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

NCT05177939 | Unknown Phase 3

• 1 other identifier: NPB-01-19/C-01
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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.

Conditions

Autoimmune Encephalitis

Outcome Measures

Primary Outcomes (1)

• 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.

  4 weeks

Secondary Outcomes (11)

• CASE

  1, 2, 3, 4, 6, 8, 12 weeks

• mRS

  1, 2, 3, 4, 6, 8, 12 weeks

• GCS

  1, 2, 3, 4, 6, 8, 12 weeks

• MMSE-J

  4, 8, 12 weeks

• FAB

  4, 8, 12 weeks

Study Arms (2)

NPB-01

EXPERIMENTAL

Intravenous immunoglobulin

Drug: NPB-01

NPB-01-ME

ACTIVE COMPARATOR

methylprednisolone sodium succinate

Drug: NPB-01-ME

Interventions

NPB-01 DRUG

NPB-01 will be administered for the treatment of autoimmune encephalitis

Also known as: Intravenous immunoglobulin

NPB-01

NPB-01-ME DRUG

NPB-01-ME will be administered for the treatment of autoimmune encephalitis

Also known as: methylprednisolone sodium succinate

NPB-01-ME

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• \< 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):
• 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

You may not qualify if:

• \< 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

Sponsors & Collaborators

• Nihon Pharmaceutical Co., Ltd: lead

Study Sites (1)

Trial site 1

Ube, Yamaguchi, Japan

RECRUITING

MeSH Terms

Conditions

Autoimmune Diseases of the Nervous System

Interventions

Immunoglobulins, Intravenous; Methylprednisolone Hemisuccinate

Condition Hierarchy (Ancestors)

Nervous System Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Methylprednisolone; Prednisolone; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Mamoru Ota

  Nihon Pharmaceutical Co., Ltd

  STUDY CHAIR

Central Study Contacts

Mamoru Ota

CONTACT

03-5148-7574; kaihatsu@nihon-pharm.co.jp

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 3, 2021
• First Posted: January 5, 2022
• Study Start: March 3, 2022
• Primary Completion: May 31, 2024
• Study Completion: October 31, 2024
• Last Updated: December 1, 2022

Record last verified: 2022-01

Locations

JP (1)