Use Of 3,4-Diaminopyridine (3,4-DAP) In The Treatment Of Lambert Eaton Myasthenic Syndrome (34-DAP)

Compassionate use of orphan drug 3,4-Diaminopyridine(DAP) in Treatment of Lambert Eaton Myasthenic Syndrome (LEMS). 3,4-DAP is used to decrease the muscle weakness associated with LEMS and hopefully will decrease the need for prednisone and all other therapies that were previously required to control symptoms. How long a patient will take 3,4 DAP depends upon if he/she is seeing benefits from the medication or experiencing side effects that will prevent them from continuation in the study.

Study Overview

• Status: No longer available
• Conditions: Lambert-Eaton Myasthenic Syndrome
• Intervention / Treatment: Drug: 3,4 DAP

Detailed Description

3,4-diaminopyridine (3,4-DAP) decreases symptoms of weakness in patients with LEMS, and therefore can be used to decrease the amount of immune modulation therapy needed to provide an equivalent degree of disease control.

• Study Type: Expanded Access

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Cleveland, Ohio, United States, 44139
      • Cleveland Clinic Foundation

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Clinical diagnosis of LEMS with or without any of the following: evidence of underlying malignancy, presence of P/Q or N-type calcium channel antibodies, electrodiagnostic evidence of a presynaptic defect of neuromuscular junction transmission.None of these laboratory findings are required for inclusion in this study.
2. P/Q and N type calcium channel antibodies are measured in the blood as a routine laboratory test during the course of initial diagnosis, but 10-20% of patients with LEMS do not have elevated levels of these antibodies.

Exclusion Criteria:

1. Hypersensitivity to any component of this medication.
2. History of past or current seizures.
3. History of asthma.
4. Evidence of prolonged QT syndrome. There is no absolute upper limit of normal for the QTc interval.
5. Family history of prolonged QTc syndrome, history of unexplained syncope, seizures or cardiac arrest.

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: The Cleveland Clinic
• Investigators: Principal Investigator: Kerry H Levin, M.D., The Cleveland Clinic

Study record dates

Study Major Dates

• Study Start: September 1, 1997
• Primary Completion (Anticipated): September 1, 2012

Study Registration Dates

• First Submitted: June 13, 2011
• First Submitted That Met QC Criteria: June 13, 2011
• First Posted (Estimate): June 14, 2011

Study Record Updates

• Last Update Posted (Estimate): July 20, 2016
• Last Update Submitted That Met QC Criteria: July 18, 2016
• Last Verified: July 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Neoplasms; Autoimmune Diseases of the Nervous System; Autoimmune Diseases; Neoplasms by Site; Disease; Neuromuscular Diseases; Neurodegenerative Diseases; Nervous System Neoplasms; Paraneoplastic Syndromes, Nervous System; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myasthenia Gravis; Syndrome; Lambert-Eaton Myasthenic Syndrome; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; Membrane Transport Modulators; Neuromuscular Agents; Potassium Channel Blockers; Amifampridine
• Other Study ID Numbers: 102,384