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Randomized Study of 3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome

This study has been completed.
Study NCT00004832.   Last updated on June 23, 2005.   Information provided by FDA Office of Orphan Products Development

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Descriptive Information Fields
Brief Title  Randomized Study of 3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome
Official Title 
Brief Summary

OBJECTIVES: I. Evaluate the safety and effectiveness of 3,4-diaminopyridine (DAP) in the treatment of patients with Lambert-Eaton myasthenic syndrome (LEMS).

II. Determine the side-effects and benefits associated with DAP.

Detailed Description

PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled study. Patients are randomized to receive 3,4-diaminopyridine (DAP) or placebo orally 3 times daily for 5 days, after which treatment is discontinued and patients are observed for at least 24 hours. At the end of the blinded study, patients may then elect to take open label DAP orally 3 times daily for 6 months; those who do so are monitored for clinical effects and side effects for at least 6 months.

Study Phase
Study Type  Interventional
Study Design  Treatment, Randomized, Double-Blind, Placebo Control
Primary Outcome Measure 
Secondary Outcome Measure 
Condition  Lambert-Eaton Myasthenic Syndrome
Intervention  Drug: 3,4-diaminopyridine
MEDLINE PMIDs
Links
Recruitment Information Fields
Recruitment Status  Completed
Enrollment  26
Start Date  August 1994
Completion Date June 1998
Eligibility Criteria 

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Lambert-Eaton myasthenic syndrome (LEMS) based on weakness that predominates in proximal limb muscles and electromyography (EMG) findings of small amplitude muscle responses to nerve stimulation, which decrease further during nerve stimulation at 5 Hz and which increase at least 2-fold after maximum voluntary contraction of the muscle for 10-20 seconds Quantified Myasthenia Gravis (QMG) clinical score at least 5 --Prior/Concurrent Therapy-- Chemotherapy: No concurrent chemotherapy Endocrine therapy: Patients receiving immunosuppressants must be on the same dose of medication for at least 3 months prior to study entry Radiotherapy: No concurrent radiotherapy Surgery: No concurrent surgery Other: Patients receiving cholinesterase inhibitors must discontinue the medication at study entry if possible, or else be on the same dose of medication for at least 1 month prior to study entry --Patient Characteristics-- Hematopoietic: No significant hematologic disease Hepatic: No significant hepatic disease Renal: No significant renal disease Cardiovascular: No cardiac arrhythmia or significant cardiac disease Neurologic: No seizure disorder Other: Not pregnant Negative pregnancy test required of fertile women Effective contraception required of fertile women

Gender Both
Ages 18 Years and older
Accepts Healthy Volunteers No
Contacts ††
Location Countries 
Administrative Information Fields
NCT ID  NCT00004832
Organization ID 199/13441
Secondary IDs †† DUMC-577, DUMC-FDR001068
Study Sponsor  FDA Office of Orphan Products Development
Collaborators †† Duke University
Investigators 
Study Chair:     Donald B. Sanders     Duke University    
Information Provided By FDA Office of Orphan Products Development
Verification Date July 1998
First Received Date  February 24, 2000
Last Updated Date June 23, 2005

 †    Required WHO trial registration data element.
††   WHO trial registration data element that is required only if it exists.




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