Efgartigimod in Acute Neuromyelitis Optica Spectrum Disorders

NCT06118398 | Unknown

• 1 other identifier: EANMO-001
• Study Type: observational
• Target: 24
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Neuromyelitis Optica; Efgartigimod

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (14)

• Change in Expanded Disability Status Scale (EDSS) score from baseline.

  3 months, 6 months

• Percentage of Participants with Disability Improvement

  1 month, 3 months, 6 months

• Change in modified Rankin score (mRS) from baseline.

  1 month, 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)

  6 months

Study Arms (2)

Exposed group

Intravenous methylprednisolone (IVMP) plus Efgartigimod

Drug: Intravenous methylprednisolone (IVMP) and Efgartigimod

Control group

IVMP

Drug: IVMP

Interventions

Intravenous methylprednisolone (IVMP) and Efgartigimod 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.)

Exposed group

IVMP DRUG

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

Control group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

NMOSD patients with acute attacks

You may qualify if:

• \. Age ≥ 18 years with anti-AQP4-IgG seropositive NMOSD as defined by 2015 NMOSD diagnostic criteria by IPND (International Panel for NMO Diagnosis).
• \. 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).
• \. Patients who were approved for Efgartigimod treatment would be enrolled in the exposed group.
• \. Expanded disability status scale (EDSS) score ≤ 8 and ≥ 2.5 before treatment.
• \. Patients have given their written informed consent.

You may not qualify if:

• \. Lactating and pregnant females before treatment.
• \. Participated in other interventional studies within 30 days before treatment.
• \. Received plasma exchange, immunoadsorption, or intravenous immunoglobulin (IVIG） therapy within 1 month before treatment.
• \. History of malignancies.
• \. Combined with severe mental disorders and other conditions that unable to cooperate with follow-up.
• \. After being evaluated by experts, patients with active hepatitis, active tuberculosis, or other special conditions which were ineligible to participate in this study.

Sponsors & Collaborators

• Feng Jinzhou: lead

Biospecimen

Retention: SAMPLES WITH DNA

Blood samples are collected before the treatment and at the last visit.

MeSH Terms

Conditions

Neuromyelitis Optica

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Jinzhou Feng, Ph.D

  First Affiliated Hospital of Chongqing Medical University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jinzhou Feng, Ph.D

CONTACT

02389012487; 203756@cqmu.edu.cn

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Associate professor

Study Record Dates

• First Submitted: November 1, 2023
• First Posted: November 7, 2023
• Study Start: November 5, 2023
• Primary Completion: November 5, 2024
• Study Completion: May 5, 2025
• Last Updated: November 7, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share