Autoimmune EncephalitisPhase 3NCT05177939
Phase III Clinical Study of NPB-01 in Patients With Autoimmune Encephalitis
A Phase 3 study testing NPB-01 and NPB-01-ME in people with Autoimmune Encephalitis, run by Nihon Pharmaceutical Co., Ltd at 1 site in Japan.
Trial summary
To compare the efficacy and safety of NPB-01 in patients with autoimmune encephalitis refractory to steroid pulse therapy using steroid pulse therapy as a control. Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Autoimmune Encephalitis
- What is being tested?
- NPB-01 and NPB-01-ME
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteJapan
- Study dates
- 3 Mar 2022to 31 Oct 2024
- Phase
- Phase 3
- 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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- < At 1st registration > Patients meeting the possible diagnostic criteria for autoimmune encephalitis
- < At 1st registration > Patients with a CASE score of 5 to 22 during the screening period
- < At 1st registration > Patients with autoimmune encephalitis in progress (active and requiring therapeutic intervention)
- < At 1st registration > IVIG therapy and steroid pulse therapy are considered necessary by the investigator.
- < At 1st registration > Patients aged 15 years or older at the time of informed consent
- < At 2nd registration > Patients who meet any of the following (1) to (6):
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- Definite diagnostic criteria for autoimmune limbic encephalitis
- MRI evidence of demyelination (probable autoimmune encephalitis)
- Probabilistic diagnostic criteria for anti-NMDAR encephalitis
- Probabilistic diagnostic criteria for Bickerstaff brainstem encephalitis
- Probabilistic diagnostic criteria for Hashimoto's encephalopathy
- Diagnostic Criteria for Autoimmune Encephalitis with Negative but Probable Autoantibodies
- < At 2nd registration > CASE score of 5 to 22 on Day 8 of the previous treatment period
- < At 2nd registration > Patients who have had an inadequate response to steroid pulse therapy
Who cannot join
- < At 1st registration > Patients with strongly suspected infectious encephalitis
- < At 1st registration > Patients who received immunoglobulin preparations within 8 weeks prior to informed consent
- < At 1st registration > Patients who received plasma exchange within 4 weeks prior to informed consent
- < At 1st registration > Patients who received immunosuppressants (Rituximab, cyclophosphamide, etc.) within 4 weeks prior to informed consent
- < At 1st registration > Patients who have had tumor resection associated with autoimmune encephalitis within 4 weeks prior to informed consent
- < At 1st registration > Patients with a history of shock or hypersensitivity to the ingredients of NPB-01
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- < At 1st registration > Patients with known IgA deficiency
- < At 1st registration > Patients with renal disorder
- < At 1st registration > Patients with a current or previous history of cerebral or cardiovascular disorders (Asymptomatic cerebral infarction and myocardial infarction that occurred more than 5 years ago are not applicable.)
- < At 1st registration > Patients at high risk of thromboembolism
- < At 1st registration > Patients with haemolytic/blood loss anaemia
- < At 1st registration > Immunosuppressed/immunocompromised patients
- < At 1st registration > Patients with decreased cardiac function
- < At 1st registration > Pregnant, expected (desired or planned) pregnant, or breastfeeding patients
- < At 1st registration > Use of prohibited medications or treatment in this study
- < At 1st registration > Patients who received investigational product in this study (re-enrollment prohibited)
- < At 1st registration > Patients who have received treatment with investigational product other than this study within 4 months prior to informed consent
- < At 1st registration > Patients with a history of hypersensitivity to methylprednisolone sodium succinate
- < At 1st registration > Patients who have a tumor associated with autoimmune encephalitis and are considered to require resection during the study period.
- < At 1st registration > Patients receiving intravenous general anesthetics or sedative hypnotics
- < At 1st registration > Patients in coma
- < At 1st registration > Ventilated patients
- < At 1st registration > Patients who cannot undergo protocol-specified tests/assessments
- < At 1st registration > Other patients considered ineligible for the study by the investigator
- < At 2nd registration > Positive herpes simplex virus DNA qualitative test in the screening period.
- < At 2nd registration > Serum creatinine ≥ 2 times the upper limit of normal during the screening period.
- < At 2nd registration > Total protein ≥ 9 g/dL during the screening period.
- < At 2nd registration > Patients with hematocrit ≥ 55% during the screening period
- < At 2nd registration > Patients who meet any of the exclusion criteria at the time of first registration
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase III Study to Evaluate the Efficacy and Safety of NPB-01 in Patients With Autoimmune Encephalitis Refractory to Steroid Pulse Therapy
To compare the efficacy and safety of NPB-01 in patients with autoimmune encephalitis refractory to steroid pulse therapy using steroid pulse therapy as a control.
- Conditions
- Autoimmune Encephalitis
- Being tested
- NPB-01, NPB-01-ME
- Medical subject headings
- Autoimmune Diseases of the Nervous System
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NPB-01Drug
Also called Intravenous immunoglobulin
NPB-01 will be administered for the treatment of autoimmune encephalitis
NPB-01-MEDrug
Also called methylprednisolone sodium succinate
NPB-01-ME will be administered for the treatment of autoimmune encephalitis
| Study group | Role | What they receive |
|---|---|---|
| NPB-01 | Experimental | Intravenous immunoglobulin Receives: Drug: NPB-01 |
| NPB-01-ME | Active comparator | methylprednisolone sodium succinate Receives: Drug: NPB-01-ME |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of responders in CASE (Clinical Assessment Scale in Autoimmune Encephalitis)
A responder is defined as a patient whose CASE score at Week 4 of the post-treatment follow-up period after treatment with investigational product improved by 40% or more compared to the pre-treatment period.
Measured: 4 weeks
Secondary outcomes (11)
CASE
The change in CASE score at each time point after the start of treatment with investigational product compared with that on Day 8 of the pretreatment period will be compared between the arms. Changes in CASE scores divided into three segments (0 -4: excellent, 5 -9: moderate, 10 -27: poor) will also be compared.
In addition, the period until CASE score becomes 4 points or less after the start of treatment with investigational product will be checked.
Measured: 1, 2, 3, 4, 6, 8, 12 weeks
mRS
Changes in mRS at each time point after the start of investigational product treatment compared with Day 8 of the pretreatment period will be compared between the arms.
Measured: 1, 2, 3, 4, 6, 8, 12 weeks
GCS
To compare the change in GCS at each time point after the start of investigational product with that on Day 8 of the pretreatment period between the arms.
Measured: 1, 2, 3, 4, 6, 8, 12 weeks
MMSE-J
The change in MMSE-J at each time point after the start of investigational product as compared with Day 8 of the pretreatment period will be compared between the arms.
Measured: 4, 8, 12 weeks
FAB
The change in FAB at each time point after the start of investigational product as compared with Day 8 of the pretreatment period will be compared between the arms.
Measured: 4, 8, 12 weeks
Disappearance of abnormal EEG findings
The proportion of subjects in whom abnormal findings in EEG disappeared at each time point after the start of investigational product as compared with Day 8 of the pretreatment period will be compared between the arms.
Measured: 4, 12 weeks
Disappearance of abnormal head MRI findings
The proportion of subjects in whom abnormal findings in head MRI disappeared at each time point after the start of investigational product as compared with Day 8 of the pretreatment period will be compared between the arms.
Measured: 4, 12 weeks
Cerebrospinal fluid test
The proportion of subjects in whom the cell count returned to within the reference range (≤ 5/μl) and the proportion of subjects in whom the protein count returned to within the reference range (15.0 ~ 45.0 mg/dL) at each time point after the start of investigational product treatment as compared with Day 8 of the pretreatment period will be checked.
Measured: 4, 12 weeks
Duration of hospitalization
Duration of hospitalization after the start of treatment with investigational product to be compared between the arms.
Measured: 12 weeks
mRS proportion
The proportions of subjects with an mRS score of ≤ 2, subjects with an improvement of ≥ 1 point, and subjects with an improvement of ≥ 2 points will also be compared.
Also, the time to mRS improvement after the start of treatment with investigational product (≤ 2 points, ≥ 1 point improvement, ≥ 2 points improvement) .
Measured: 1, 2, 3, 4, 6, 8, 12 weeks
GCS proportion
Changes in GCS when divided into three segments (15-13: Mild, 12-9: Moderate, 8-3: Severe) will also be compared.
In addition, the period until the GCS score reaches 13 or higher after the start of treatment with investigational product will be checked.
Measured: 1, 2, 3, 4, 6, 8, 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2022
- Study start3 Mar 2022
- Primary completion31 May 2024 (estimated)
- Study completion31 Oct 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune EncephalitisStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nihon Pharmaceutical Co., Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mamoru Ota, Study chair, Nihon Pharmaceutical Co., Ltd
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05177939
- Sponsor's protocol ID
- NPB-01-19/C-01
- Status last verified
- Jan 2022
- First submitted
- 3 Dec 2021
- Last updated
- 1 Dec 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase III Study to Evaluate the Efficacy and Safety of NPB-01 in Patients With Autoimmune Encephalitis Refractory to Steroid Pulse Therapy
- ClinicalTrials.gov ID
- NCT05177939
- Acronym / sponsor ID
- NPB-01-19/C-01
- Conditions
- Autoimmune Encephalitis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Nihon Pharmaceutical Co., LtdIndustry (pharma, biotech, devices)
- Study start
- 3 Mar 2022 (actual)
- Primary completion
- 31 May 2024 (estimated)
- Study completion
- 31 Oct 2024 (estimated)
- First posted
- 5 Jan 2022 (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.
To compare the efficacy and safety of NPB-01 in patients with autoimmune encephalitis refractory to steroid pulse therapy using steroid pulse therapy as a control.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NPB-01 | Experimental | Drug: NPB-01Intravenous immunoglobulin |
| NPB-01-ME | Active comparator | Drug: NPB-01-MEmethylprednisolone sodium succinate |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (14 listed)
- < At 1st registration > Patients meeting the possible diagnostic criteria for autoimmune encephalitis
- < At 1st registration > Patients with a CASE score of 5 to 22 during the screening period
- < At 1st registration > Patients with autoimmune encephalitis in progress (active and requiring therapeutic intervention)
- < At 1st registration > IVIG therapy and steroid pulse therapy are considered necessary by the investigator.
- < At 1st registration > Patients aged 15 years or older at the time of informed consent
Key exclusion criteria (29 listed)
- < At 1st registration > Patients with strongly suspected infectious encephalitis
- < At 1st registration > Patients who received immunoglobulin preparations within 8 weeks prior to informed consent
- < At 1st registration > Patients who received plasma exchange within 4 weeks prior to informed consent
- < At 1st registration > Patients who received immunosuppressants (Rituximab, cyclophosphamide, etc.) within 4 weeks prior to informed consent
- < At 1st registration > Patients who have had tumor resection associated with autoimmune encephalitis within 4 weeks prior to informed consent
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 |
|---|---|---|
| NPB-01Also: Intravenous immunoglobulinNPB-01 will be administered for the treatment of autoimmune encephalitis | Drug | NPB-01 |
| NPB-01-MEAlso: methylprednisolone sodium succinateNPB-01-ME will be administered for the treatment of autoimmune encephalitis | Drug | NPB-01-ME |
Primary outcome
Proportion of responders in CASE (Clinical Assessment Scale in Autoimmune Encephalitis)
Time frame: 4 weeks
A responder is defined as a patient whose CASE score at Week 4 of the post-treatment follow-up period after treatment with investigational product improved by 40% or more compared to the pre-treatment period.
11 secondary outcomes
CASE
Time frame: 1, 2, 3, 4, 6, 8, 12 weeks
mRS
Time frame: 1, 2, 3, 4, 6, 8, 12 weeks
GCS
Time frame: 1, 2, 3, 4, 6, 8, 12 weeks
MMSE-J
Time frame: 4, 8, 12 weeks
FAB
Time frame: 4, 8, 12 weeks
Disappearance of abnormal EEG findings
Time frame: 4, 12 weeks
Disappearance of abnormal head MRI findings
Time frame: 4, 12 weeks
Cerebrospinal fluid test
Time frame: 4, 12 weeks
Duration of hospitalization
Time frame: 12 weeks
mRS proportion
Time frame: 1, 2, 3, 4, 6, 8, 12 weeks
GCS proportion
Time frame: 1, 2, 3, 4, 6, 8, 12 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Dec 2022.
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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.
Mamoru Ota study chair
Nihon Pharmaceutical Co., Ltd
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NPB-01 and NPB-01-ME in people with Autoimmune Encephalitis, run by Nihon Pharmaceutical Co., Ltd 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 14 inclusion and 29 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 1 Dec 2022. 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Mamoru Ota (03-5148-7574, kaihatsu@nihon-pharm.co.jp), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of responders in CASE (Clinical Assessment Scale in Autoimmune Encephalitis) (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 1 Dec 2022.