12. Congenital myasthenic syndrome
[
4 clinical trials,
5 drugs（DrugBank:
3 drugs),
3 target genes / 11 target pathways ]
Searched query = "Congenital myasthenic syndrome", "End plate acetylcholine receptor deficiency", "Sodium channel myasthenia", "End plate acetylcholine esterase deficiency"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03062631 | February 20, 2017 | 11 June 2018 | Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia and Lambert-Eaton Syndrome | Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia and Lambert-Eaton Syndrome | Congenital Myasthenic Syndrome;Lambert-Eaton Myasthenic Syndrome | Drug: 3,4-Diaminopyridine | Ricardo Maselli | Jacobus Pharmaceutical | Not recruiting | 3 Months | 75 Years | All | Phase 1 | United States | |
| 2 | NCT02189720 | August 2014 | 2 September 2019 | Expanded 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 Syndrome | Drug: Amifampridine Phosphate | Catalyst Pharmaceuticals, Inc. | Not recruiting | 2 Years | N/A | All | Phase 2 | United States | ||
| 3 | NCT01203592 | September 2010 | 18 January 2016 | Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes | Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes | Congenital Myasthenic Syndrome | Drug: Albuterol | Mayo Clinic | Not recruiting | 2 Years | N/A | Both | 21 | Phase 1 | United States | |
| 4 | NCT00872950 | June 2001 | 25 June 2018 | 3,4-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 Syndrome | Drug: 3,4-DIAMINOPYRIDINE;Drug: 3,4-Diaminopyridine | Lahey Clinic | Recruiting | 18 Years | N/A | All | 25 | N/A | United States |