- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00872950
3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS) (LEMS)
March 24, 2022 updated by: Lahey Clinic
Open Label Trial Of 3,4-Diaminopyridine In Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS)
The purpose of this study is to determine the effectiveness and adverse effects of 3,4-diaminopyridine for the treatment of the Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS).
Study Overview
Status
Approved for marketing
Intervention / Treatment
Detailed Description
This is an open-label, non-randomized, non-comparative expanded access study.
Up to 25 patients with clinically proven paraneoplastic or primary autoimmune LEMS per EMG and positive voltage-gated calcium channel antibody serology, OR patients with clinically proven CMS per electromyogram (EMG), biopsy or genetic testing who meet the selection criteria outlined in sections 3.1 and 3.2 will be enrolled in this study.
Subjects will receive 3,4-diaminopyridine (3,4-DAP) starting with a low dose and titrating up per efficacy and patient tolerance to a maximum daily dose of 100mg .
Treatment will continue until the subject experiences a treatment-limiting toxicity, voluntarily withdraws consent, transfers to another site, dies, becomes lost to follow-up, is no longer receiving clinical benefit from 3,4-DAP (in the opinion of the subject and/or the investigator), or the Jacobus Pharmaceuticals or the FDA discontinues the study.
Study Type
Expanded Access
Expanded Access Type
- Treatment IND/Protocol
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Massachusetts
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Burlington, Massachusetts, United States, 01805
- Lahey Hospital & Medical Center
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- LEMS diagnosis OR Pre or Post Synaptic CMS diagnosis
- 18 years or older
- Females must have negative pregnancy test and be willing to practice an effective form of birth control
- No prolonged QT syndrome as indicated by baseline EKG
Exclusion Criteria:
- Known sensitivity to 3,4-DIAMINOPYRIDINE
- History of seizures and/or severe asthma
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Jayashri Srinivasan, MD,MCRP,PhD, Lahey Clinic
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Registration Dates
First Submitted
March 30, 2009
First Submitted That Met QC Criteria
March 31, 2009
First Posted (Estimate)
April 1, 2009
Study Record Updates
Last Update Posted (Actual)
April 11, 2022
Last Update Submitted That Met QC Criteria
March 24, 2022
Last Verified
March 1, 2022
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Immune System Diseases
- Neoplasms
- Autoimmune Diseases of the Nervous System
- Autoimmune Diseases
- Neoplasms by Site
- Disease
- Genetic Diseases, Inborn
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Nervous System Neoplasms
- Paraneoplastic Syndromes, Nervous System
- Paraneoplastic Syndromes
- Neuromuscular Junction Diseases
- Myasthenia Gravis
- Syndrome
- Lambert-Eaton Myasthenic Syndrome
- Myasthenic Syndromes, Congenital
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Peripheral Nervous System Agents
- Membrane Transport Modulators
- Neuromuscular Agents
- Potassium Channel Blockers
- Amifampridine
Other Study ID Numbers
- 2001-040
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Lambert-Eaton Myasthenic Syndrome
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David P. Richman, MDJacobus PharmaceuticalNo longer available
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University of Colorado, DenverApproved for marketingLambert Eaton Myasthenic SyndromeUnited States
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Jacobus PharmaceuticalCompletedLambert-Eaton Myasthenic Syndrome | Eaton-Lambert Myasthenic SyndromeUnited States
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Catalyst Pharmaceuticals, Inc.CompletedA Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS)Lambert Eaton Myasthenic SyndromeUnited States, Spain, Serbia, Russian Federation, Hungary, France, Germany, Poland
-
Louis H. Weimer, MDNo longer availableLambert Eaton Myasthenic Syndrome (LEMS)United States
-
Oregon Health and Science UniversityJacobus PharmaceuticalNo longer availableLambert-Eaton Myasthenic Syndrome (LEMS) | Congenital Myasthenia (CM)United States
-
argenxRecruitingCongenital Myasthenic SyndromeUnited States, Canada, France
-
Catalyst Pharmaceuticals, Inc.CompletedMyasthenic Syndromes, CongenitalUnited States
-
Ricardo MaselliCatalyst Pharmaceuticals, Inc.No longer availableCongenital Myasthenic SyndromeUnited States
-
Mayo ClinicCompletedCongenital Myasthenic SyndromeUnited States
Clinical Trials on 3,4-DIAMINOPYRIDINE
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Jacobus PharmaceuticalCompletedLambert-Eaton Myasthenic Syndrome | Eaton-Lambert Myasthenic SyndromeUnited States
-
Ricardo MaselliCatalyst Pharmaceuticals, Inc.No longer availableCongenital Myasthenic SyndromeUnited States
-
Catalyst Pharmaceuticals, Inc.No longer availableLambert-Eaton Myasthenic Syndrome | Congenital Myasthenic Syndrome | Nystagmus, AcquiredUnited States
-
Catalyst Pharmaceuticals, Inc.CompletedA Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS)Lambert Eaton Myasthenic SyndromeUnited States, Spain, Serbia, Russian Federation, Hungary, France, Germany, Poland
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National Center for Research Resources (NCRR)Mayo ClinicCompletedPolyradiculoneuropathy, Chronic Inflammatory Demyelinating
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Oregon Health and Science UniversityJacobus PharmaceuticalNo longer availableLambert-Eaton Myasthenic Syndrome (LEMS) | Congenital Myasthenia (CM)United States
-
David Lacomis, MDJacobus PharmaceuticalNo longer availableLambert-Eaton Myasthenic Syndrome
-
Louis H. Weimer, MDNo longer availableLambert Eaton Myasthenic Syndrome (LEMS)United States
-
Centre Hospitalier Universitaire, AmiensCompleted
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FDA Office of Orphan Products DevelopmentDuke UniversityCompletedLambert-Eaton Myasthenic Syndrome