submitted That Met QC Criteria: June 5, 2023
• First Posted (Actual): June 6, 2023

Study Record Updates

• Last Update Posted (Actual): June 29, 2023
• Last Update Submitted That Met QC Criteria: June 26, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: observational study; Neuromyelitis Optica Spectrum Disorder; inebilizumab
• Additional Relevant MeSH Terms: Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Eye Diseases; Optic Nerve Diseases; Cranial Nerve Diseases; Myelitis, Transverse; Optic Neuritis; Neuromyelitis Optica; Physiological Effects of Drugs; Immunologic Factors; Immunosuppressive Agents
• Other Study ID Numbers: XMEC-2023-003

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No