Assessment of Chronic Guillain-Barre Syndrome Improvement With Use of 4-Aminopyridine - Full Text View

Sponsor:	FDA Office of Orphan Products Development
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00056810

Purpose

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.

Condition	Intervention	Phase
Guillain-Barre Syndrome	Drug: 4-aminopyridine (4-AP)	Phase II

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Double-Blind
Official Title:	Assessment of Chronic GBS Improvement With Use of 4-AP

Resource links provided by NLM:

Genetics Home Reference related topics: Guillain-Barré syndrome

MedlinePlus related topics: Guillain-Barre Syndrome Potassium

Drug Information available for: 4-Aminopyridine

U.S. FDA Resources

Further study details as provided by FDA Office of Orphan Products Development:

Primary Outcome Measures:

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:

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

Estimated Enrollment:	30
Study Start Date:	September 2002
Estimated Study Completion Date:	May 2005

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.

Eligibility

Ages Eligible for Study:	19 Years to 75 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00056810

Locations

United States, Michigan
Rehabilitation Institute of Michigan at Detroit Medical Center
Detroit, Michigan, United States, 48201-2417

Sponsors and Collaborators

FDA Office of Orphan Products Development

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00056810 History of Changes
Other Study ID Numbers:	2129
Study First Received:	March 24, 2003
Last Updated:	May 18, 2006
Health Authority:	United States: Food and Drug Administration

Keywords provided by FDA Office of Orphan Products Development:

Guillain Barre Syndrome
4-aminopyridine

Additional relevant MeSH terms:

Guillain-Barre Syndrome
Polyradiculoneuropathy
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Polyneuropathies
Autoimmune Diseases

Immune System Diseases
4-Aminopyridine
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Cardiovascular Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on February 13, 2012