CompletedPhase 2

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

United States32 participantsStarted 2012-01

Plain-language summary

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

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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

Exclusion criteria

✕. 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

What they're measuring

Number of Participants With 30% or More Deterioration in Triple Timed Up & Go (3TUG) Test, Compared to Time-matched Baseline

Timeframe: Baseline period (days 0, 1, 2); Randomized treatment period (starting with last dose of day 2, and days 3, 4, 5, and ending with first dose on day 6 when pre-randomization regimen was resumed, or rescue, if indicated sooner)

Trial details

NCT IDNCT01511978

SponsorJacobus Pharmaceutical

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2014-02

Results submitted2017-06-07

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