12. Congenital myasthenic syndrome
5 clinical trials,   7 drugs   (DrugBank: 3 drugs),   5 drug target genes,   12 drug target pathways

Searched query = "Congenital myasthenic syndrome", "End-plate acetylcholine receptor deficiency", "Sodium channel myasthenia", "End-plate acetylcholine esterase deficiency", "Dok-7 myasthenia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03062631
(ClinicalTrials.gov)
February 20, 201721/2/2017Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia and Lambert-Eaton SyndromeTreatment Use of 3,4 Diaminopyridine in Congenital Myasthenia and Lambert-Eaton SyndromeCongenital Myasthenic Syndrome;Lambert-Eaton Myasthenic SyndromeDrug: 3,4-DiaminopyridineRicardo MaselliJacobus PharmaceuticalAvailable3 Months75 YearsAllUnited States
2NCT02189720
(ClinicalTrials.gov)
July 20145/7/2014Expanded Access Study Amifampridine Phosphate in Congenital Myasthenic Syndrome (CMS)An Open-Label, Expanded Access Protocol for Amifampridine Phosphate Treatment in Patients With Congenital Myasthenic Syndrome (CMS)Congenital Myasthenic SyndromeDrug: Amifampridine PhosphateCatalyst Pharmaceuticals, Inc.NULLNo longer available2 YearsN/AAllUnited States
3NCT01765140
(ClinicalTrials.gov)
January 20126/1/2013Treatment Use of 3,4-DiaminopyridineTreatment Use of 3,4-Diaminopyridine in Congenital Myasthenic SyndromeMyasthenic Syndromes, CongenitalDrug: 3,4-diaminopyridineVern C. Juel, M.D.NULLNo longer availableN/AN/AAllUnited States
4NCT01203592
(ClinicalTrials.gov)
September 201015/9/2010Efficacy of Albuterol in the Treatment of Congenital Myasthenic SyndromesEfficacy of Albuterol in the Treatment of Congenital Myasthenic SyndromesCongenital Myasthenic SyndromeDrug: AlbuterolMayo ClinicNULLCompleted2 YearsN/ABoth21Phase 1United States
5NCT00872950
(ClinicalTrials.gov)
June 200130/3/20093,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)Open Label Trial Of 3,4-Diaminopyridine In Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS)Lambert-Eaton Myasthenic Syndrome;Congenital Myasthenic SyndromeDrug: 3,4-DIAMINOPYRIDINELahey ClinicNULLActive, not recruiting18 YearsN/AAll22N/AUnited States