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

NCT00004832 | Completed

• 3 other identifiers: 199/13441DUMC-577DUMC-FDR001068
• Study Type: interventional
• Target: 26
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Lambert-Eaton Myasthenic Syndrome

Keywords

Lambert-Eaton myasthenic syndrome; neurologic and psychiatric disorders; rare disease

Interventions

3,4-diaminopyridine DRUG

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• FDA Office of Orphan Products Development: lead
• Duke University: collaborator

MeSH Terms

Conditions

Lambert-Eaton Myasthenic Syndrome; Neurologic Manifestations; Mental Disorders; Rare Diseases

Interventions

Amifampridine

Condition Hierarchy (Ancestors)

Myasthenia Gravis; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Disease Attributes; Pathologic Processes

Intervention Hierarchy (Ancestors)

4-Aminopyridine; Aminopyridines; Amines; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Donald B. Sanders

  Duke University

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Sponsor Type: FED

Study Record Dates

• First Submitted: February 24, 2000
• First Posted: February 25, 2000
• Study Start: August 1, 1994
• Study Completion: June 1, 1998
• Last Updated: March 25, 2015

Record last verified: 1998-07