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Completed

NCT Number: NCT00056810

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

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.

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Key information

Conditions

Guillain-Barre Syndrome Autoimmune Diseases Autoimmune Diseases of the Nervous System Chronic Disease Demyelinating Diseases Disease Attributes Immune System Diseases Nervous System Diseases Neuromuscular Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Peripheral Nervous System Diseases Polyneuropathies Polyradiculoneuropathy Post-Infectious Disorders

Age range

19 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Rehabilitation Institute of Michigan at Detroit Medical Center

Detroit, Michigan, 48201-2417, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

4-aminopyridine (4-AP)

Drug

Primary outcomes

  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 outcomes

  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)

Sponsors and collaborators

Lead sponsor

FDA Office of Orphan Products Development

Fed

Registry information

Official study title

Assessment of Chronic GBS Improvement With Use of 4-AP

Important dates

Study start
2002
Study completion
2005
First posted
Mar 25, 2003
Registry last updated
Mar 25, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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