NCT01765140

Brief Summary

This protocol provided 3,4 diaminopyridine (DAP) under a treatment-use IND to patients with congenital myasthenic syndrome (CMS). It is now closed.

Trial Health

55
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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 6, 2013

Completed
4 days until next milestone

First Posted

Study publicly available on registry

January 10, 2013

Completed
Last Updated

January 20, 2025

Status Verified

January 1, 2025

First QC Date

January 6, 2013

Last Update Submit

January 17, 2025

Conditions

Myasthenic Syndromes, Congenital

Keywords

3,4 diaminopyridine (DAP)

Interventions

3,4-diaminopyridineDRUG

Treatment use of 3,4-DAP for patients with congenital myasthenic syndrome (CMS)

Also known as: DAP

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • Known seizure disorder
  • Pregnancy
  • Known cardiac arrhythmia or evidence of significant arrhythmia on screening ECG
  • Known hepatic, renal or hematologic disease

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Duke University Hospital

Durham, North Carolina, 27710, United States

Location

Related Publications (1)

  • Sanders DB, Juel VC, Harati Y, Smith AG, Peltier AC, Marburger T, Lou JS, Pascuzzi RM, Richman DP, Xie T, Demmel V, Jacobus LR, Ales KL, Jacobus DP; Dapper Study Team. 3,4-diaminopyridine base effectively treats the weakness of Lambert-Eaton myasthenia. Muscle Nerve. 2018 Apr;57(4):561-568. doi: 10.1002/mus.26052. Epub 2018 Feb 2.

    PMID: 29280483BACKGROUND

MeSH Terms

Conditions

Myasthenic Syndromes, Congenital

Interventions

Amifampridine

Condition Hierarchy (Ancestors)

Neuromuscular Junction DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

4-AminopyridineAminopyridinesAminesOrganic ChemicalsPyridinesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Study Officials

  • Vern C. Juel, M.D.

    Duke University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
expanded access
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Professor of Neurology

Study Record Dates

First Submitted

January 6, 2013

First Posted

January 10, 2013

Last Updated

January 20, 2025

Record last verified: 2025-01

Locations

US(1)