Back to resultsTreatment of Lambert-Eaton Myasthenic Syndrome (LEMS) With 3, 4 DAP
Primary Purpose
Lambert Eaton Myasthenic Syndrome (LEMS)
Status
No longer available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
3,4-diaminopyridine
Sponsored by
About this trial
This is an expanded access trial for Lambert Eaton Myasthenic Syndrome (LEMS)
Eligibility Criteria
Inclusion Criteria:
- Be diagnosed with LEMS or a type of CMS likely to respond to 3, 4-DAP.
- If female of childbearing age, have negative pregnancy test, and be willing to practice and effective form of birth control during the study.
- Tested and found by ECG not to have a prolonged QTc syndrome.
- Agrees to have a second ECG at the time of peak drug effect. Has understood and signed the Informed Consent.
Exclusion Criteria:
- Is known to have a sensitivity to 3, 4-DAP.
- Has a history of past or current seizures or of severe asthma, or has an epileptiform EEG.
- Is believed by the investigator to be unable to comply with the protocol.
- Is unable to give informed consent.
- No patient will be excluded based on race, ethnicity, gender, or HIV status
Sites / Locations
- Columbia University Medical Center
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01378546
Brief Title
Treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) With 3, 4 DAP
Official Title
Treatment of Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndromes With 3, 4-Diaminopyridine
Study Type
Expanded Access
2. Study Status
Record Verification Date
July 2013
Overall Recruitment Status
No longer available
Study Start Date
May 2005 (undefined)
Primary Completion Date
May 2015 (Anticipated)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Louis H. Weimer, MD
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
Lambert Eaton Myasthenic Syndrome (LEMS) is rare neurological disorder that results in muscle weakness and limited reflex activity. More than half of LEMS cases are associated with a malignancy, usually small cell lung cancer, and tend to progress more quickly than cases not coupled with malignant cells.
3,4diaminopyridine (3,4DAP)is a drug that has been demonstrated to be effective in treating the weakness associated with LEMS as it increases strength and improves autonomic symptoms in LEMS patients. It is not currently approved by the FDA for use in the United States. The investigators plan to use 3,4DAP to treat patients with LEMS here at the Columbia University MDA/ALS Research Center.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Lambert Eaton Myasthenic Syndrome (LEMS)
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
3,4-diaminopyridine
Intervention Description
Treatment will begin with 5mg three times a day or less.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Be diagnosed with LEMS or a type of CMS likely to respond to 3, 4-DAP.
If female of childbearing age, have negative pregnancy test, and be willing to practice and effective form of birth control during the study.
Tested and found by ECG not to have a prolonged QTc syndrome.
Agrees to have a second ECG at the time of peak drug effect. Has understood and signed the Informed Consent.
Exclusion Criteria:
Is known to have a sensitivity to 3, 4-DAP.
Has a history of past or current seizures or of severe asthma, or has an epileptiform EEG.
Is believed by the investigator to be unable to comply with the protocol.
Is unable to give informed consent.
No patient will be excluded based on race, ethnicity, gender, or HIV status
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Louis H Weimer, MD
Organizational Affiliation
Columbia University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) With 3, 4 DAP
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