Title: Open Label Trial of 3,4 Diaminopyridine in Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS) Study Identifier: LCID:2001-040; 3,4-DAP study Principal Investigator: Jayashri Srinivasan, MD, PhD, FRCP
Sponsor: Lahey Hospital & Medical Center
Purpose: This study is looking at the effectiveness and adverse effects of 3, 4 Diaminopyridine (3, 4 DAP) for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS).
Summary: Options for treating Lambert-Eaton Myasthenic Syndrome (LEMS) are limited. Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare neuromuscular condition characterized by muscle weakness, hyporeflexia (abnormal or absent reflexes), and autonomic (involuntary functions such as digestion, urination, respiration) dysfunction. It is usually associated with another disease (most often small cell lung cancer) but may also be the primary condition. The drug, 3,4-Diaminopyridine (3,4-DAP) has been demonstrated to be effective in treating weakness associated with LEMS. This study is an open-label study (meaning all study participants) receive 3,4-DAP.Study Type: Investigational Drug - 3,4-Diaminopyridine (3,4-DAP)Length/Duration: The study consists of monthly clinic visits for the first 4 months. After 4 months, clinic visits will occur every 6 months. Treatment will be continued indefinitely if a good clinical response is achieved and side effects are tolerated.ELIGIBILITY: If you are interested in this study and would like to see if you may qualify, please complete a brief eligibility form.
Contact: For more information on this study, please contact the study coordinator at Jordan.Jara@lahey.org or (781)744-2958.Clinical Trials.gov Identifier: NCT00872950