Efgartigimod in Acute Neuromyelitis Optica Spectrum Disorders

Sponsor

Feng Jinzhou (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT06118398

Collaborator

(none)

Enrollment

Anticipated Duration (Months)

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Neuromyelitis Optica Efgartigimod	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 Design

Study Type:

Observational

Anticipated Enrollment :

24 participants

Observational Model:

Cohort

Time Perspective:

Retrospective

Official Title:

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

Anticipated Study Start Date :

Nov 5, 2023

Anticipated Primary Completion Date :

Nov 5, 2024

Anticipated Study Completion Date :

May 5, 2025

Arms and Interventions

Arm	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.

Arm

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.

Outcome Measures

Primary Outcome Measures

Change in Expanded Disability Status Scale (EDSS) score from baseline. [1 month]
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).

Secondary Outcome Measures

Change in Expanded Disability Status Scale (EDSS) score from baseline. [3 months, 6 months]
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).
Percentage of Participants with Disability Improvement [1 month, 3 months, 6 months]
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).
Change in modified Rankin score (mRS) from baseline. [1 month, 3 months, 6 months]
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).
Time to first relapse [6 months]
Number of New, and/or Enlarging T2 Hyperintense Lesions Detected by Magnetic Resonance Imaging (MRI) [6 months]
Number of New, and/or Enlarging T2 Hyperintense Lesions Detected by Magnetic Resonance Imaging (MRI) at the last visit.
Change in timed 25 Foot Walk Test from baseline. [1 month, 3 months, 6 months]
Change in time taken to complete the timed 25 Foot Walk Test from baseline.
Number of NMOSD attacked during follow-up [6 months]
Number of NMOSD treatment related to acute attack during follow-up.
Change in serum GFAP levels from baseline [1 month, 6 months]
Change in serum GFAP levels from baseline to the last visit.
Change in AQP4-ab titres from baseline [1month, 6 months]
Change in AQP4-ab titres from baseline to the last visit
Change in serum NfL levels from baseline [1 month, 6 months]
Change in serum NfL levels from baseline to the last visit
Change in Visual Acuity (VA) from baseline [1 month, 6 months]
Change in Visual Acuity (VA) at1 month 1, and 6 month.
Changes in EQ-5D-5L scores from baseline [6 months]
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).
Change in retinal nerve fibre layer (RNFL) loss from baseline [1 month,6 months]
Change in retinal nerve fibre layer (RNFL) loss measured by optical coherence tomography (OCT) from baseline at month 1, month 6.
Adverse reactions during treatment and follow-up [6 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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).
1. 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).
1. Patients who were approved for Efgartigimod treatment would be enrolled in the exposed group.
1. Expanded disability status scale (EDSS) score ≤ 8 and ≥ 2.5 before treatment.
1. Patients have given their written informed consent.

Exclusion Criteria:

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