111. 先天性ミオパチー Congenital myopathy Clinical trials / Disease details
臨床試験数 : 10 / 薬物数 : 17 - (DrugBank : 5) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 9
| 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 | NCT04915846 (ClinicalTrials.gov) | December 18, 2020 | 6/5/2021 | Tamoxifen Therapy for Myotubular Myopathy | TAM4MTM: A Phase 1/2 Randomized, Placebo-Controlled, Double-Blinded, Single Crossover Study to Determine the Safety and Efficacy of Tamoxifen Therapy for Myotubular Myopathy (XLMTM) | X Linked Myotubular Myopathy | Drug: ApoTamox 10mg;Drug: Placebo | James Dowling | Canadian Institutes of Health Research (CIHR);Cures Within Reach;The Joshua Frase Foundation USA;Will Cure USA;Mogford Campbell Family Chair Fund;Myotubular Trust;Great Ormond Street Hospital Charity;Sparks | Recruiting | 2 Years | N/A | Male | 16 | Phase 1/Phase 2 | United States;Canada;United Kingdom |