Assessment of Chronic Guillain-Barre Syndrome Improvement With Use of 4-aminopyridine

Phase 2

Completed

• Conditions: Guillain-Barre Syndrome

• Registration Number: NCT00056810
• Lead Sponsor: FDA Office of Orphan Products Development

Brief Summary

In developed countries, Guillain-Barre Syndrome (GBS) is the most common cause of acute neuromuscular paralysis, afflicting about 5,000 persons annually in the United States. Over 20% of GBS patients have permanent residual motor deficits that affect their activities of daily living.

The goal of this study is to assess the potential usefulness and safety of 4-aminopyridine (4-AP) in those patients who suffer chronic functional deficits from GBS.This medication is a potassium channel blocker that has the potential to improve nerve conduction, particularly across partially demyelinated axons. It is felt that by increasing nerve conduction there will be improved motor performance for walking and activities of daily living, as well as decreased fatiguability. This medication has demonstrated potential usefulness in central demyelinating diseases such as multiple sclerosis.Because the peripheral nervous system is much more accessible to systemic medication delivery it is felt that this medication may improve the functional status of those patients who are suffering from the residual side effects of this medication.

Detailed Description

Objective.- To determine the safety and efficacy of orally delivered 4-aminopyridine for motor weakness due to Guillain-Barre Syndrome (GBS) under a FDA approved protocol (IND No: 58,029).

Setting.- Tertiary care outpatient rehabilitation center directly attached to a university hospital.

Subjects.- Subjects who are unable to ambulate more than 200 feet without assistive devices and have residual nonprogressive motor weakness due to GBS more than one year out from the initial episode.

Design.- Subjects will be randomized to a double-blind, placebo-controlled, cross-over design, which had two eight-week treatment arms with a three-week washout. The average dosage at 4 weeks will be 30 milligrams (mg) per day.

Patients who demonstrate improvement will be continued on the medication for an additional three months. Assessments will be performed every two weeks during the randomized trial and every month for those continued for up to three months on the medication.

Study Timeline

• Study Start: 2002-09
• Study First Submitted: 2003-03-24
• Study First Submitted QC: 2003-03-24
• Study First Posted: 2003-03-25
• Study Completion: 2005-05
• Status Verified: 2006-05
• Last Update Submitted: 2015-03-24
• Last Update Posted: 2015-03-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Primary Outcome Measures

Name	Time	Method
American Spinal Injury Association (ASIA) Motor Score at 8 weeks and 19 weeks
Functional Independence Measure (FIM) Motor scale at 8 weeks and 19 weeks

Secondary Outcome Measures

Name	Time	Method
The following are all at 8 weeks and 19 weeks: Hand Dynamometer
The Get Up and Go Test
6-Minute Walk Test
Visual Analog Pain Scale
Jebsen-Taylor Hand Function Test
SF-12 Health Survey
Minnesota Rate of Manipulation and Manual Dexterity Tests
Positive and Negative Affect Schedule (PANAS)
McGill Pain Questionnaire-Short Form
Center for Epidemiological Studies Depression Scale (CES-D)
Neuromuscular Functional Assessment Index
Craig Handicap Assessment and Reporting Technique (CHART

Trial Locations

Locations (1)

Rehabilitation Institute of Michigan at Detroit Medical Center; 🇺🇸 Detroit, Michigan, United States