Phase III Clinical Trial of NPB-01 in Patients With Guillain-Barré Syndrome

Patients diagnosed with Guillain-Barré syndrome were confirmed based on the diagnostic criteria for Guillain-Barré syndrome. Patients who meet all inclusion criteria and do not conflict with the exclusion criteria will receive NPB-01 (intravenous immunoglobulin) 400mg/kg/day for five consecutive days.

Patients evaluate the Functional Grade（FG） and Arm Grade（AG） et al.

As a safety endpoint, the safety of NPB-01 will be investigated the occurrence of adverse events by the start of the study treatment.

Study Overview

• Status: Completed
• Conditions: Guillain-Barré Syndrome
• Intervention / Treatment: Drug: NPB-01

• Study Type: Interventional
• Enrollment (Actual): 22
• Phase: Phase 3

Contacts and Locations

Study Locations

• Japan
  • Osaka, Japan
    • Nihon Pharmaceutical Co., Ltd

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. In principle, patients are able to receive study drug within 2weeks(with limits of 4weeks) from the start of symptoms.
2. Patients with predominant motor neuropathy and Hughes's Functional Grade(FG) is grade4 or grade5(in this regard, if symptoms is progressive, patients with FG is grade3 involve in this study).
3. Patients with plasmapheresis, steroids(in prednisolone equivalent, more than 100mg/day) and immune globulin therapy is no operation for this onset.
4. Patients with greater than or equal to 18 years old at informed consent.

Exclusion Criteria:

1. Patients with history of shock for NPB-01.
2. Patients with history of hypersensitivity for NPB-01.
3. Patients with history of volatile organic solvent abuse, abnormal porphyrin metabolism, history of pharynx or cutaneous diphtheria, plumbism, tephromyelitis, botulism, hysterical paralysis, toxic neuropathy(nitrofurantoin, dapsone, organophosphorous compound), serious diabetic neuropathy,peripheral neuropathy due to HIV,impaired peripheral neuropathy except Guillain-Barré syndrome.
4. Patients with malignancy at informed consent.
5. Patients treated with immune globulin at 8 weeks before informed consent.
6. Patients with IgA deficiency.
7. Patients with impaired liver function.
8. Patients with impaired renal function.
9. Patients with cerebro- or cardiovascular disorders.
10. Patients with high risk of thromboembolism.
11. Patients with hemolytic/hemorrhagic anemia.
12. Patients with decreased cardiac function.
13. Patients with decreased platelet.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NPB-01	Drug: NPB-01

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
proportion of patients with more than 1grade improvement in the Hughes functional grading scale(FG) relative to baseline at 4weeks.	Baseline,4weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
days required for 1 grade improvement of the Hughes functional grading scale(FG)	Baseline,1,2,3,4,5,6,7,8,9,10,11,12 weeks
days required for 2 grade improvement of the Hughes functional grading scale(FG)	Baseline,1,2,3,4,5,6,7,8,9,10,11,12 weeks
changes in Hughes functional grading scale(FG)	Baseline,1,2,3,4,5,6,7,8,9,10,11,12 weeks
proportion of patients with more than 1 grade improvement in the Arm Grade(AG) relative to baseline at 4weeks.	Baseline,4weeks
days required for 1 grade improvement of the Arm Grade(AG)	Baseline,1,2,3,4,5,6,7,8,9,10,11,12 weeks
days required for 2 grade improvement of the Arm Grade(AG)	Baseline,1,2,3,4,5,6,7,8,9,10,11,12 weeks
changes in Arm Grade(AG)	Baseline,1,2,3,4,5,6,7,8,9,10,11,12 weeks
changes in manual muscle testing(MMT)	Baseline,1,2,3,4,8,12 weeks
changes in grip strength	Baseline,1,2,3,4,8,12 weeks
changes in activity of daily living(ADL)	Baseline,1,2,3,4,8,12 weeks
changes in electrophysiological findings	Baseline,4,12 weeks

Collaborators and Investigators

• Sponsor: Nihon Pharmaceutical Co., Ltd

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2014
• Primary Completion (Actual): August 1, 2015
• Study Completion (Actual): August 1, 2015

Study Registration Dates

• First Submitted: August 12, 2014
• First Submitted That Met QC Criteria: August 19, 2014
• First Posted (Estimate): August 20, 2014

Study Record Updates

• Last Update Posted (Actual): April 12, 2017
• Last Update Submitted That Met QC Criteria: April 11, 2017
• Last Verified: April 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Disease; Neuromuscular Diseases; Peripheral Nervous System Diseases; Polyradiculoneuropathy; Polyneuropathies; Syndrome; Guillain-Barre Syndrome
• Other Study ID Numbers: NPB-01-13/C-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No