BioMarin Pharmaceutical of Novato, Calif., is conducting a multicenter study of 3,4-diaminopyridinephosphate (3,4-DAP), also known as amifampridine phosphate, in adults with Lambert-Eaton myasthenic syndrome (LEMS).
There are eight U.S. trial sites, with additional sites planned for France, Germany, Italy, Poland and Spain.
In December 2009, BioMarin received approval to market 3,4-DAP as Firdapsefor LEMS in the European Union.
The company is planning to seek approval from the U.S. Food and Drug Administration (FDA) to market the drug for LEMS in the United States.
3,4-DAP increases the release of acetylcholine, a chemical transmitter of signals from the nervous system that activates muscle fibers.
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).
Prospective participants in the clinical trial must:
U.S. sites are in Birmingham, Ala.; Scottsdale, Ariz.; Little Rock, Ark.; Orange, Calif.; Jacksonville, Fla.; Kansas City, Kan.; New York City; and Philadelphia.
Contact Kenny Jones at BioMarin in California at firstname.lastname@example.org or (415) 506-6700.
For details, see A Phase 3 study of Amifampridine Phosphate in Patients With Lambert-Eaton Myasthenic Syndrome (LEMS); or enter NCT01377922 into the search box at ClinicalTrials.gov.
Access to 3,4-DAP for treatment of LEMS and other conditions via an expanded access program exists at several sites. An expanded access program is a means by which manufacturers make investigational new drugs available under certain circumstances outside a formal clinical trial.
The drug is being supplied by Jacobus Pharmaceutical, based in Princeton, N.J., and possibly other companies, at medical centers in several U.S. cities.
For up-to-date information, go to ClinicalTrials.gov, and enter 3,4-diaminopyridine in the search box.
Editor's note: This story was updated 1/6/12 to reflect the availability of 3,4-diaminopyridine through expanded access programs.