Skin biopsy ( 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 | 1 | 
| 84 | サルコイドーシス | 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 | 
84. サルコイドーシス
臨床試験数 : 149 / 薬物数 : 227 - (DrugBank : 81) / 標的遺伝子数 : 82 - 標的パスウェイ数 : 167
| 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 | NCT02134717 (ClinicalTrials.gov)  | January 2014 | 30/4/2014 | Impact of Chemokine Receptor 5 (CCR5) Inhibition on Sarcoidosis Immunophenotypes | An Interventional Study of the Effect of CCR5 Inhibition With Maraviroc on Immune Cells in the the Lung and in Peripheral Blood of Patients With Sarcoidosis | Sarcoidosis | Drug: all subjects will receive maraviroc 300mg orally twice a day for 6 weeks;Procedure: Bronchoscopy with bronchoalveolar lavage;Procedure: venipunctures;Procedure: Skin biopsy | Kevin F. Gibson | University of Pittsburgh | Terminated | 18 Years | 80 Years | All | 3 | N/A | United States |