Efgartigimod in Acute Neuromyelitis Optica Spectrum Disorders

NCT ID: NCT06118398

Last Updated: 2023-11-07

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Total Enrollment: 24 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2023-11-05
• Study Completion Date: 2025-05-05

Brief Summary

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

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.

Conditions

Neuromyelitis Optica; Efgartigimod

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Study Groups

Exposed group

Intravenous methylprednisolone (IVMP) plus Efgartigimod

Intravenous methylprednisolone (IVMP) and Efgartigimod

Intervention Type: DRUG

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

IVMP

Intervention Type: DRUG

IVMP 800-1000mg/day for 3-5 days.

Interventions

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.)

Intervention Type: DRUG

IVMP

IVMP 800-1000mg/day for 3-5 days.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Feng Jinzhou

OTHER

Sponsor Role: lead

Responsible Party

Feng Jinzhou

Associate professor

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Jinzhou Feng, Ph.D

Role: PRINCIPAL_INVESTIGATOR

First Affiliated Hospital of Chongqing Medical University

Central Contacts

Jinzhou Feng, Ph.D

Role: CONTACT

203756@cqmu.edu.cn

02389012487

Other Identifiers

EANMO-001

Identifier Type: -

Identifier Source: org_study_id