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

Nihon Pharmaceutical

Status and phase: Enrolling; Phase 3

Conditions: Autoimmune Encephalitis

Treatments: Drug: NPB-01-ME; Drug: NPB-01

Study type: Interventional

Funder types: Industry

Identifiers

NCT05177939

NPB-01-19/C-01

Details and patient eligibility

About

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.

Enrollment: 40 estimated patients

Sex: All

Ages: 15+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

• < 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):

  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

• < 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

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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40 participants in 2 patient groups

NPB-01

Experimental group

Description:

Intravenous immunoglobulin

Treatment:

Drug: NPB-01

NPB-01-ME

Active Comparator group

Description:

methylprednisolone sodium succinate

Treatment:

Drug: NPB-01-ME