Neuromyelitis OpticaNCT06118398
Efgartigimod in Acute Neuromyelitis Optica Spectrum Disorders
An observational study of Neuromyelitis Optica and Efgartigimod, run by Feng Jinzhou.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neuromyelitis Optica, Efgartigimod
- What is being tested?
- Intravenous methylprednisolone (IVMP) and Efgartigimod and IVMP
- Type of treatment
- Injection or infusion
- How many people
- 24 participants
- Where
- No sites listed yet
- Study dates
- 5 Nov 2023to 5 May 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: NMOSD patients with acute attacks
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Effectiveness and Safety of Efgartigimod in the Acute Phase of Neuromyelitis Optica Spectrum Disorders-a Multicentric, Controlled, Retrospective, Real-Word Study
This study aims to retrospective investigate the safety and effectiveness of Efgartigimod in the acute phase of neuromyelitis optica spectrum disorders (NMOSD) patients.
Read the 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.
- Conditions
- Neuromyelitis Optica, Efgartigimod
- Being tested
- Intravenous methylprednisolone (IVMP) and Efgartigimod, IVMP
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intravenous methylprednisolone (IVMP) and EfgartigimodDrug
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.)
IVMPDrug
IVMP 800-1000mg/day for 3-5 days.
| Study group | Role | What they receive |
|---|---|---|
| Exposed group | Intravenous methylprednisolone (IVMP) plus Efgartigimod Receives: Drug: Intravenous methylprednisolone (IVMP) and Efgartigimod | |
| Control group | IVMP Receives: Drug: IVMP |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 1 month
Secondary outcomes (14)
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).
Measured: 3 months, 6 months
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).
Measured: 1 month, 3 months, 6 months
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).
Measured: 1 month, 3 months, 6 months
Time to first relapse
Measured: 6 months
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.
Measured: 6 months
Change in timed 25 Foot Walk Test from baseline.
Change in time taken to complete the timed 25 Foot Walk Test from baseline.
Measured: 1 month, 3 months, 6 months
Number of NMOSD attacked during follow-up
Number of NMOSD treatment related to acute attack during follow-up.
Measured: 6 months
Change in serum GFAP levels from baseline
Change in serum GFAP levels from baseline to the last visit.
Measured: 1 month, 6 months
Change in AQP4-ab titres from baseline
Change in AQP4-ab titres from baseline to the last visit
Measured: 1month, 6 months
Change in serum NfL levels from baseline
Change in serum NfL levels from baseline to the last visit
Measured: 1 month, 6 months
Change in Visual Acuity (VA) from baseline
Change in Visual Acuity (VA) at1 month 1, and 6 month.
Measured: 1 month, 6 months
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).
Measured: 6 months
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.
Measured: 1 month,6 months
Adverse reactions during treatment and follow-up
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2023
- Study start5 Nov 2023 (estimated)
- Primary completion5 Nov 2024 (estimated)
- Study completion5 May 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis OpticaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Feng Jinzhou
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Feng Jinzhou, First Affiliated Hospital of Chongqing Medical University
- Investigators
- Jinzhou Feng, Ph.D, Principal investigator, First Affiliated Hospital of Chongqing Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06118398
- Sponsor's protocol ID
- EANMO-001
- Status last verified
- Nov 2023
- First submitted
- 1 Nov 2023
- Last updated
- 7 Nov 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effectiveness and Safety of Efgartigimod in the Acute Phase of Neuromyelitis Optica Spectrum Disorders-a Multicentric, Controlled, Retrospective, Real-Word Study
- ClinicalTrials.gov ID
- NCT06118398
- Acronym / sponsor ID
- EANMO-001
- Conditions
- Neuromyelitis Optica, Efgartigimod
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Feng JinzhouUniversity, hospital or other
- Study start
- 5 Nov 2023 (estimated)
- Primary completion
- 5 Nov 2024 (estimated)
- Study completion
- 5 May 2025 (estimated)
- First posted
- 7 Nov 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Exposed group | – | Drug: Intravenous methylprednisolone (IVMP) and EfgartigimodIntravenous methylprednisolone (IVMP) plus Efgartigimod |
| Control group | – | Drug: IVMPIVMP |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- NMOSD patients with acute attacks
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intravenous methylprednisolone (IVMP) and EfgartigimodIVMP 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.) | Drug | Exposed group |
| IVMPIVMP 800-1000mg/day for 3-5 days. | Drug | Control group |
Primary outcome
Change in Expanded Disability Status Scale (EDSS) score from baseline.
Time frame: 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).
14 secondary outcomes
Change in Expanded Disability Status Scale (EDSS) score from baseline.
Time frame: 3 months, 6 months
Percentage of Participants with Disability Improvement
Time frame: 1 month, 3 months, 6 months
Change in modified Rankin score (mRS) from baseline.
Time frame: 1 month, 3 months, 6 months
Time to first relapse
Time frame: 6 months
Number of New, and/or Enlarging T2 Hyperintense Lesions Detected by Magnetic Resonance Imaging (MRI)
Time frame: 6 months
Change in timed 25 Foot Walk Test from baseline.
Time frame: 1 month, 3 months, 6 months
Number of NMOSD attacked during follow-up
Time frame: 6 months
Change in serum GFAP levels from baseline
Time frame: 1 month, 6 months
Change in AQP4-ab titres from baseline
Time frame: 1month, 6 months
Change in serum NfL levels from baseline
Time frame: 1 month, 6 months
Change in Visual Acuity (VA) from baseline
Time frame: 1 month, 6 months
Changes in EQ-5D-5L scores from baseline
Time frame: 6 months
Change in retinal nerve fibre layer (RNFL) loss from baseline
Time frame: 1 month,6 months
Adverse reactions during treatment and follow-up
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jinzhou Feng, Ph.D principal investigator
First Affiliated Hospital of Chongqing Medical University
Questions about this trial
What is this clinical trial studying?
An observational study of Neuromyelitis Optica and Efgartigimod, run by Feng Jinzhou.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 7 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Jinzhou Feng, Ph.D (02389012487, 203756@cqmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Expanded Disability Status Scale (EDSS) score from baseline. (time frame: 1 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Nov 2023; AzTrials last refreshed its copy on 6 Nov 2026.