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

Guillain-Barre Syndrome Clinical Trial

Official title:

Assessment of Chronic GBS Improvement With Use of 4-AP

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00056810
• Other study ID #: 2129
• Status: Completed
• Phase: Phase 2
• First received: March 24, 2003
• Last updated: March 24, 2015
• Start date: September 2002
• Est. completion date: May 2005

Study information

• Verified date: May 2006
• Source: FDA Office of Orphan Products Development
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: May 2005
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 19 Years to 75 Years

Eligibility

Inclusion Criteria

• Male or Female, 19 to 75 years of age, irrespective of race.

• Subject is able to and has voluntarily given informed consent prior to the performance of any study specific procedures.

• Subject has neurological impairment secondary to GBS, which has been stable for more than 12 months.

• Subject has motor strength that averages less than 5.0 but greater than 3.0 on the ASIA motor scale.

• Subject is able and willing to comply with protocol.

• Subjects will agree to no change in their outpatient therapy, or home exercise programs during enrollment in the study.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double-Blind

Related Conditions & MeSH terms

• Guillain-Barre Syndrome

Intervention

Drug:
• 4-aminopyridine (4-AP)

Locations

Country	Name	City	State
United States	Rehabilitation Institute of Michigan at Detroit Medical Center	Detroit	Michigan

Sponsors (1)

Lead Sponsor	Collaborator
FDA Office of Orphan Products Development

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	American Spinal Injury Association (ASIA) Motor Score at 8 weeks and 19 weeks
Primary	Functional Independence Measure (FIM) Motor scale at 8 weeks and 19 weeks
Secondary	The following are all at 8 weeks and 19 weeks: Hand Dynamometer
Secondary	Visual Analog Pain Scale
Secondary	McGill Pain Questionnaire-Short Form
Secondary	Neuromuscular Functional Assessment Index
Secondary	Jebsen-Taylor Hand Function Test
Secondary	Minnesota Rate of Manipulation and Manual Dexterity Tests
Secondary	The Get Up and Go Test
Secondary	6-Minute Walk Test
Secondary	Craig Handicap Assessment and Reporting Technique (CHART
Secondary	SF-12 Health Survey
Secondary	Center for Epidemiological Studies Depression Scale (CES-D)
Secondary	Positive and Negative Affect Schedule (PANAS)