Neuromyelitis Optica Spectrum Disorder AttackPhase 1/2SIVAR-NMOSDNCT07738952
Safety and Efficacy Study of Sivelestat in Neuromyelitis Optica Spectrum Disorder
A Phase 1/2 study testing Sivelestat sodium hydrate in people with Neuromyelitis Optica Spectrum Disorder Attack, run by Kyushu University at 1 site in Japan.
Trial summary
The primary objective of this study is to evaluate the safety and tolerability of sivelestat sodium hydrate administered in combination with standard steroid pulse therapy in patients experiencing an acute NMOSD attack. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorder Attack
- What is being tested?
- Sivelestat sodium hydrate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteJapan
- Study dates
- Oct 2026to 31 May 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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
Who may be able to join
- Patients diagnosed with anti-AQP4 antibody-positive NMOSD according to the international diagnostic criteria for NMOSD (Wingerchuk, Neurology 2015).
- Patients experiencing a relapse including any of the following, with the relapse occurring within 14 days of obtaining consent:
- Unilateral or bilateral optic neuritis ii. Myelitis
- Patients whose FS domain has worsened by at least 1 point due to relapse.
- Patients with one or more relapse lesions identified on MRI (however, if the relapse is considered to have occurred in the same location as an existing MRI lesion neurologically, identification of a new relapse lesion is not required).
- Patients aged 18 years or older at the time of obtaining consent.
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- Female patients of childbearing potential who agree to use appropriate contraception from the time of obtaining consent until 180 days after the end of investigational product administration.
- Male patients who agree to use appropriate contraception until 90 days after the end of investigational product administration.
- Patients who can provide written informed consent.
Who cannot join
- Patients with multi-organ dysfunction involving 4 or more organs.
- Patients with severe chronic respiratory disease.
- Patients with autoimmune diseases other than NMOSD that are expected to require additional treatment during the study period.
- Patients with active systemic bacterial, viral, or fungal infections.
- Patients who have received either or both of the following prior treatments after an NMOSD relapse:
- Two or more courses of steroid pulse therapy ii. Plasmapheresis iii. High-dose immunoglobulin therapy
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- Patients with severe hepatic dysfunction.
- Patients with alcohol dependence, drug dependence, or psychiatric disorders that would interfere with study participation.
- Patients who have received other investigational drugs within 3 months prior to obtaining consent.
- Pregnant women, women suspected of being pregnant, or breastfeeding women.
- Patients with allergies to the investigational product or concomitant medications.
- Patients with severe allergies or a history of severe allergies.
- Patients with suicidal tendencies meeting any of the following criteria:
- Within 1 month prior to the screening assessment, there was suicidal behavior or ideation corresponding to "Yes" for Item 4 (Active suicidal ideation -some intent to act, but no specific plan) or Item 5 (Active suicidal ideation -specific plan and intent) of the Columbia-Suicide Severity Rating Scale (C-SSRS). (For subjects who only met Items 1-3, inclusion may be permitted at the discretion of the principal investigator or sub-investigator.) ii. Any suicidal behavior based on Item 6 of the C-SSRS occurred within the past 3 months.
- Other patients judged inappropriate by the principal investigator or sub-investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Japan1 site, 1 recruiting
- Recruiting
Kyushu University Hospital
Fukuoka
Mitsuru Watanabe, +81-92-642-5340, shinkein@med.kyushu-u.ac.jp
About this study
Official title: A Phase I/IIa Investigator-Initiated Clinical Trial to Evaluate the Safety and Efficacy of Sivelestat in Patients With Acute Relapse of Neuromyelitis Optica Spectrum Disorder
The primary objective of this study is to evaluate the safety and tolerability of sivelestat sodium hydrate administered in combination with standard steroid pulse therapy in patients experiencing an acute NMOSD attack. Safety assessments will include adverse events, laboratory parameters, vital signs, and other clinically relevant findings. In addition, the study will explore whether the addition of sivelestat sodium hydrate to standard steroid pulse therapy improves neurological outcomes in patients with acute NMOSD.
Participants will receive intravenous sivelestat sodium hydrate at a dose of 4.8 mg/kg/day administered as a continuous infusion (0.2 mg/kg/hour) for 5 consecutive days, receive steroid pulse therapy according to the study protocol, and be followed for 28 days after treatment initiation for safety and efficacy evaluations.
- Being tested
- Sivelestat sodium hydrate
- Keywords
- NMOSD, Sivelestat
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sivelestat sodium hydrateDrug
Sivelestat sodium hydrate is administered intravenously at a dose of 4.8 mg/kg/day as a continuous infusion (0.2 mg/kg/hour) for 5 consecutive days in combination with steroid pulse therapy in patients with acute NMOSD attacks.
| Study group | Role | What they receive |
|---|---|---|
| Sivelestat | Experimental | Not described Receives: Drug: Sivelestat sodium hydrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Measured: 4 weeks
Secondary outcomes (14)
Change from baseline in body temperature at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration.
Measured: 6 days
Change from baseline in blood pressure at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration.
Measured: 6 days
Change from baseline in pulse rate at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration.
Measured: 6 days
Change from baseline in percutaneous arterial oxygen saturation at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration.
Measured: 6 days
Change from baseline in Expanded Disability Status Scale (EDSS) score at 4 weeks (Day 28).
The Expanded Disability Status Scale (EDSS) ranges from 0 to 10. Higher scores indicate greater neurological disability and worse clinical status.
Measured: 4 weeks
Proportion of cases where Expanded Disability Status Scale (EDSS) score improved by 1 point or more from baseline at 4 weeks (Day 28).
The Expanded Disability Status Scale (EDSS) ranges from 0 to 10. Higher scores indicate greater neurological disability and worse clinical status.
Measured: 4 weeks
Change from baseline in Functional System (FS) domain scores at 4 weeks
Functional System (FS) scores are neurological disability scores that contribute to the Expanded Disability Status Scale (EDSS). Functional systems assessed include visual, brainstem, pyramidal, cerebellar, sensory, bowel and bladder, and cerebral functions. Individual FS scores generally range from 0 (normal function) to 5 or 6 (maximal impairment), depending on the functional system assessed. Higher scores indicate greater neurological impairment and worse clinical status.
Measured: 4 weeks
Change from baseline in Opticospinal Impairment Scale (OSIS) score at 4 weeks (Day 28).
The Opticospinal Impairment Scale (OSIS) is a disability scale for neuromyelitis optica spectrum disorder that assesses visual acuity, motor function, sensory function, and sphincter function. Total scores range from 0 to 25, with higher scores indicating greater neurological impairment and worse clinical status.
Measured: 4 weeks
Proportion of participants with recovery to pre-relapse neurological disability status at 4 weeks (Day 28).
Recovery to pre-relapse neurological disability status is defined as a return of the Expanded Disability Status Scale (EDSS; range 0-10, higher scores indicate greater disability) and the Opticospinal Impairment Scale (OSIS; range 0-25, higher scores indicate greater neurological impairment) to their respective pre-relapse scores.
Measured: 4 weeks
Change from baseline in best-corrected visual acuity at 4 weeks (Day 28)
Measured: 4 weeks
Change from baseline in critical flicker fusion frequency at 4 weeks (Day 28).
Measured: 4 weeks
Change from baseline in retinal nerve fiber layer and ganglion cell-inner plexiform layer thickness measured by optical coherence tomography (OCT) at 4 weeks (Day 28).
Measured: 4 weeks
Proportion of cases with gadolinium-enhancing lesions on MRI at 4 weeks.
Measured: 4 weeks
Proportion of cases requiring a second course of steroid pulse therapy, plasmapheresis, or high-dose immunoglobulin therapy.
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2026
- Study startOct 2026 (estimated)
- Primary completion31 Jan 2028 (estimated)
- Study completion31 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis Optica Spectrum Disorder AttackStudies 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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kyushu University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mitsuru Watanabe, Kyushu University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07738952
- Sponsor's protocol ID
- CTR542-01
- Acronym
- SIVAR-NMOSD
- Status last verified
- Oct 2026
- First submitted
- 17 Jul 2026
- Last updated
- 7 Oct 2026
- Results posted
- No
- Data monitoring committee
- 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 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/IIa Investigator-Initiated Clinical Trial to Evaluate the Safety and Efficacy of Sivelestat in Patients With Acute Relapse of Neuromyelitis Optica Spectrum Disorder
- ClinicalTrials.gov ID
- NCT07738952
- Acronym / sponsor ID
- SIVAR-NMOSD / CTR542-01
- Keywords
- NMOSD, Sivelestat
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 7 participants (estimated)
- Lead sponsor
- Kyushu UniversityUniversity, hospital or other
- Study start
- Oct 2026 (estimated)
- Primary completion
- 31 Jan 2028 (estimated)
- Study completion
- 31 May 2028 (estimated)
- First posted
- 31 Jul 2026 (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.
The primary objective of this study is to evaluate the safety and tolerability of sivelestat sodium hydrate administered in combination with standard steroid pulse therapy in patients experiencing an acute NMOSD attack. Safety assessments will include adverse events, laboratory parameters, vital signs, and other clinically relevant findings. In addition, the study will explore whether the addition of sivelestat sodium hydrate to standard steroid pulse therapy improves neurological outcomes in patients with acute NMOSD.
Participants will receive intravenous sivelestat sodium hydrate at a dose of 4.8 mg/kg/day administered as a continuous infusion (0.2 mg/kg/hour) for 5 consecutive days, receive steroid pulse therapy according to the study protocol, and be followed for 28 days after treatment initiation for safety and efficacy evaluations.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sivelestat | Experimental | Drug: Sivelestat sodium hydrate |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Patients diagnosed with anti-AQP4 antibody-positive NMOSD according to the international diagnostic criteria for NMOSD (Wingerchuk, Neurology 2015).
- Patients experiencing a relapse including any of the following, with the relapse occurring within 14 days of obtaining consent:
- Patients whose FS domain has worsened by at least 1 point due to relapse.
- Patients with one or more relapse lesions identified on MRI (however, if the relapse is considered to have occurred in the same location as an existing MRI lesion neurologically, identification of a new relapse lesion is not required).
- Patients aged 18 years or older at the time of obtaining consent.
Key exclusion criteria (15 listed)
- Patients with multi-organ dysfunction involving 4 or more organs.
- Patients with severe chronic respiratory disease.
- Patients with autoimmune diseases other than NMOSD that are expected to require additional treatment during the study period.
- Patients with active systemic bacterial, viral, or fungal infections.
- Patients who have received either or both of the following prior treatments after an NMOSD relapse:
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 |
|---|---|---|
| Sivelestat sodium hydrateSivelestat sodium hydrate is administered intravenously at a dose of 4.8 mg/kg/day as a continuous infusion (0.2 mg/kg/hour) for 5 consecutive days in combination with steroid pulse therapy in patients with acute NMOSD attacks. | Drug | Sivelestat |
Primary outcome
Incidence of adverse events
Time frame: 4 weeks
14 secondary outcomes
Change from baseline in body temperature at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration.
Time frame: 6 days
Change from baseline in blood pressure at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration.
Time frame: 6 days
Change from baseline in pulse rate at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration.
Time frame: 6 days
Change from baseline in percutaneous arterial oxygen saturation at 3 hours, Day 2, Day 3, Day 4, Day 5, and Day 6 after the start of investigational product administration.
Time frame: 6 days
Change from baseline in Expanded Disability Status Scale (EDSS) score at 4 weeks (Day 28).
Time frame: 4 weeks
Proportion of cases where Expanded Disability Status Scale (EDSS) score improved by 1 point or more from baseline at 4 weeks (Day 28).
Time frame: 4 weeks
Change from baseline in Functional System (FS) domain scores at 4 weeks
Time frame: 4 weeks
Change from baseline in Opticospinal Impairment Scale (OSIS) score at 4 weeks (Day 28).
Time frame: 4 weeks
Proportion of participants with recovery to pre-relapse neurological disability status at 4 weeks (Day 28).
Time frame: 4 weeks
Change from baseline in best-corrected visual acuity at 4 weeks (Day 28)
Time frame: 4 weeks
Change from baseline in critical flicker fusion frequency at 4 weeks (Day 28).
Time frame: 4 weeks
Change from baseline in retinal nerve fiber layer and ganglion cell-inner plexiform layer thickness measured by optical coherence tomography (OCT) at 4 weeks (Day 28).
Time frame: 4 weeks
Proportion of cases with gadolinium-enhancing lesions on MRI at 4 weeks.
Time frame: 4 weeks
Proportion of cases requiring a second course of steroid pulse therapy, plasmapheresis, or high-dose immunoglobulin therapy.
Time frame: 4 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2026.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Sivelestat sodium hydrate in people with Neuromyelitis Optica Spectrum Disorder Attack, run by Kyushu University at 1 site in Japan.
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 9 inclusion and 15 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: looking for participants now, as of the sponsor's last update on 7 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mitsuru Watanabe, MD, PhD (+81926425340, shinkein@med.kyushu-u.ac.jp), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (time frame: 4 weeks). 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 Oct 2026.