Phase III Clinical Study of NPB-01 in Patients With Autoimmune Encephalitis

JRCT ID: jRCT2061210070

Registered date:17/01/2022

Basic Information

• Recruitment status: Pending
• Health condition(s) or Problem(s) studied: Autoimmune Encephalitis
• Date of first enrollment: 03/03/2022
• Target sample size: 40
• Study type: Interventional

Intervention(s)

- Experimental: NPB-01: Intravenous immunoglobulin
- 400 mg(8mL) /kg/d (max 1g/d) , 5 consecutive days, IV administration
- Active Comparator: NPB-01-ME: Methylprednisolone sodium succinate
- 30 mg/kg/d (max 1g/d) 5 consecutive days, IV administration

Outcome(s)

Primary Outcome

- 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.
[Time Frame: 4 weeks]

Secondary Outcome

1) CASE
2) mRS
3) GCS
4) MMSE-J
5) FAB
6) Disappearance of abnormal EEG findings
7) Disappearance of abnormal head MRI findings
8) Cerebrospinal fluid test
9) Duration of hospitalization

Key inclusion & exclusion criteria

• Age minimum: >= 15age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1) Patients aged 15 years or older at the time of informed consent
2) Patients who meet any of the following (1) to (6):
(1) Definite diagnostic criteria for autoimmune limbic encephalitis
(2) MRI evidence of demyelination (probable autoimmune encephalitis)
(3) Probabilistic diagnostic criteria for anti-NMDAR encephalitis
(4) Probabilistic diagnostic criteria for Bickerstaff brainstem encephalitis
(5) Probabilistic diagnostic criteria for Hashimoto's encephalopathy
(6) Diagnostic Criteria for Autoimmune Encephalitis with Negative but Probable Autoantibodies
3) CASE score of 5 to 22
4) Patients who have had an inadequate response to steroid pulse therapy

Exclude criteria

1) Patients with strongly suspected infectious encephalitis
2) Patients who received immunoglobulin preparations within 8 weeks prior to informed consent
3) Patients who received plasma exchange within 4 weeks prior to informed consent
4) Patients who received immunosuppressants (Rituximab, cyclophosphamide, etc.) within 4 weeks prior to informed consent
5) Patients who have had tumor resection associated with autoimmune encephalitis within 4 weeks prior to informed consent
6) Patients with a history of shock or hypersensitivity to the ingredients of NPB-01
7) Patients with known IgA deficiency
8) Patients with renal disorder
9) 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.)
10) Patients at high risk of thromboembolism
11) Patients with haemolytic/blood loss anaemia
12) Immunosuppressed/immunocompromised patients
13) Patients with decreased cardiac function
14) Pregnant, expected (desired or planned) pregnant, or breastfeeding patients
15) Use of prohibited medications or treatment in this study
16) Patients who received investigational product in this study (re-enrollment prohibited)
17) Patients who have received treatment with investigational product other than this study within 4 months prior to informed consent
18) Patients with a history of hypersensitivity to methylprednisolone sodium succinate
19) Patients who have a tumor associated with autoimmune encephalitis and are considered to require resection during the study period.
20) Patients receiving intravenous general anesthetics or sedative hypnotics
21) Patients in coma
22) Ventilated patients
23) Patients who cannot undergo protocol-specified tests/assessments
24) Other patients considered ineligible for the study by the investigator or subinvestigator
25) Positive herpes simplex virus DNA qualitative test in the screening period.
26) Serum creatinine 2 times or more the upper limit of normal during the screening period.
27) Total protein 9 g/dL or more during the screening period.
28) Patients with hematocrit 55% or more during the screening period

Related Information

• Primary Sponsor: Ota Mamoru