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Effectiveness of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome

Descriptive Information
Brief Title † Effectiveness of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome
Official Title † Inpatient Double-Blind Placebo-Controlled Withdrawal Study of 3,4-Diaminopyridine Base (3,4-DAP) in Subjects With Known Lambert-Eaton Myasthenic Syndrome
Brief Summary Hypothesis: 3,4-DAP improves Lambert-Eaton Myasthenic Syndrome (LEMS)-related weakness.
Detailed Description The objectives of the study are to confirm the safety and to test the efficacy of 3,4-DAP in the treatment of LEMS-related weakness. This is a phase 2 randomized double-blind placebo-controlled withdrawal study in subjects with known clinically active LEMS who have been on a chronic stable dose of compassionate distribution Jacobus 3,4-DAP provided through FDA-approved individual investigator-held INDs.
Study Phase Phase 2
Study Type † Interventional
Study Design † Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary Outcome Measure † Triple Timed Up & Go (TUG) Test
Secondary Outcome Measure † Self-assessment of LEMS-related weakness
Condition † Lambert-Eaton Myasthenic Syndrome Eaton-Lambert Myasthenic Syndrome
Intervention † Drug3,4-DAP
Study Arms / Comparison Groups 3,4-DAP Half of the subjects will be randomized to continue taking their usual regimen of 3,4-DAP 3,4-DAP Taper to Placebo Half of the subjects will be randomized to gradually taper their dose of 3,4-DAP down to placebo
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 30
Start Date † January 2012
Completion Date July 2014
Primary Completion Date February 2014
Eligibility Criteria † Inclusion Criteria: 1. Age 18 or over 2. Ambulatory while taking 3,4-DAP, i.e. the patient can perform the timed up and go (TUG), either with or without an assistive device 3. Established diagnosis of LEMS, with documentation provided 4. Continuous use of JPC 3,4-DAP for at least 3 months 5. Minimum of 3 doses per day with no single dose less than 10 mg of 3,4-DAP 6. The patient needs 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] 7. Stable regimen of all LEMS-related treatments for at least 3 months 8. Stable daily regimen of other medications (prescription and over-the-counter) for a minimum of 1 month 9. Willing to chance being tapered off of 3,4-DAP 10. Fluency in English 11. If applicable, agrees to use birth control during heterosexual intercourse until at least 2 weeks after completion of study 12. A signed informed consent by the study subject Exclusion Criteria: 1. Last monoclonal antibody treatment (e.g. rituximab) was less than 6 months ago (i.e., recent treatment is an exclusion) 2. 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 3. Respiratory failure requiring intubation while on 3,4-DAP with no precipitating event or medication 4. Use of any investigational drug other than 3,4-DAP within the last 30 days 5. Pregnant or lactating 6. Current use of other aminopyridines (e.g.4-AP) or guanidine 7. Does 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
Gender Both
Ages 18 Years - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT01511978
Organization ID JPC 3,4-DAPPER
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Jacobus Pharmaceutical
Collaborators ††
Investigators † Study Director: Kathy L Aleš, MD, Jacobus Pharmaceutical
Information Provided By
Verification Date July 2014
First Received Date † December 24, 2011
Last Updated Date July 1, 2014
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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