65. 原発性免疫不全症候群 Primary immunodeficiency Clinical trials / Disease details
臨床試験数 : 482 / 薬物数 : 653 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 212
| 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 | NCT04513002 (ClinicalTrials.gov) | February 2022 | 26/7/2020 | Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis | A Phase 2A/2B Placebo-controlled Randomised Clinical Trial to Test the Ability of Triheptanoin to Protect Primary Airway Epithelial Cells Obtained From Participants With Ataxia-telangiectasia Against Death Induced by Glucose Deprivation | Ataxia Telangiectasia | Dietary Supplement: Triheptanoin | The University of Queensland | National Health and Medical Research Council, Australia | Not yet recruiting | N/A | N/A | All | 30 | Phase 2 | Australia |
| 2 | NCT04275479 (ClinicalTrials.gov) | January 10, 2020 | 10/2/2020 | Diabetes/ Endocrine Surveillance in SDS | Endocrine Diabetes Screening in Patients With Shwachman-Diamond Syndrome DIABETES/ ENDOCRINE SURVEILLANCE IN SDS | Shwachman-Diamond Syndrome | Diagnostic Test: Oral Glucose Tolerance Test;Other: Modified Oral Glucose Tolerance Test;Other: Modified Mixed Meal Tolerance Test;Device: Continuous Glucose Monitor;Other: Food Diary;Other: Medical History Questionnaires | Washington University School of Medicine | Shwachman Diamond Syndrome Foundation;Barnes-Jewish Hospital | Recruiting | 3 Years | N/A | All | 60 | United States | |
| 3 | EUCTR2018-004191-35-BE (EUCTR) | 12/04/2019 | 05/03/2019 | Use of an antidiabetic drug (Empagliflozin) to lower the blood level of 1,5-anhydroglucitol in patients deficient in the glucose-6-phosphate transporter (GSD1b) and the phosphatase G6PC3, both of the endoplasmic reticulum, to treat their recurrent infections by normalizing their blood neutrophil counts. Neutrophils are the most abundant white blood cells in our blood that are essential to help fighting infections. 1,5-anhydroglucitol is a sugar derivative with no known function. | Evaluation of the safety and efficacy of administration of Empagliflozin in a new treatment for neutropenia in patients with Glycogen Storage Disease type 1b (GSD1b) and G6PC3 deficiency. - GLYCO-1B | (1) Severe Congenital Neutropenia type 4 (SNC4) due to a deficiency in G6PC3, a phosphatase of the endoplasmic also known a Ubiquitous glucose-6-phosphatase and (2) the neutropenia in Glycogen Storage Disease type 1b due to a deficiency in the glucose-6-phosphate transporter (G6PT / SLC37A4) of the endoplasmic reticulum.;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Jardiance Product Name: Empagliflozin Product Code: A10BK03 | Cliniques universitaires Saint-Luc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 4 | Belgium | ||
| 4 | NCT00220766 (ClinicalTrials.gov) | August 2002 | 13/9/2005 | Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients | IGIV-C 10% Rapid Infusion Trial in Primary Immune Deficient Patients | Immunologic Deficiency Syndrome;Agammaglobulinemia;Severe Combined Immunodeficiency;Wiskott-Aldrich Syndrome;Common Variable Immunodeficiency | Drug: Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified;Drug: Dextrose, 5% in Water | Grifols Therapeutics Inc. | NULL | Completed | 18 Years | 75 Years | Both | 100 | Phase 3 | United States;Canada |