Echocardiogram ( DrugBank: - )
2 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 19 | ライソゾーム病 | 2 |
| 215 | ファロー四徴症 | 1 |
19. ライソゾーム病
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
| 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 | NCT03952637 (ClinicalTrials.gov) | August 19, 2019 | 15/5/2019 | A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis | A Phase 1-2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis | Lysosomal Diseases;Gangliosidosis;GM1 | Biological: AAV9-GLB1;Drug: Rituximab;Drug: Sirolimus;Drug: Methylprednisolone;Drug: Prednisone;Diagnostic Test: Audiology assessmentwith ABR;Diagnostic Test: Bone density scan (DEXA;Diagnostic Test: Electrocardiogram (EKG);Diagnostic Test: Echocardiogram;Other: Electroencephalogram (EEG) awake andextended overnight;Diagnostic Test: Laboratory tests;Procedure: Lumbar puncture;Procedure: Brain MRI/MRS/fMRI;Behavioral: Neurocognitive testing;Other: Neurology exam;Behavioral: PICC line placement;Procedure: Skeletal survey;Procedure: Skin biopsy;Procedure: Speech and modified barium swallow study;Procedure: Ophthalmology exam | National Human Genome Research Institute (NHGRI) | Axovant Sciences, Inc.;Sio Gene Therapies (Sponsor) | Recruiting | 6 Months | 12 Months | All | 45 | Phase 1/Phase 2 | United States |
| 2 | NCT04002531 (ClinicalTrials.gov) | November 10, 2018 | 25/3/2019 | A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children | A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children | Quality of Life;Renal Insufficiency;Cardiac Event | Other: General and Neurological examination;Other: Vital signs;Procedure: 12 lead electrocardiogram;Procedure: Echocardiogram;Procedure: Blood draw;Procedure: Urine collection;Procedure: 2-hour Holter Monitor;Other: Brief Pain Inventory questionnaire;Other: Quality of Life questionnaire | Baylor Research Institute | Shire | Enrolling by invitation | 18 Years | N/A | All | 12 | N/A | United States |
215. ファロー四徴症
臨床試験数 : 22 / 薬物数 : 48 - (DrugBank : 19) / 標的遺伝子数 : 18 - 標的パスウェイ数 : 49
| 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 | NCT04713657 (ClinicalTrials.gov) | March 30, 2022 | 13/1/2021 | Beta-blocker Administration for Cardiomyocyte Division | Mechanistic Clinical Trial of Beta-Blocker Administration For Reactivating Cardiomyocyte Division In Tetrology of Fallot | Tetralogy of Fallot | Other: N-thymidine;Other: Urine Collection;Procedure: Echocardiogram;Procedure: Cardiac MRI;Drug: Propranolol Hydrochloride;Drug: Placebo;Procedure: Physical Exam;Other: Specimen Collection | Bernhard Kuhn | University of Pittsburgh | Not yet recruiting | N/A | 2 Months | All | 40 | Phase 1 | United States |