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Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia

NCT03062631 · Status: NO_LONGER_AVAILABLE · Type: EXPANDED_ACCESS

Last updated 2023-06-02

No results posted yet for this study

Summary

Congenital myasthenia is a potentially lethal disorder, which, even with careful management, significantly impedes participation in normal daily functions. Currently approved therapies have had little impact on promoting a normal quality of life activity in these patients. The goal is to systematically examine the effect of 3,4-DAP on the natural course of this disease and to gain additional experience in titrating 3,4-DAP with other available therapies to maximize clinical function and development in this patient population.

The specific aim of this study is to evaluate the use of 3,4 Diaminopyridine (DAP) on selected patients proven by genetic or serum antibody testing to have Congenital Myasthenic Syndrome (CMS), prescribe 3,4 DAP, and then clinically evaluate the response.

Conditions

  • Congenital Myasthenic Syndrome

Interventions

DRUG

3,4-Diaminopyridine

The investigator will prescribe an initial dose of 3,4 diaminopyridine (DAP) by mouth based on the participant's weight. This initial dose may be modified later according to the participant's response to treatment.

Sponsors & Collaborators

Principal Investigators

  • Ricardo Maselli, MD · University of California, Davis

Eligibility

Min Age
3 Months
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03062631 on ClinicalTrials.gov