Efficacy and Safety Study of GB-0998 for Guillain-Barré Syndrome

This study will carry out to assess the efficacy of GB-0998 (intravenous immunoglobulin;400mg/kg/day for five days) in the treatment of the Guillain-Barré Syndrome based on the changes in Hughes Functional Grade (FG) as primary endpoint, and in addition, to assess the safety of GB-0998.

Study Overview

• Status: Completed
• Conditions: Guillain-Barré Syndrome
• Intervention / Treatment: Drug: GB-0998

• Study Type: Interventional
• Enrollment (Actual): 21
• Phase: Phase 3

Contacts and Locations

Study Locations

• Japan
  • Saitama
    • Kawagoe, Saitama, Japan
      • Department of Neurology, Saitama Medical Center, Saitama Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. In principle, patients are able to receive the treatment within 2 weeks (with limits of 4 weeks) from the start of symptoms.
2. Patients with predominant motor neuropathy and FG is grade 4 or grade 5 (if symptoms is progressive, patients with FG is grade 3 involve in this study).
3. Patients with plasmapheresis, steroids and immune globulin therapy is no operation for this onset.

Exclusion Criteria:

1. Patients who have the anamnesis of shock or hypersensitivity to GB-0998.
2. Patients who have been diagnosed as hereditary fructose intolerance.
3. Patients who have impaired peripheral neuropathy except Guillain-Barré syndrome.
4. Patients with history of volatile organic solvent abuse, abnormal porphyrin metabolism, history of pharynx or cutaneous diphtheria, plumbism, poliomyelitis, botulism, hysterical paralysis, toxic neuropathy.
5. Patients who have received treatment of malignant tumors.
6. Patients who were administered immunoglobulin within 8 weeks before informed consent.
7. Patients who have been diagnosed IgA deficiency in their past history.
8. Patients with severe renal disorder or decreased cardiac function.
9. Patients who have the anamnesis of cerebro- or cardiovascular disorders, or symptom of these diseases.
10. Patients with high risk of thromboembolism.
11. Pregnant, lactating, and probably pregnant patients.
12. Patients who were administered other investigational drug within 12 weeks before consent.

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: GB-0998	Drug: GB-0998; Other Names: Venoglobulin-IH

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Proportion of patients with more than 1 grade improvement in Hughes Functional Grade (FG)	4 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
days required for 1 grade improvement of FG	1,2,3,4,6,8,12 weeks
days required for 2 grade improvement of FG	1,2,3,4,6,8,12 weeks
changes in FG	1,2,3,4,6,8,12 weeks
proportion of patients with more than 1 grade improvement in the Arm Grade (AG) relative to baseline	4weeks
days required for 1 grade improvement of the AG	1,2,3,4,6,8,12 weeks
days required for 2 grade improvement of the AG	1,2,3,4,6,8,12 weeks
changes in AG	1,2,3,4,6,8,12 weeks
changes in grip strength	1,2,4,8,12 weeks
changes in manual muscle testing (MMT)	1,2,4,8,12 weeks
changes in activity of daily living (ADL)	1,2,4,8,12 weeks
changes in motor nerve conduction velocity	4,12 weeks
changes in FG on rescue treatment	1,2,3,4,6,8,12 weeks

Collaborators and Investigators

• Sponsor: Japan Blood Products Organization
• Investigators: Study Chair: Kyoichi Nomura, Department of Neurology, Saitama Medical Center, Saitama Medical University, Saitama, Japan

Study record dates

Study Major Dates

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

Study Registration Dates

• First Submitted: December 25, 2014
• First Submitted That Met QC Criteria: January 14, 2015
• First Posted (Estimate): January 19, 2015

Study Record Updates

• Last Update Posted (Estimate): February 4, 2016
• Last Update Submitted That Met QC Criteria: February 3, 2016
• Last Verified: December 1, 2015

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; Physiological Effects of Drugs; Immunologic Factors; Immunoglobulins, Intravenous
• Other Study ID Numbers: B211-13