Efgartigimod in Acute Neuromyelitis Optica Spectrum Disorders

Effectiveness and Safety of Efgartigimod in the Acute Phase of Neuromyelitis Optica Spectrum Disorders-a Multicentric, Controlled, Retrospective, Real-Word Study

This study aims to retrospective investigate the safety and effectiveness of Efgartigimod in the acute phase of neuromyelitis optica spectrum disorders (NMOSD) patients.

Study Overview

• Status: Not yet recruiting
• Conditions: Neuromyelitis Optica; Efgartigimod
• Intervention / Treatment: Drug: Intravenous methylprednisolone (IVMP) and Efgartigimod; Drug: IVMP

Detailed Description

This is a multicentre, controlled, retrospective, real-world study which aims to compare the safety and effectiveness of Intravenous methylprednisolone (IVMP) with Efgartigimod injection add-on treatment with IVMP treatment in acute NMOSD patients. Twenty-four patients from 6 centres in China will be enrolled.

• Study Type: Observational
• Enrollment (Estimated): 24

Contacts and Locations

• Study Contact: Name: Jinzhou Feng, Ph.D; Phone Number: 02389012487; Email: 203756@cqmu.edu.cn

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. Patients in the acute phase of NMOSD (definition of acute phase: new neurological symptoms or aggravation of existing symptoms within 30 days before received treatment, lasting at least 24 hours without fever), who had poor response to IVMP and without having received second-line therapies such as plasma exchange or intravenous immunoglobulin consequencely (Poor response is defined as a reduction in EDSS score of: I. <1.0 from the baseline EDSS score when the baseline score was <=5.5 II. < 0.5 when the baseline EDSS score > 5.5).
• 3. Patients who were approved for Efgartigimod treatment would be enrolled in the exposed group.
• 4. Expanded disability status scale (EDSS) score ≤ 8 and ≥ 2.5 before treatment.
• 5. Patients have given their written informed consent.

Exclusion Criteria:

• 1. Lactating and pregnant females before treatment.
• 2. Participated in other interventional studies within 30 days before treatment.
• 3. Received plasma exchange, immunoadsorption, or intravenous immunoglobulin (IVIG） therapy within 1 month before treatment.
• 4. History of malignancies.
• 5. Combined with severe mental disorders and other conditions that unable to cooperate with follow-up.
• 6. After being evaluated by experts, patients with active hepatitis, active tuberculosis, or other special conditions which were ineligible 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 methylprednisolone (IVMP) plus Efgartigimod	Drug: Intravenous methylprednisolone (IVMP) and Efgartigimod; IVMP 800-1000mg/day for 3-5 days plus Efgartigimod (Efgartigimod: 10mg/kg IV on Day 1, Day 8, Day 15 and Day 22 after IVMP.)
Control group; IVMP	Drug: IVMP; IVMP 800-1000mg/day for 3-5 days.

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 to 1 month after treatment (EDSS: Minimum Score 1, Maximum score 10, higher scores mean a worse outcome).	1 month

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to first relapse		6 months
Change in Expanded Disability Status Scale (EDSS) score from baseline.	Change in Expanded Disability Status Scale (EDSS) score from baseline to 3 months, 6 months after treatment (EDSS: Minimum Score 1, Maximum score 10, higher scores mean a worse outcome).	3 months, 6 months
Percentage of Participants with Disability Improvement	Disability improvement is defined as a reduction in EDSS score of: A) ≥1.0 point from the baseline EDSS score when the baseline score was ≤5.5; B) ≥0.5 point when the baseline EDSS score > 5.5(EDSS: Minimum Score 1, Maximum score 10, higher scores mean a worse outcome).	1 month, 3 months, 6 months
Change in modified Rankin score (mRS) from baseline.	Change in modified Rankin score (mRS) from baseline at 1 month 1, 3 month 3 and 6 month (mRS: Minimum Score 0, Maximum score 6, higher scores mean a worse outcome).	1 month, 3 months, 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 month, 3 months, 6 months
Number of NMOSD attacked during follow-up	Number of NMOSD treatment related to acute attack during follow-up.	6 months
Change in serum GFAP levels from baseline	Change in serum GFAP levels from baseline to the last visit.	1 month, 6 months
Change in AQP4-ab titres from baseline	Change in AQP4-ab titres from baseline to the last visit	1month, 6 months
Change in serum NfL levels from baseline	Change in serum NfL levels from baseline to the last visit	1 month, 6 months
Change in Visual Acuity (VA) from baseline	Change in Visual Acuity (VA) at1 month 1, and 6 month.	1 month, 6 months
Changes in EQ-5D-5L scores from baseline	Changes in EQ-5D scores from baseline to month 6(EQ-5D-5L: Minimum Score 5, Maximum score 25, lower scores mean a better quality of life).	6 months
Change in retinal nerve fibre layer (RNFL) loss from baseline	Change in retinal nerve fibre layer (RNFL) loss measured by optical coherence tomography (OCT) from baseline at month 1, month 6.	1 month,6 months
Adverse reactions during treatment and follow-up		6 months

Collaborators and Investigators

• Sponsor: Feng Jinzhou
• Investigators: Principal Investigator: Jinzhou Feng, Ph.D, First Affiliated Hospital of Chongqing Medical University

Study record dates

Study Major Dates

• Study Start (Estimated): November 5, 2023
• Primary Completion (Estimated): November 5, 2024
• Study Completion (Estimated): May 5, 2025

Study Registration Dates

• First Submitted: November 1, 2023
• First Submitted That Met QC Criteria: November 1, 2023
• First Posted (Actual): November 7, 2023

Study Record Updates

• Last Update Posted (Actual): November 7, 2023
• Last Update Submitted That Met QC Criteria: November 1, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• 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; Autonomic Agents; Peripheral Nervous System Agents; Anti-Inflammatory Agents; Antineoplastic Agents; Antiemetics; Gastrointestinal Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Neuroprotective Agents; Protective Agents; Prednisolone; Methylprednisolone Acetate; Methylprednisolone; Methylprednisolone Hemisuccinate; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate
• Other Study ID Numbers: EANMO-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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