Guillain-Barre SyndromePhase 2NCT00056810
Assessment of Chronic Guillain-Barre Syndrome Improvement With Use of 4-aminopyridine
A Phase 2 study testing 4-aminopyridine (4-AP) in people with Guillain-Barre Syndrome, run by FDA Office of Orphan Products Development at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 19 to 75 years
- Conditions the study is for
- Guillain-Barre Syndrome
- What is being tested?
- 4-aminopyridine (4-AP)
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2002to May 2005
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Rehabilitation Institute of Michigan at Detroit Medical Center
Detroit, Michigan, 48201-2417
About this study
Official title: Assessment of Chronic GBS Improvement With Use of 4-AP
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.
Read the 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.
- Conditions
- Guillain-Barre Syndrome
- Being tested
- 4-aminopyridine (4-AP)
- Keywords
- Guillain Barre Syndrome, 4-aminopyridine
- Medical subject headings
- Guillain-Barre Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
4-aminopyridine (4-AP)Drug
What is being measured
The primary outcome is the main question the study is designed to answer.
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 outcomes (12)
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)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2003 (estimated)
- Study startSep 2002
- Study completionMay 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Guillain-Barre SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Enrolment
- 30 participants
Sponsor and investigators
- Lead sponsor
- FDA Office of Orphan Products Development
- Sponsor type
- US federal agency
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00056810
- Sponsor's protocol ID
- 2129
- Status last verified
- May 2006
- First submitted
- 24 Mar 2003
- Last updated
- 25 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Chronic GBS Improvement With Use of 4-AP
- ClinicalTrials.gov ID
- NCT00056810
- Acronym / sponsor ID
- 2129
- Conditions
- Guillain-Barre Syndrome
- Keywords
- Guillain Barre Syndrome, 4-aminopyridine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants
- Lead sponsor
- FDA Office of Orphan Products DevelopmentUS federal agency
- Study start
- Sep 2002
- Study completion
- May 2005
- First posted
- 25 Mar 2003 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
Who may qualify
- Ages
- 19 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 4-aminopyridine (4-AP) | Drug | – |
Primary outcome
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
12 secondary outcomes
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)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 4-aminopyridine (4-AP) in people with Guillain-Barre Syndrome, run by FDA Office of Orphan Products Development at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
American Spinal Injury Association (ASIA) Motor Score at 8 weeks and 19 weeks. The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2015.