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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00056810
Recruitment Status : Completed
First Posted : March 25, 2003
Last Update Posted : March 25, 2015
Information provided by:
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.

Condition or disease Intervention/treatment Phase
Guillain-Barre Syndrome Drug: 4-aminopyridine (4-AP) Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double
Official Title: Assessment of Chronic GBS Improvement With Use of 4-AP
Study Start Date : September 2002
Study Completion Date : May 2005

Primary Outcome Measures :
  1. American Spinal Injury Association (ASIA) Motor Score at 8 weeks and 19 weeks
  2. Functional Independence Measure (FIM) Motor scale at 8 weeks and 19 weeks

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

Information from the National Library of Medicine

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Ages Eligible for Study:   19 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00056810

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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

Layout table for additonal information Identifier: NCT00056810    
Other Study ID Numbers: 2129
First Posted: March 25, 2003    Key Record Dates
Last Update Posted: March 25, 2015
Last Verified: May 2006
Keywords provided by FDA Office of Orphan Products Development:
Guillain Barre Syndrome
Additional relevant MeSH terms:
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Guillain-Barre Syndrome
Pathologic Processes
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action