Conditions: Lambert-Eaton Myasthenic Syndrome Eaton-Lambert Myasthenic Syndrome
Keywords: Lambert-Eaton Eaton-Lambert myasthenia myasthenic LEMS LES DAP diaminopyridine 3,4-diaminopyridine 3,4-DAP
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 32
Healthy volunteers: no

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 withdrawal study in subjects with known clinically active LEMS who had been on a chronic stable dose of compassionate distribution Jacobus 3,4-DAP provided through FDA-approved individual investigator-held INDs.

Arms:
arm 1: Subjects continued taking their usual individualized regimen of 3,4-DAP base, 30 to 100 mg daily divided into at least 3 doses. arm 2: Subjects were tapered over 3 days from their usual individualized regimen of 3,4-DAP base (30 to 100 mg daily divided into at least 3 doses) to placebo with up to an additional 16 hours of placebo before resuming their usual pre-study regimen of 3,4-DAP base

Interventions: intervention 1: Continuous 3,4-DAP intervention 2: Taper 3,4-DAP to Placebo
Intervention descriptions: intervention 1: Subjects were maintained on their usual personal dose and schedule of 3,4-DAP base intervention 2: Subjects were tapered over 3 days from their usual regimen of 3,4-DAP base to placebo with up to an additional 16 hours of placebo before resuming their usual pre-study regimen of 3,4-DAP base

Protocol document: