- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01765140
Treatment Use of 3,4-Diaminopyridine
Treatment Use of 3,4-Diaminopyridine in Congenital Myasthenic Syndrome
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
CMS diagnoses are made based on clinical, electromyographic and molecular genetic findings, and all patients have been referred to the PI for DAP treatment. This study enrolled minors and adults.
CMS patients under age 18 years were included if their parent or guardian gave written permission. Minors who turned 18 while in the program were re-consented as adults.
The dose of DAP is determined individually for each patient. Adults were started with a dose of 10 mg 3-4 times daily, increased over several weeks to the dose that produced the maximum symptomatic response, not to exceed 100 mg daily. Pyridostigmine bromide (PB) was often added at low doses and increased to the dose that produced the best response, not to exceed 360 mg daily. In children, equivalent doses of these medications was calculated on a surface area basis. The doses of DAP and PB are periodically adjusted to assure that the smallest effective doses are used.
Patients with significant clinical benefit from DAP, as judged by the study PI and the patient, may continue taking DAP as long as the drug is available from the sponsor, and as long as they return for regular follow-up evaluations at the Duke MG Clinic. Patients who are unable to return for regular follow-up will be required to obtain DAP from commercial or other compassionate sources via a physician actively managing their care.
Study Type
Contacts and Locations
Study Locations
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North Carolina
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Durham, North Carolina, United States, 27710
- Duke University Hospital
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Diagnosis of congenital myasthenic syndrome (CMS)
- Women of childbearing potential must have negative pregnancy test and agree to practice adequate contraception while taking DAP
- Must be competent to give consent
Exclusion Criteria:
- Known seizure disorder
- Pregnancy
- Known cardiac arrhythmia or evidence of significant arrhythmia on screening ECG
- Known hepatic, renal or hematologic disease
Study Plan
How is the study designed?
Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: Vern C. Juel, M.D., Duke University
Publications and helpful links
Study record dates
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
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
- Pro00007811
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.
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