Jacobus Pharmaceutical of Princeton, N.J., is continuing its placebo-controlled trial of 3,4-diaminopyridine (3,4-DAP) for people with Lambert-Eaton myasthenic syndrome (LEMS) who have been receiving the drug through Jacbobus' "expanded access" (also called "compassionate use") program. The study, which began in January 2012, was originally by invitation only. It now has expanded to include additional participants.
The study is being conducted to confirm the safety and test the efficacy of 3,4-DAP in the treatment of LEMS-related weakness.
LEMS is caused by a misdirected attack of the immune system on nerve endings at the neuromuscular junction, the place where nerve and muscle fibers connect. The disorder causes muscle weakness and sometimes other symptoms, such as dry mouth or constipation.
In the United States, the disease is treated with medications that suppress the immune system and/or medications that slow the breakdown of acetylcholine (cholinesterase inhibitors).
3,4-DAP increases the release of acetylcholine, a chemical messenger that carries signals from nerve cells to muscle fibers.
Expanded 3,4-DAP trial
In this expanded phase 2 trial, participants will be randomly assigned to continue their usual dosage of 3,4-DAP or to gradually reduce (taper) their 3,4-DAP down to zero (a placebo). Neither the investigators nor the participants will know who is taking their usual dose and who is in the "taper to placebo" group until the trial has been completed.
Study sites are in Sacramento, Calif.; Indianapolis; Durham, N.C.; Portland, Ore.; Nashville, Tenn.; Houston; and Salt Lake City.
Each participant will be screened and then, if considered eligible for the trial, asked to spend up to a week in a clinical research center.
Participants will take "timed up and go" functional tests, which measure how long it takes for someone to move from a sitting to a standing position and then walk a short distance; they also will report their own assessment of LEMS-related weakness.
Participants must not:
Contact Kathy Ales, M.D., at Jacobus Pharmaceutical, at (609) 921-7447, ext. 238, or firstname.lastname@example.org; or contact the study site nearest you.
For details, including contact information for each site, see Effectiveness of 3,4-Diaminpyridine in Lambert-Eaton Myasthenic Syndrome (DAPPER); or go to ClinicalTrials.gov and enter NCT01511978 in the search box.
About Clinical Trials
A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.