Efgartigimod in Acute Neuromyelitis Optica Spectrum Disorders

Not yet recruiting

• Conditions: Neuromyelitis Optica; Efgartigimod
• Interventions: Drug: Intravenous methylprednisolone (IVMP) and Efgartigimod; Drug: IVMP

• Registration Number: NCT06118398
• Lead Sponsor: Feng Jinzhou

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.

Study Timeline

• Status Verified: 2023-11
• Study First Submitted: 2023-11-01
• Study First Submitted QC: 2023-11-01
• Last Update Submitted: 2023-11-01
• Study Start: 2023-11-05
• Study First Posted: 2023-11-07
• Last Update Posted: 2023-11-07
• Primary Completion: 2024-11-05
• Study Completion: 2025-05-05

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 24

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.

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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.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Exposed group	Intravenous methylprednisolone (IVMP) and Efgartigimod	Intravenous methylprednisolone (IVMP) plus Efgartigimod
Control group	IVMP	IVMP

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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.
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).
Number of NMOSD attacked during follow-up	6 months	Number of NMOSD treatment related to acute attack during follow-up.
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 serum NfL levels from baseline	1 month, 6 months	Change in serum NfL levels from baseline to 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.
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
Adverse reactions during treatment and follow-up	6 months
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).
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).
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).