This trial is active, not recruiting.

Conditions lambert-eaton myasthenic syndrome, eaton-lambert myasthenic syndrome
Treatments 3,4-dap, 3,4-dap taper to placebo
Phase phase 2
Sponsor Jacobus Pharmaceutical
Start date January 2012
End date February 2014
Trial size 30 participants
Trial identifier NCT01511978, JPC 3,4-DAPPER


Hypothesis: 3,4-DAP improves Lambert-Eaton Myasthenic Syndrome (LEMS)-related weakness.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
(Active Comparator)
Half of the subjects will be randomized to continue taking their usual regimen of 3,4-DAP
3,4-dap 3,4-Diaminopyridine
Subjects will maintain their usual dosage using tablets on their regular personal schedule
(Placebo Comparator)
Half of the subjects will be randomized to gradually taper their dose of 3,4-DAP down to placebo
3,4-dap taper to placebo 3,4-Diaminopyridine
Subjects will take decreasing amounts of 3,4-DAP using tablets on their regular personal schedule

Primary Outcomes

Triple Timed Up & Go (TUG) Test
time frame: Participants will be followed for up to 7 days

Secondary Outcomes

Self-assessment of LEMS-related weakness
time frame: Participants will be followed for up to 7 days

Eligibility Criteria

Male or female participants at least 18 years old.

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

Additional Information

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
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.
Trial information was received from ClinicalTrials.gov and was last updated in July 2014.
Information provided to ClinicalTrials.gov by Jacobus Pharmaceutical.