113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details
臨床試験数 : 646 / 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170
| 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 | NCT04120168 (ClinicalTrials.gov) | April 1, 2019 | 7/10/2019 | Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease | Multicenter Non-Drug Screening Study to Determine the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease in Children With Unexplained Transaminase Elevation | Duchenne Muscular Dystrophy;Pompe Disease (Late-onset) | Genetic: Laboratory Tests | Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition | NULL | Completed | 3 Months | 18 Years | All | 590 | Turkey | |
| 2 | JPRN-UMIN000031965 | 2018/12/14 | 30/03/2018 | A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy | A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy - Tranilast-MD | Heart failure patients with muscular dystrophy who showed brain natriuretic peptide (BNP) of 100 pg/mL or more in spite of myocardial protection treatment | Tranilast 300 mg/day is administered three times per day as the study treatment. The start date of study treatment is defined as the medication start date, and 28 weeks of study treatment will be performed (in principle, by outpatient administration). We reconfirm consent for continuation of administration at 28 weeks, and if consent is obtained, further treatment for 116 weeks will be carried out. As long as there are no particular problems, the outpatient visit during the observation period is in 4 weeks (21-35 days), 12 weeks (77-91 days), 20 weeks (134-147 days), 24 weeks (161-175 days), and 28 weeks (190-203 days) after starting medication. A prescription until the next outpatient visit will be issued at the consultation. The outpatient visit after consent reconfirmation is done at 12-weeks intervals after 36 weeks, and the prescription until the next outpatient visit will be issued until the next 144 weeks after starting medication at the consultation. Evaluation of various examination findings including clinical findings, cardiac functions, respiratory functions, motor function, QOL Questionnaire (MDQoL-60, SF-12), and adverse events at the designated timing. In order to confirm TRPV2 inhibitory effect by tranilast and to assess its effectiveness as a biomarker, central laboratory tests for the TRPV2 expression analysis will be performed. | National Hospital Organization Toneyama National Hospital | NULL | Complete: follow-up continuing | 13years-old | Not applicable | Male and Female | 20 | Not selected | Japan |