Treatment of Lambert-Eaton Syndrome With 3,4 DAP

Expanded access is currently available for this treatment.
Verified November 2012 by University of California, Davis
Sponsor:
Collaborator:
Jacobus Pharmaceutical
Information provided by (Responsible Party):
David P. Richman, MD, University of California, Davis
ClinicalTrials.gov Identifier:
NCT00704925
First received: June 23, 2008
Last updated: November 7, 2012
Last verified: November 2012
  Purpose

A new drug called 3,4-Diaminopyridine (3,4-DAP) is currently under investigation for treatment of the symptoms of Lambert-Eaton Myasthenic Syndrome. This is an expanded access trial, which means that although data from this study will be collected and reported to the US Food and Drug Administration (FDA)and the drug manufacturer, this is not a formal study of drug in Lambert-Eaton Syndrome.

If you decide to volunteer, you will be evaluated by a neurologist to determine your eligibility to receive 3, 4-DAP by a review of your medical history, medication regimen (the medications you are taking) and a neurological examination. If you are a female of child-bearing potential, a serum pregnancy test will be done to ensure that you are not pregnant. Once it is determined that this treatment is appropriate for your care, you will begin taking 3, 4 DAP by mouth in slowly increasing doses. Treatment will begin with 5mg three times a day, as clinically needed, and if tolerated. You will be monitored for strength and side effects by routine clinic visits at initial intervals of once a month, increasing to intervals of every 2-3 months as permitted. Blood will be drawn (approximately 1 tablespoon) at every clinic visit to assess your liver/kidney function and blood counts. You will have an EKG (a test to see how your heart is functioning) at your first study visit, after 6 months of taking 3,4 DAP and again every 2 years. Treatment will be continued indefinitely if a good clinical response is achieved. This study is planned to last indefinitely.

You will be required to keep a diary of the dosage taken. During therapy your doctor will instruct you to stop taking 3,4 DAP for a period of time. You will need to report any return of symptoms that occur. If the symptoms return as a result of being off of 3, 4 DAP, you will have demonstrated that the medication is still needed. This will be done once during the study.

The dosage of 3, 4DAP is individually adjusted. The usual range is 10-15 mg, 3-4 times per day for the full effect and will increase by 50% every two weeks to 10-15 mg, three to six times a day, as needed and if tolerated. Dosages above the full effect level will not provide an additional benefit and should not be used. 3, 4 DAP is a convulsant (causes seizures). A total of 100 mg/day is the maximum dosage allowed.


Condition Intervention
Lambert Eaton Myasthenic Syndrome
Drug: 3, 4 DAP

Study Type: Expanded Access     What is Expanded Access?
Official Title: Treatment of Lambert-Eaton Syndrome With 3, 4-Diaminopyridine

Resource links provided by NLM:


Further study details as provided by University of California, Davis:

Study Start Date: January 2000
Estimated Study Completion Date: June 2011
Estimated Primary Completion Date: June 2010 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: 3, 4 DAP
    Patients with clinically-confirmed LEMS will receive 3, 4 DAP by mouth in slowly increasing doses. Treatment will begin with 5 mg three times a day and will increase by 50% every two weeks to 10-15 mg three to six times a day, as clinically needed, and if tolerated.
Detailed Description:

Patients with clinically-confirmed LEMS will receive 3, 4 DAP by mouth in slowly increasing doses. Treatment will begin with 5 mg, three times a day and will increase by 50% every two weeks to 10-15 mg, three to six times a day, as clinically needed, and if tolerated. Patients will be monitored for strength and side effects via routine out-patient clinic visits at initial intervals of 1 month, increasing to intervals of 6 months as permitted. Subjects will be asked to temporarily stop study drug to determine if there is a return of symptoms and need for continued treatment with 3, 4 DAP. Results of treatment and adverse events will be reported to the FDA. Treatment will be continued indefinitely if a good clinical response is achieved and side effects are tolerable.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Be 18 years or older, diagnosed with LEMS
  • If female, have negative pregnancy test and if premenopausal, be willing to practice an effective form of birth control during the study.

Exclusion Criteria:

  • Is known to have a sensitivity to 3, 4 DAP
  • Has a history of past or current seizures
  • Has a history of past or current severe asthma
  • Is believed by the investigator to be unable to comply with the protocol
  • Unable to give informed consent
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00704925

Contacts
Contact: Janelle Butters, RN 916-734-6276 janelle.butters@ucdmc.ucdavis.edu

Locations
United States, California
University of California, Davis
Sacramento, California, United States, 95827
Contact: Janelle Butters, RN    916-734-6276    janelle.butters@ucdmc.ucdavis.edu   
Contact: Molly Lindsay    916-734-6312    molly.lindsay@ucdmc.ucdavis.edu   
Principal Investigator: David Richman, MD         
Sponsors and Collaborators
University of California, Davis
Jacobus Pharmaceutical
Investigators
Principal Investigator: David Richman, MD University of California, Davis
  More Information

No publications provided

Responsible Party: David P. Richman, MD, Principal Investigator, Professor, University of California, Davis
ClinicalTrials.gov Identifier: NCT00704925     History of Changes
Other Study ID Numbers: 200311036
Study First Received: June 23, 2008
Last Updated: November 7, 2012
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by University of California, Davis:
Lambert Eaton Myasthenic Syndrome
3,4 DAP
3,4 Diaminopyridine

Additional relevant MeSH terms:
Syndrome
Lambert-Eaton Myasthenic Syndrome
Paraneoplastic Syndromes, Nervous System
Paraneoplastic Syndromes
Disease
Pathologic Processes
Nervous System Neoplasms
Neoplasms by Site
Neoplasms
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases

ClinicalTrials.gov processed this record on September 16, 2014