Compassionate Use of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome


About this study

This study is being done to determine the long-term response of patients with Lambert-Eaton Myasthenic Syndrome (LEMS) to the investigational drug [not yet approved by the Food and Drug Administration (FDA)] 3, 4-DAP. 3, 4-DAP increases the communication between the nerve and muscle by increasing the release of an important compound in this process, acetylcholine. This drug has been shown in studies done at Mayo and elsewhere to benefit patients with LEMS as well as some patients with congenital myasthenic syndromes.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Patients with documented LEMS (based on clinical, serologic, and electrodiagnostic criteria) will be asked to participate if they fail to respond to standard therapy with cholinesterase inhibitors. 

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Eric Sorenson, M.D.

Closed for enrollment

Contact information:

Duygu Selcen M.D.

(507) 538-0517

More information


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