60. 再生不良性貧血 Aplastic anemia Clinical trials / Disease details
臨床試験数 : 235 / 薬物数 : 381 - (DrugBank : 83) / 標的遺伝子数 : 44 - 標的パスウェイ数 : 160
| 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 | NCT05049668 (ClinicalTrials.gov) | October 2021 | 13/9/2021 | RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial | Long-term Follow-up of Patients Participating in RACE: the Prospective Randomized Multicenter Study Comparing Horse Antithymocyte Globuline (hATG) + Cyclosporine A (CsA) With or Without Eltrombopag as Front-line Therapy for Severe Aplastic Anemia Patients | Severe Aplastic Anemia | Drug: ATGAM plus CsA with or without Eltrombopag | European Society for Blood and Marrow Transplantation | NULL | Enrolling by invitation | N/A | N/A | All | 197 | France | |
| 2 | ChiCTR2100047288 | 2021-06-01 | 2021-06-11 | Efficacy and safety of combined immunosuppressive therapy plus umbilical cord blood infusion in myelodysplastic syndromes/aplastic anemia patients: a clinical observation study | Efficacy and safety of combined immunosuppressive therapy plus umbilical cord blood infusion in myelodysplastic syndromes/aplastic anemia patients: a clinical observation study | myelodysplastic syndromes/aplastic anemi | Experimental group1:According to the classification, ATG + CsA/CSA alone is used as the pretreatment plan/supportive treatment + non-blood cord blood stem cell infusion treatment;Experimental group2:Decitabine+cytarabine/decitabine alone is used as the pretreatment plan/supportive treatment+non-blood cord blood stem cell infusion treatment according to the classification; | The Second Affiliated Hospital of Xi'an Jiaotong University (Xibei Hospital) | NULL | Recruiting | 18 | 90 | Both | Experimental group1:10;Experimental group2:10; | China | |
| 3 | NCT04328727 (ClinicalTrials.gov) | November 4, 2020 | 9/3/2020 | Combination of Eltrombopag With Immunosuppressive Therapy in East-Asian Patients With Severe Aplastic Anemia | A Non-randomized, Open Label, Multi-center, Phase II Study to Assess the Safety and Efficacy of Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin (r-ATG) and Cyclosporine A (CsA) in East-Asian Patients With Treatment Naive Severe Aplastic Anemia (REACTS) | Severe Aplastic Anemia (SAA) | Drug: eltrombopag;Drug: rabbit anti-thymocyte globulin (r-ATG);Drug: cyclosporine A (CsA) | Novartis Pharmaceuticals | NULL | Active, not recruiting | 6 Years | N/A | All | 36 | Phase 2 | China;Japan;Korea, Republic of;Taiwan |
| 4 | JPRN-jRCTs071190032 | 26/11/2019 | 21/10/2019 | W-JHS AA02 | Investigation on the effectiveness of rabbit ATG + cyclosporine + eltrombopag therapy for patients with aplastic anemia - W-JHS AA02 | aplastic anemia | 1. The administration of rATG; 2.5 mg/kg, iv. daily, day 1 - day 5 + CsA; 5 mg/kg, po. bid (before breakfast and before dinner) +adrenalcorticosteroid (the dose is mentioned after) is started. CsA used is Neoral or generic drug emulsified in the same way with Neoral. A blood level of CsA is measured and the dose which CsA blood level 2 hours after oral administration (C2) reachs 600 - 900 ng/mL is adjusted. The investigator shall reduce the dose of CsA by 25% on this occasion when blood trough level (C0) just before the administration is beyond 250 ng/mL because renal function disorder may occur (2). When serum creatinine level also becomes higher than 150% of baselines, the 25% dose reduction of CsA shall be performed. If C2 did not reach to 600 ng/mL, then dose of CsA is appropriately increased. 2. EPAG; 75 mg, po. daily (before sleep, requires to pass more than at least 2 hours after dinner) is started from day 6. 3. The dose of steroid is as follows: Day 1 - day 5: methylprednisolone 2 mg/kg/day Day 6: Methylprednisolone 1 mg/kg/day Day 8, 10, 12, 14, 16, 18, 20: prednisolone 0.5 mg/kg/day Discontinuation after day 21 4. The administration of CsA and EPAG is continued for 26 weeks. When it passed 26 weeks, further treatment mentioned above is entrusted to the investigator in each medical institution after 27 weeks if patients reached Camitta criteria CR or PR. However, the administration is continued for 52 weeks even if the dose of CsA is reduced. The treatment after 53 weeks is not specified. If patients did not reach CR or PR at 26 weeks, treatment after 27 weeks is entrusted to the investigator in each medical institution (it is not specified in this study). | Ishiyama Ken | NULL | Recruiting | >= 18age old | < 80age old | Both | 60 | Phase 2 | Japan |
| 5 | JPRN-UMIN000030453 | 2017/12/20 | 18/12/2017 | Efficacy of cyclosporine therapy in the treatment of non-severe aplastic anemia | Efficacy of cyclosporine therapy in the treatment of non-severe aplastic anemia - Aplastic anemia 01 study in West Japan Hematology Study Group (W-JHS AA01 study) | aplastic anemia | 1.Start CsA (Neoral or generic drugs emulsified in the same way as Neoral) at a dose of 3.5 mg/kg, bid (before breakfast and before dinner). 2.Measure the blood concentration of cyclosporine at 2 hours (C2) after taking the drug and determine the minimum dose of cyclosporine that produces C2 >600 ng/mL. Continue the dose of cyclosporine for 8 weeks. 3.Continue the treatment for further 44 weeks (a total of 52 weeks) when patients meet the response criteria of HI-E or HI-P at the end of the 8 week treatment. Treatments after the 52 weeks are not specified. When patients do not meet the IWG response criteria 2006 at the end of 8 weeks, the treatment is terminated and treatments after 9 weeks are not specified. | Cooperative study between the West Japan Hematology Study Group and Clinical Research Support Center Kyushu | NULL | Complete: follow-up continuing | 16years-old | Not applicable | Male and Female | 33 | Not selected | Japan |
| 6 | NCT03025698 (ClinicalTrials.gov) | September 30, 2017 | 12/1/2017 | A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia | A Phase II, Open-label, Non-controlled, Intra-patient Dose-escalation Study to Characterize the Pharmacokinetics After Oral Administration of Eltrombopag in Pediatric Patients With Refractory, Relapsed or Treatment Naive Severe Aplastic Anemia or Recurrent Aplastic Anemia | Aplastic Anemia | Drug: Eltrombopag;Drug: hATG;Drug: CsA | Novartis Pharmaceuticals | NULL | Active, not recruiting | 1 Year | 18 Years | All | 51 | Phase 2 | United States;Hong Kong;Portugal;Russian Federation;Thailand;United Kingdom;Netherlands |
| 7 | NCT02838992 (ClinicalTrials.gov) | February 2017 | 6/7/2016 | ATG Combined With Cyclophosphamide And Cord Blood Transfusion in Treating Patients With Severe Aplastic Anemia | Multi-center Clinical Study of Immunosuppressants, Cyclophosphamide, And Cord Blood Transfusion in Treating Patients With Severe Aplastic Anemia | Aplastic Anemia | Drug: Rabbit ATG, (Genzyme);Drug: Cy;Drug: CsA;Biological: Cord blood | Jinan Military General Hospital | Shandong University of Traditional Chinese Medicine;Jining Medical University;Affiliated Hospital of Weifang Medical University;Guangzhou First People's Hospital;Harbin Hematology and Oncology Institute;JINING No.1 People's Hospital;JIANGXI Provincal People's Hospital;Jinhua Central Hospital;Linyi People's Hospital;Shandong Cord Blood Bank;Qingdao Center Medical Group;Qingdao University;Taian City Central Hospital;Yantai Yuhuangding Hospital;Yishui Central Hospital of LINYI;Institute of Hematology & Blood Diseases Hospital;Shengjing Hospital | Not yet recruiting | 1 Year | 60 Years | All | 130 | Phase 4 | China |
| 8 | NCT03413306 (ClinicalTrials.gov) | December 10, 2016 | 22/12/2017 | Eltrombopag+hATG+CsA vs. hATG+CsA in Children With Severe AA | A Phase II Multicenter Randomized Study of Eltrombopag Combined With Cyclosporine and hATG Versus hATG and CsA as First Line Treatment in Pediatric Patients With Severe Acquired Aplastic Anemia | Acquired Aplastic Anemia | Drug: Eltrombopag;Drug: IST (ATG + CsA) | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | NULL | Recruiting | 2 Years | 18 Years | All | 100 | Phase 3 | Russian Federation |
| 9 | EUCTR2014-000363-40-NL (EUCTR) | 07/01/2016 | 01/09/2015 | A trial in which will be studied if the addition of a medicine that is used for improving platelet counts (eltrombopag) to the regular treatment for aplastic anemia (hATG + CsA) also improves the numbers of other cell lines and therefore the overall bone marrow function. | A prospective Randomized multicenter study comparing horse Antithymocyte globuline (hATG) + Cyclosporine A (CsA) with or without Eltrombopag as front-line therapy for severe aplastic anemia patients. - RACE | Idiopathic (Severe) Aplastic Anemia, a bone marrow failure syndrome which results in the normal hematopoietic tissue completely missing from the bone marrow, accounting for the subsequent pancytopenia. MedDRA version: 19.0;Level: PT;Classification code 10002967;Term: Aplastic anaemia;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: REVOLADE INN or Proposed INN: ELTROMBOPAG | European Society for Blood and Marrow Transplantation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | Spain;Netherlands | ||
| 10 | EUCTR2014-000363-40-ES (EUCTR) | 26/10/2015 | 07/09/2015 | A trial in which will be studied if the addition of a medicine that is used for improving platelet counts (eltrombopag) to the regular treatment for aplastic anemia (hATG + CsA) also improves the numbers of other cell lines and therefore the overall bone marrow function. | A prospective Randomized multicenter study comparing horse Antithymocyte globuline (hATG) + Cyclosporine A (CsA) with or without Eltrombopag as front-line therapy for severe aplastic anemia patients. - RACE | Idiopathic (Severe) Aplastic Anemia, a bone marrow failure syndrome which results in the normal hematopoietic tissue completely missing from the bone marrow, accounting for the subsequent pancytopenia. MedDRA version: 18.0;Level: PT;Classification code 10002967;Term: Aplastic anaemia;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Revolade INN or Proposed INN: ELTROMBOPAG | European Society for Blood and Marrow Transplantation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Spain | |||
| 11 | NCT02099747 (ClinicalTrials.gov) | July 2015 | 6/3/2014 | hATG+CsA vs hATG+CsA+Eltrombopag for SAA | A Prospective Randomized Multicenter Study Comparing Horse Antithymocyte Globuline (hATG) + Cyclosporine A (CsA) With or Without Eltrombopag as Front-line Therapy for Severe Aplastic Anemia Patients. | Severe Aplastic Anemia | Drug: hATG;Drug: CsA;Drug: Eltrombopag | European Group for Blood and Marrow Transplantation | Novartis;Pfizer | Active, not recruiting | 15 Years | N/A | All | 202 | Phase 3 | France;Italy;Netherlands;Spain;Switzerland;United Kingdom;Germany |
| 12 | NCT02404025 (ClinicalTrials.gov) | May 12, 2015 | 26/2/2015 | Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A in Naive Aplastic Anemia (AA) Subjects | A Non-randomized, Phase II Study of Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A (ATG/CsA) in Subjects With Moderate or More Severe Aplastic Anemia Who Have Not Received Prior ATG/Anti-lymphocyte Globulin (ALG)-Based Immunosuppressive Therapy | Aplastic Anemia | Drug: Eltrombopag;Drug: Rabbit ATG;Drug: CsA | Novartis Pharmaceuticals | NULL | Completed | 18 Years | 75 Years | All | 10 | Phase 2 | Japan |
| 13 | NCT02773225 (ClinicalTrials.gov) | January 27, 2015 | 29/2/2016 | Efficacy and Safety of Eltrombopag + CSA in Patients With Moderate Aplastic Anemia (EMAA) | Efficacy and Safety of Thrombopoetin-Receptor Agonist Eltrombopag in in Combination With Ciclosporin A in Moderate Aplastic Anemia (EMAA): Prospective Randomized Multicenter Study | Anemia, Aplastic | Drug: Eltrombopag;Drug: Placebo (for Eltrombopag) | B. Höchsmann | NULL | Recruiting | 18 Years | N/A | All | 116 | Phase 2/Phase 3 | Germany |
| 14 | NCT01623167 (ClinicalTrials.gov) | July 2, 2012 | 14/6/2012 | Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia | Eltrombopag Added to Standard Immunosuppression in Treatment-Naive Severe Aplastic Anemia | Severe Aplastic Anemia | Drug: Cohort 1: hATG, CsA, EPAG Day 14 to Month 6;Drug: Cohort 2: hATG, CsA, EPAG Day 14 to Month 3;Drug: Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6;Drug: Extension Cohort | National Heart, Lung, and Blood Institute (NHLBI) | Novartis | Recruiting | 2 Years | 95 Years | All | 207 | Phase 1/Phase 2 | United States |
| 15 | EUCTR2006-006577-25-SE (EUCTR) | 25/07/2007 | 11/06/2007 | A prospective randomized study comparing rapamune and tacrolimus vs. cyclosporine and methotrexate as immune prophylaxis in allogeneic hematopoietic stem cell transplantation, using HLA-A, -B, -DRB1 identical related or unrelated donors. A Nordic multicenter study. - Rapa + FK in stem cell transplantation | A prospective randomized study comparing rapamune and tacrolimus vs. cyclosporine and methotrexate as immune prophylaxis in allogeneic hematopoietic stem cell transplantation, using HLA-A, -B, -DRB1 identical related or unrelated donors. A Nordic multicenter study. - Rapa + FK in stem cell transplantation | Graft versus host disease prophylaxis in patients receiving stem cell transplantation due to: chronic myeloid leukemia (CML) in 1st or 2nd chronic phase, acute myeloid leukemia (AML) in complete remission, acute lymphoblastic leukemia (ALL) in complete remission, myelodysplastic syndrome, chronic lymphocytic leukemia, lymphoma, non-malignant disorders, severe aplastic anemia, hemoglobinopathies and metabolic disorders MedDRA version: 9.1;Level: LLT;Classification code 10018799;Term: GVHD | Trade Name: Rapamune Product Name: Rapamune INN or Proposed INN: SIROLIMUS Other descriptive name: Rapamycin Trade Name: Prograf Product Name: Prograf INN or Proposed INN: TACROLIMUS Other descriptive name: FK-506 Trade Name: Sandimmun Neoral Product Name: Sandimmun Neoral INN or Proposed INN: CICLOSPORIN Other descriptive name: CsA Trade Name: Methotrexate Product Name: Methotrexate INN or Proposed INN: METHOTREXATE Other descriptive name: MTX | Karolinska Institutet | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Finland;Sweden | |||
| 16 | NCT01231841 (ClinicalTrials.gov) | March 2005 | 29/10/2010 | Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia | Protocol for Prospective Phase II Study of Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) as a First Line Immunosuppressive (IS) Therapy for Severe Aplastic Anemia (sAA) | Aplastic Anemia | Drug: cyclosporine;Biological: anti-thymocyte globulin | The Cleveland Clinic | NULL | Completed | 12 Years | N/A | All | 20 | Phase 2 | United States |
| 17 | NCT00065260 (ClinicalTrials.gov) | July 2003 | 18/7/2003 | Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia | A Randomized Trial of Immunosuppression in Aplastic Anemia Patients With Refractory Pancytopenia or Suboptimal Hematologic Response After h-ATG/CsA Treatment | Aplastic Anemia | Drug: Campath-1H;Drug: r-ATG;Drug: CsA | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Completed | 2 Years | N/A | All | 54 | Phase 2 | United States |
| 18 | NCT00061360 (ClinicalTrials.gov) | June 26, 2003 | 23/5/2003 | Improving Immunosuppressive Treatment for Patients With Severe Aplastic Anemia | A Randomized Trial of a Novel Immunosuppressive Combination of ATG, CsA and Sirolimus (Rapamune) vs a Slow Taper Cyclosporine Regimen in Subjects With Severe Aplastic Anemia | Severe Aplastic Anemia | Drug: ATG+Rapamune+cyclosporine;Drug: ATG+cyclosporine | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Completed | 2 Years | 110 Years | All | 77 | Phase 2 | United States |
| 19 | NCT00005935 (ClinicalTrials.gov) | June 2000 | 6/7/2000 | Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic Anemia | A Randomized Trial for the Treatment of Relapsing Aplastic Anemia With Mycophenolate Mofetil (MMF) and Cyclosporine (CSA) | Aplastic Anemia | Drug: Mycophenolate mofetil;Drug: Cyclosporine | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Completed | N/A | N/A | Both | 130 | Phase 2 | United States |
| 20 | NCT00001964 (ClinicalTrials.gov) | March 17, 2000 | 18/1/2000 | Combination Therapy of Severe Aplastic Anemia | Treatment of Severe Aplastic Anemia With Combined Immunosuppression: Antithymocyte Globulin (ATG) and Cyclosporine A (CSA), and Mycophenolate Mofetil (MMF) | Severe Aplastic Anemia | Drug: Cyclosporine A;Drug: ATG;Drug: MMF | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Completed | 1 Year | 99 Years | All | 104 | Phase 2 | United States |