Status:
COMPLETED
Effectiveness of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome
Lead Sponsor:
Jacobus Pharmaceutical
Conditions:
Lambert-Eaton Myasthenic Syndrome
Eaton-Lambert Myasthenic Syndrome
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
Hypothesis: 3,4-Diaminopyridine base (3,4-DAP) improves Lambert-Eaton Myasthenic Syndrome (LEMS)-related weakness.
Detailed Description
The objectives of the study were to confirm the safety and to test the efficacy of 3,4-DAP in the treatment of LEMS-related weakness. This was a phase 2 randomized double-blind placebo-controlled wit...
Eligibility Criteria
Inclusion
- Age 18 or over
- Ambulatory while taking 3,4-DAP, i.e. the patient was able to perform the timed up and go (TUG), either with or without an assistive device
- Established diagnosis of LEMS, with documentation provided
- Continuous use of Jacobus 3,4-DAP for at least 3 months
- Minimum of 3 doses per day with no single dose less than 10 mg of 3,4-DAP
- The patient needed to wait about 15 to 30 minutes to experience an unequivocal improvement in a LEMS-induced dysfunction after they take their first dose of 3,4-DAP in the morning \[a patient who remains in bed past this point by choice may still be eligible\]
- Stable regimen of all LEMS-related treatments for at least 3 months
- Stable daily regimen of other medications (prescription and over-the-counter) for a minimum of 1 month
- Willing to chance being tapered off of 3,4-DAP
- Fluency in English
- If applicable, agreed to use birth control during heterosexual intercourse until at least 2 weeks after completion of study
- A signed informed consent by the study subject
Exclusion
- Last monoclonal antibody treatment (e.g. rituximab) was less than 6 months ago (i.e., recent treatment is an exclusion)
- Clinically significant or poorly controlled condition that in the opinion of the study personnel might pose an unacceptable risk to the patient if entered into the study
- Respiratory failure requiring intubation while on 3,4-DAP with no precipitating event or medication
- Use of any investigational drug other than 3,4-DAP within the last 30 days
- Pregnant or lactating
- Current use of other aminopyridines (e.g.4-AP) or guanidine
- Did not display a sufficiently large response to 3,4-DAP during the baseline observation period in the CRU to detect a decline during withdrawal of 3,4-DAP
Key Trial Info
Start Date :
January 1 2012
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2015
Estimated Enrollment :
32 Patients enrolled
Trial Details
Trial ID
NCT01511978
Start Date
January 1 2012
End Date
July 1 2015
Last Update
July 11 2017
Active Locations (7)
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1
University of California at Davis
Sacramento, California, United States, 95817
2
Indiana University
Indianapolis, Indiana, United States, 46202
3
Duke University
Durham, North Carolina, United States, 27710
4
Oregon Health & Science University
Portland, Oregon, United States, 97239