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.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03062631 (ClinicalTrials.gov) | February 20, 2017 | 21/2/2017 | 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 | Available | 3 Months | 75 Years | All | United States | ||
| 2 | NCT02189720 (ClinicalTrials.gov) | July 2014 | 5/7/2014 | 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. | NULL | No longer available | 2 Years | N/A | All | United States | ||
| 3 | NCT01765140 (ClinicalTrials.gov) | January 2012 | 6/1/2013 | Treatment Use of 3,4-Diaminopyridine | Treatment Use of 3,4-Diaminopyridine in Congenital Myasthenic Syndrome | Myasthenic Syndromes, Congenital | Drug: 3,4-diaminopyridine | Vern C. Juel, M.D. | NULL | No longer available | N/A | N/A | All | United States | ||
| 4 | NCT01203592 (ClinicalTrials.gov) | September 2010 | 15/9/2010 | 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 | NULL | Completed | 2 Years | N/A | Both | 21 | Phase 1 | United States |
| 5 | NCT00872950 (ClinicalTrials.gov) | June 2001 | 30/3/2009 | 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 | Lahey Clinic | NULL | Active, not recruiting | 18 Years | N/A | All | 22 | N/A | United States |