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

NCT ID: NCT00004832

Last Updated: 2015-03-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 26 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 1994-08-31
• Study Completion Date: 1998-06-30

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.

Conditions

Lambert-Eaton Myasthenic Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Interventions

3,4-diaminopyridine

Intervention Type: DRUG

Eligibility Criteria

Inclusion 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Duke University

OTHER

Sponsor Role: collaborator

FDA Office of Orphan Products Development

FED

Sponsor Role: lead

Principal Investigators

Donald B. Sanders

Role: STUDY_CHAIR

Duke University

Other Identifiers

DUMC-577

Identifier Type: -

Identifier Source: secondary_id

DUMC-FDR001068

Identifier Type: -

Identifier Source: secondary_id

199/13441

Identifier Type: -

Identifier Source: org_study_id