Clinical trial NCT06118398: Efgartigimod in Acute Neuromyelitis Optica Spectrum Disorders
Official title: Effectiveness and Safety of Efgartigimod in the Acute Phase of Neuromyelitis Optica Spectrum Disorders-a Multicentric, Controlled, Retrospective, Real-Word Study
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Neuromyelitis Optica
Efgartigimod
Lead sponsor: First Affiliated Hospital of Chongqing Medical University
Sponsor class: OTHER
First posted: 2023-11-07
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: NMOSD patients with acute attacks

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.

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.

Primary outcomes:
measure: Change in Expanded Disability Status Scale (EDSS) score from baseline.
timeFrame: 1 month
Secondary outcomes:
measure: Change in Expanded Disability Status Scale (EDSS) score from baseline.
timeFrame: 3 months, 6 months
measure: Percentage of Participants with Disability Improvement
timeFrame: 1 month, 3 months, 6 months
measure: Change in modified Rankin score (mRS) from baseline.
timeFrame: 1 month, 3 months, 6 months
measure: Time to first relapse
timeFrame: 6 months
measure: Number of New, and/or Enlarging T2 Hyperintense Lesions Detected by Magnetic Resonance Imaging (MRI)
timeFrame: 6 months
measure: Change in timed 25 Foot Walk Test from baseline.
timeFrame: 1 month, 3 months, 6 months
measure: Number of NMOSD attacked during follow-up
timeFrame: 6 months
measure: Change in serum GFAP levels from baseline
timeFrame: 1 month, 6 months
measure: Change in AQP4-ab titres from baseline
timeFrame: 1month, 6 months
measure: Change in serum NfL levels from baseline
timeFrame: 1 month, 6 months
measure: Change in Visual Acuity (VA) from baseline
timeFrame: 1 month, 6 months
measure: Changes in EQ-5D-5L scores from baseline
timeFrame: 6 months
measure: Change in retinal nerve fibre layer (RNFL) loss from baseline
timeFrame: 1 month,6 months
measure: Adverse reactions during treatment and follow-up
timeFrame: 6 months
Other outcomes:
Locations:
Study officials:
affiliation: First Affiliated Hospital of Chongqing Medical University
name: Jinzhou Feng, Ph.D
role: PRINCIPAL_INVESTIGATOR
References: