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

Phase 3
Recruiting
Conditions
Autoimmune Encephalitis
Interventions
Registration Number
NCT05177939
Lead Sponsor
Nihon Pharmaceutical Co., Ltd
Brief 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.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
40
Inclusion Criteria

Not provided

Exclusion Criteria
  • < 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
  • < 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

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
NPB-01NPB-01Intravenous immunoglobulin
NPB-01-MENPB-01-MEmethylprednisolone sodium succinate
Primary Outcome Measures
NameTimeMethod
Proportion of responders in CASE (Clinical Assessment Scale in Autoimmune Encephalitis)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.

Secondary Outcome Measures
NameTimeMethod
CASE1, 2, 3, 4, 6, 8, 12 weeks

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.

MMSE-J4, 8, 12 weeks

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.

mRS1, 2, 3, 4, 6, 8, 12 weeks

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.

GCS1, 2, 3, 4, 6, 8, 12 weeks

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.

FAB4, 8, 12 weeks

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.

Duration of hospitalization12 weeks

Duration of hospitalization after the start of treatment with investigational product to be compared between the arms.

Disappearance of abnormal EEG findings4, 12 weeks

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.

Disappearance of abnormal head MRI findings4, 12 weeks

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.

Cerebrospinal fluid test4, 12 weeks

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.

mRS proportion1, 2, 3, 4, 6, 8, 12 weeks

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) .

GCS proportion1, 2, 3, 4, 6, 8, 12 weeks

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.

Trial Locations

Locations (1)

Trial site 1

🇯🇵

Ube, Yamaguchi, Japan

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