Efgartigimod in Acute Neuromyelitis Optica Spectrum Disorders

Neuromyelitis Optica Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06118398
• Other study ID #: EANMO-001
• Status: Not yet recruiting
• Start date: November 5, 2023
• Est. completion date: May 5, 2025

Study information

• Verified date: November 2023
• Source: First Affiliated Hospital of Chongqing Medical University
• Contact: Jinzhou Feng, Ph.D
• Phone: 02389012487
• Email: 203756[@]cqmu.edu.cn
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 24
• Est. completion date: May 5, 2025
• Est. primary completion date: November 5, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Neuromyelitis Optica

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Feng Jinzhou

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	Time to first relapse		6 months
Secondary	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
Secondary	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
Secondary	Number of NMOSD attacked during follow-up	Number of NMOSD treatment related to acute attack during follow-up.	6 months
Secondary	Change in serum GFAP levels from baseline	Change in serum GFAP levels from baseline to the last visit.	1 month, 6 months
Secondary	Change in AQP4-ab titres from baseline	Change in AQP4-ab titres from baseline to the last visit	1month, 6 months
Secondary	Change in serum NfL levels from baseline	Change in serum NfL levels from baseline to the last visit	1 month, 6 months
Secondary	Change in Visual Acuity (VA) from baseline	Change in Visual Acuity (VA) at1 month 1, and 6 month.	1 month, 6 months
Secondary	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
Secondary	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
Secondary	Adverse reactions during treatment and follow-up		6 months