NCT01511978

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

Inpatient Double-Blind Placebo-Controlled Withdrawal Study of 3,4-Diaminopyridine Base (3,4-DAP) in Subjects With Known Lambert-Eaton Myasthenic Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: Jacobus Pharmaceutical · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Lambert-Eaton Myasthenic Syndrome
• Eaton-Lambert Myasthenic Syndrome

Interventions

Type	Name	Description
DRUG	Continuous 3,4-DAP	Subjects were maintained on their usual personal dose and schedule of 3,4-DAP base
DRUG	Taper 3,4-DAP to Placebo	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

Timeline

• Start date: 2012-01-01
• Primary completion: 2014-02-01
• Completion: 2015-07-01
• First posted: 2012-01-19
• Last updated: 2017-07-11
• Results posted: 2017-07-11

Locations

7 sites across 1 country: United States

Regulatory

• FDA-regulated drug study