Rabbit ( DrugBank: Rabbit )
8 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 11 | 重症筋無力症 | 1 |
| 13 | 多発性硬化症／視神経脊髄炎 | 4 |
| 49 | 全身性エリテマトーデス | 2 |
| 51 | 全身性強皮症 | 3 |
| 60 | 再生不良性貧血 | 25 |
| 65 | 原発性免疫不全症候群 | 3 |
| 96 | クローン病 | 4 |
| 285 | ファンコニ貧血 | 3 |
11. 重症筋無力症
臨床試験数 : 332 / 薬物数 : 234 - (DrugBank : 81) / 標的遺伝子数 : 45 - 標的パスウェイ数 : 127
| 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 | NCT00424489 (ClinicalTrials.gov) | February 2002 | 18/1/2007 | Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis | Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis | Myasthenia Gravis | Biological: Hematopoietic Stem Cell Transplantation;Drug: Cyclophosphamide;Drug: ATG (rabbit);Drug: Mesna;Drug: Methylprednisolone;Drug: G-CSF | Northwestern University | NULL | Terminated | 15 Years | 65 Years | All | 9 | Phase 1 | United States |
13. 多発性硬化症／視神経脊髄炎
臨床試験数 : 3,340 / 薬物数 : 2,163 - (DrugBank : 383) / 標的遺伝子数 : 241 - 標的パスウェイ数 : 238
| 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 | EUCTR2019-001549-42-GB (EUCTR) | 27/03/2020 | 03/02/2020 | Stem cell transplantation versus disease modifying therapy (alemtuzumab or ocrelizumab) for patients with highly active relapsing remitting MS | A multicentre, randomised controlled trial to evaluate the efficacy of autologous haematopoietic stem cell transplantation versus alemtuzumab or ocrelizumab in relapsing remitting multiple sclerosis. - StarMS | Highly active relapsing remitting multiple sclerosis MedDRA version: 20.1;Level: PT;Classification code 10028245;Term: Multiple sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Cyclophosphamide INN or Proposed INN: Cyclophosphamide monohydrate INN or Proposed INN: Cyclophosphamide monohydrate Product Name: Filgrastim INN or Proposed INN: Filgrastim Trade Name: Thymoglobuline Product Name: Thymoglobuline INN or Proposed INN: Rabbit anti-human thymocyte immunoglobulin INN or Proposed INN: Rabbit anti-human thymocyte immunoglobulin INN or Proposed INN: Rabbit anti-human thymocyte immunoglobulin Trade Name: Alemtuzumab Product Name: Alemtuzumab INN or Proposed INN: Alemtuzumab Product Name: Lenograstim INN or Proposed INN: Lenograstim Trade Name: Ocrelizumab Product Name: Ocrelizumab | Sheffield Teaching Hospitals | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 198 | Phase 3 | United Kingdom | ||
| 2 | EUCTR2010-023560-40-SE (EUCTR) | 23/03/2011 | 21/12/2010 | Blood stem cell transplantation for patients with relapsiong-remitting multiple sclerosis, in whom standard treatment has failed. | Haematopoetic Stem Cell Therapy for Patients with Inflammatory Multiple Sclerosis Failing Alternate Approved Therapy: A Randomized Study - MIST | Relapsing-remitting multiple sclerosis MedDRA version: 13.1;Level: PT;Classification code 10063399;Term: Relapsing-remitting multiple sclerosis;System Organ Class: 10029205 - Nervous system disorders | Trade Name: Tysabri INN or Proposed INN: NATALIZUMAB Trade Name: Sendoxan Other descriptive name: CYCLOPHOSPHAMIDE MONOHYDRATE Trade Name: Thymoglobuline Other descriptive name: RABBIT HUMAN T LYMPHOCYTE IMMUNOGLOBULIN Trade Name: Neupogen Trade Name: Solu-Medrol | Uppsala l?ns landsting | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | United States;Canada;Brazil;Sweden | |||
| 3 | EUCTR2006-002562-19-IT (EUCTR) | 21/04/2006 | 24/01/2007 | Immunoablation with Cyclophosphamide at high dosage and Rabbit antithymoglobulin followed by autologous hematopoietic stem cell transplantation in severe multiple sclerosis - LIGHT | Immunoablation with Cyclophosphamide at high dosage and Rabbit antithymoglobulin followed by autologous hematopoietic stem cell transplantation in severe multiple sclerosis - LIGHT | VERIFICARE Patients with multiple sclerosis, age between 18 and 50 MedDRA version: 6.1;Level: PT;Classification code 10028245 | Trade Name: L04AA04 - IMMUNOGLOBULINA ANTITIMOCITARIA (DI CONIGLIO) IMMUNOGLOBULINA DI CONIGLIO ANTITIMOCITI UMANI THYMOGLOBULINE Product Name: Thymoglobuline INN or Proposed INN: Antithymocyte Trade Name: Endoxan - ciclofosfamide Product Name: Endoxan INN or Proposed INN: Cyclophosfamide | Dipartimento di Neuroscienze Oftalmologia e Genetica DINOG - Universita di Genova | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Italy | |||
| 4 | EUCTR2008-005773-35-IT (EUCTR) | 15/01/2013 | AUTOLOUGUS STEM CELLS TRANSPLANTATION AFTER LINFOCYTE DEPLETION AS A NEW THERAPY FOR THE AGGRESSIVE AND DRUG RESISTANT FORMS OF MULTIPLE SLEROSIS IN THE ADULTS AND CHILDREN. - AUTOLOUGUS STEM CELLS TRANSPLANTATION IN SM | AUTOLOUGUS STEM CELLS TRANSPLANTATION AFTER LINFOCYTE DEPLETION AS A NEW THERAPY FOR THE AGGRESSIVE AND DRUG RESISTANT FORMS OF MULTIPLE SLEROSIS IN THE ADULTS AND CHILDREN. - AUTOLOUGUS STEM CELLS TRANSPLANTATION IN SM | MULTIPLE SCLEROSIS MedDRA version: 15.1;Level: LLT;Classification code 10028053;Term: MS;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: ENDOXAN BAXTER INN or Proposed INN: Cyclophosphamide Trade Name: FLUDARA INN or Proposed INN: Fludarabine Trade Name: THYMOGLOBULINE INN or Proposed INN: Antithymocyte immunoglobulin (rabbit) Trade Name: MABTHERA INN or Proposed INN: Rituximab | AZIENDA OSPEDALIERA DI PADOVA | NULL | NA | Female: yes Male: yes | Phase 1 | Italy |
49. 全身性エリテマトーデス
臨床試験数 : 993 / 薬物数 : 702 - (DrugBank : 184) / 標的遺伝子数 : 116 - 標的パスウェイ数 : 200
| 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 | NCT02080195 (ClinicalTrials.gov) | September 13, 2016 | 28/2/2014 | Nonmyeloablative Conditioning and Transplantation for Patients With Refractory Systemic Lupus Erythematosus (SLE) | A Phase I/II Study of Nonmyeloablative Conditioning and Transplantation of Human Leukocyte Antigen (HLA)-Matched, Partially HLA-mismatched, HLA-haploidentical or Matched Unrelated Bone Marrow for Patients With Refractory SLE | Lupus Erythematosus;Graft-versus-host Disease | Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Tacrolimus;Drug: Mycophenolate Mofetil;Drug: Rabbit antithymocyte globulin;Radiation: Total body irradiation;Biological: Allogeneic bone marrow transplant | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Maryland Stem Cell Research Fund | Terminated | 18 Years | 75 Years | All | 1 | Phase 1/Phase 2 | United States |
| 2 | NCT00278538 (ClinicalTrials.gov) | September 23, 2005 | 15/1/2006 | Cyclophosphamide and Rabbit Antithymocyte Globulin (rATG)/Rituximab in Patients With Systemic Lupus Erythematosus | Cyclophosphamide and rATG/Rituximab in Patients With Systemic Lupus Erythematosus: Phase II Trial | Systemic Lupus Erythematosus | Biological: Hematopoietic stem cell transplantation | Northwestern University | NULL | Completed | 15 Years | 60 Years | All | 32 | Phase 2 | United States |
51. 全身性強皮症
臨床試験数 : 525 / 薬物数 : 565 - (DrugBank : 148) / 標的遺伝子数 : 114 - 標的パスウェイ数 : 217
| 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 | EUCTR2006-004598-83-GB (EUCTR) | 22/10/2008 | 24/10/2008 | High dose immunoablation and autologous hematopoietic stem cell transplantation versus monthly intravenous pulse therapy cyclophosphamide in severe systemic sclerosis ( ‘ASTIS’-TRIAL: Autologous Stem Cell Transplantation International Scleroderma Trial) - ASTIS | High dose immunoablation and autologous hematopoietic stem cell transplantation versus monthly intravenous pulse therapy cyclophosphamide in severe systemic sclerosis ( ‘ASTIS’-TRIAL: Autologous Stem Cell Transplantation International Scleroderma Trial) - ASTIS | Severe systemic sclerosis MedDRA version: 9.1;Level: LLT;Classification code 10042953;Term: Systemic sclerosis | Trade Name: Cyclophosphamide Product Name: Cyclophosphamide INN or Proposed INN: CYCLOPHOSPHAMIDE Trade Name: Neupogen Product Name: Filgrastim INN or Proposed INN: FILGRASTIM Trade Name: Thymoglobuline Product Name: Thymoglobuline Product Code: rbATG INN or Proposed INN: Anti-Thymocyte Globulin, Rabbit Other descriptive name: ANTI-HUMAN T-LYMPHOCYTE IMMUNOGLOBULIN FROM RABBITS Trade Name: Solu-Medrone Product Name: Methylprednisolone INN or Proposed INN: METHYLPREDNISOLONE Trade Name: Cyclophosphamide Product Name: Cyclophosphamide INN or Proposed INN: CYCLOPHOSPHAMIDE Trade Name: Mesna Product Name: Mesna INN or Proposed INN: MESNA Trade Name: Mesna Product Name: Mesna INN or Proposed INN: MESNA | EBMT (European group for Blood and Marrow Transplantation) | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 150 | United Kingdom | |||
| 2 | NCT00278525 (ClinicalTrials.gov) | September 2005 | 15/1/2006 | Cyclophosphamide and rATG With Hematopoietic Stem Cell Support in Systemic Scleroderma | Trial of High Dose Cyclophosphamide and Rabbit Antithymocyte Globulin (rATG) With Hematopoietic Stem Cell Support in Patients With Systemic Scleroderma: A Randomized Trial | SYSTEMIC SCLERODERMA | Drug: standard of care;Procedure: stem cell transplantation | Northwestern University | NULL | Completed | N/A | 60 Years | All | 19 | Phase 2 | United States |
| 3 | NCT00016458 (ClinicalTrials.gov) | June 2000 | 6/5/2001 | Phase II Pilot Study of Cyclophosphamide and Rabbit Anti-Thymocyte Globulin as Salvage Therapy in Patients With Diffuse Systemic Sclerosis | Systemic Sclerosis | Drug: anti-thymocyte globulin;Drug: cyclophosphamide | Fred Hutchinson Cancer Research Center | NULL | Completed | N/A | 64 Years | Both | 20 | Phase 2 | United States |
60. 再生不良性貧血
臨床試験数 : 245 / 薬物数 : 318 - (DrugBank : 86) / 標的遺伝子数 : 44 - 標的パスウェイ数 : 166
| 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 | NCT05600426 (ClinicalTrials.gov) | January 25, 2023 | 20/10/2022 | A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202) | A Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation With Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202) | Severe Aplastic Anemia | Drug: cyclosporine;Procedure: Matched Unrelated Donor Hematopoetic Stem Cell Transplant;Drug: horse anti-thymocyte globulin (ATG);Drug: rabbit anti-thymocyte globulin (ATG);Drug: Methotrexate;Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: low-dose total body irradiation (TBI);Procedure: Immunosuppressive Therapy (IST) | Boston Children's Hospital | Center for International Blood and Marrow Transplant Research;National Institutes of Health (NIH);National Heart, Lung, and Blood Institute (NHLBI);North American Pediatric Aplastic Anemia Consortium;Pediatric Transplantation and Cellular Therapy Consortium;Blood and Marrow Transplant Clinical Trials Network | Recruiting | 0 Years | 25 Years | All | 234 | Phase 3 | United States |
| 2 | 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 |
| 3 | 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 |
| 4 | NCT03531736 (ClinicalTrials.gov) | May 9, 2018 | 9/5/2018 | T Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation Conditioned With a Reduced Intensity Regimen in Patients With Hematologic Malignancies and Aplastic Anemia | Allogeneic Hematopoietic Stem Cell Transplantation of a/ß T-Lymphocyte Depleted Graft Conditioned With a Reduced Intensity Regimen in Patients With Hematologic Malignancies and Aplastic Anemia | Myeloid Diseases | Drug: Antithymocyte globulin (Rabbit);Drug: fludarabine;Radiation: total body irradiation;Drug: cyclophosphamide;Drug: Rituxan;Procedure: Allogeneic Hematopoietic Stem Cell Transplantation | Memorial Sloan Kettering Cancer Center | NULL | Recruiting | 18 Years | N/A | All | 15 | Phase 1 | United States |
| 5 | 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 |
| 6 | NCT02828592 (ClinicalTrials.gov) | September 9, 2016 | 6/7/2016 | Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia | A Study of T-Cell Replete, HLA-Mismatched Haploidentical Bone Marrow Transplantation With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Lacking HLA-Matched Related Donor | Severe Aplastic Anemia | Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Drug: Rabbit ATG | Northside Hospital, Inc. | NULL | Recruiting | 1 Year | 75 Years | All | 20 | Phase 2 | United States |
| 7 | NCT02845596 (ClinicalTrials.gov) | August 2016 | 9/5/2016 | Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia | Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia | Severe Aplastic Anemia | Drug: cyclosporine;Procedure: Matched Unrelated Donor Hematopoietic Stem Cell Transplant;Drug: horse anti-thymocyte globulin (ATG);Drug: rabbit anti-thymocyte globulin (ATG);Drug: methotrexate;Drug: fludarabine;Drug: cyclophosphamide;Radiation: low-dose total body irradiation (TBI);Procedure: Immunosuppressive Therapy (IST) | Michael Pulsipher, MD | NULL | Active, not recruiting | N/A | 25 Years | All | 40 | N/A | United States |
| 8 | 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 |
| 9 | NCT02407470 (ClinicalTrials.gov) | January 2015 | 25/3/2015 | Safety and Efficacy of Patient's Own AD-MSC and AD-HSC Transplantation in Patients With Severe Aplastic Anemia | A Multicenter, Randomized, Controlled Study of the Efficacy and Safety of the Combination of Adipose Tissue-derived Hematopoietic Stem Cells (AD-HSCs) and ATG in the Treatment of Severe Aplastic Anemia | Severe Aplastic Anemia | Drug: Rabbit antithymoglobulin (ATG);Procedure: Adipose derived mesenchymal stem cells ( AD-MSCs);Procedure: AD-MSC transdifferentiated HSCs (AD-HSCs) | Navy General Hospital, Beijing | Peking Union Medical College Hospital;General Hospital of Beijing PLA Military Region;Chinese Academy of Medical Sciences | Recruiting | 14 Years | 70 Years | Both | 90 | Phase 1/Phase 2 | China |
| 10 | NCT02203396 (ClinicalTrials.gov) | August 2014 | 27/7/2014 | A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia | A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia | Aplastic Anemia | Drug: rabbit ATG, Cyclosporine, Levamisole | Yizhou Zheng | NULL | Recruiting | 6 Years | 70 Years | Both | 40 | Phase 2 | China |
| 11 | NCT02028416 (ClinicalTrials.gov) | September 2013 | 2/1/2014 | Comparison of Two Different Doses of Rabbit ATG-Fresenius With Cyclosporin in the Treatment of Acquired Aplastic Anaemia | Protocol for Comparison of Two Different Regimens of Rabbit ATG-Fresenius With Cyclosporin in the Treatment of Acquired Aplastic Anaemia | Aplastic Anemia | Drug: ATG-fresenius | National Institute of Blood Disease Center, Pakistan | NULL | Recruiting | 2 Years | 65 Years | Both | 60 | N/A | Pakistan |
| 12 | JPRN-UMIN000011134 | 2012/05/23 | 08/07/2013 | A prospective randomized dose comparison multicenter study of the rabbit anti-thymocyte globulin (thymoglobulin, Genzyme) for patients with severe and very severe aplastic anemia | A prospective randomized dose comparison multicenter study of the rabbit anti-thymocyte globulin (thymoglobulin, Genzyme) for patients with severe and very severe aplastic anemia - APBMT AAWG-01 | Acquired Aplastic Anemia | 2.5 mg/kg/day of Thymoglobulin for 5 days 3.5 mg/kg/day of Thymoglobulin for 5 days | Nagoya University | NULL | Complete: follow-up complete | Not applicable | 69years-old | Male and Female | 320 | Not selected | Japan,Asia(except Japan) |
| 13 | NCT01844635 (ClinicalTrials.gov) | May 2012 | 29/4/2013 | Randomised Study Comparing Different Dosages of Rabbit ATG in Patients With SAA | A Prospective Randomized Multicenter Study Comparing Different Dosages of Rabbit Antithymocyte Globulin (Thymoglobuline) in Patients With Severe Aplastic Anemia | Acquired Aplastic Anemia. | Drug: Thymoglobulin | Nagoya University | NULL | Recruiting | N/A | 69 Years | Both | 320 | Phase 3 | Japan |
| 14 | EUCTR2007-000902-55-DE (EUCTR) | 17/12/2009 | 29/09/2009 | Prospective Phase II Pilot study of Rabbit Antithymocyte globulin(ATG, Thymoglobuline®, Genzyme) with Ciclosporin for Patients withAcquired Aplastic Anaemia and comparison with matched historicalpatients treated with horse ATG and Ciclosporin: a Study from theEuropean Blood and Marrow Transplant (EBMT) Severe AplasticAnaemia Working Party | Prospective Phase II Pilot study of Rabbit Antithymocyte globulin(ATG, Thymoglobuline®, Genzyme) with Ciclosporin for Patients withAcquired Aplastic Anaemia and comparison with matched historicalpatients treated with horse ATG and Ciclosporin: a Study from theEuropean Blood and Marrow Transplant (EBMT) Severe AplasticAnaemia Working Party | Acquired severe aplastic anaemia and transfusion dependent non-severe aplastic anaemia MedDRA version: 12.0;Level: LLT;Classification code 10002969;Term: Aplastic anemia | Trade Name: Thymoglobulin Product Name: Thymoglobuline®/Thymoglobulin® Product Code: anti-thymocyte globulin (rabbit) | EBMT (European Group for Blood and Marrow Transplantation) | NULL | Not Recruiting | Female: yes Male: yes | 35 | Phase 2 | France;Germany;United Kingdom | ||
| 15 | NCT01129323 (ClinicalTrials.gov) | November 2009 | 21/4/2010 | Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia | Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia | Severe Aplastic Anemia | Drug: Cyclophosphamide, Fludarabine, Rabbit ATG | City of Hope Medical Center | NULL | Withdrawn | N/A | 21 Years | Both | 0 | N/A | United States |
| 16 | NCT00944749 (ClinicalTrials.gov) | July 2009 | 22/7/2009 | Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment | Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment | Anemia, Aplastic;Anemia, Hypoplastic | Drug: h-ATG (ATGAM );Drug: Cyclosporine (Gengraf ) | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Completed | 2 Years | 82 Years | All | 23 | Phase 2 | United States |
| 17 | NCT00882323 (ClinicalTrials.gov) | November 2008 | 15/4/2009 | Reduced Toxicity Fludarabine (Flu) + Cyclophosphamide (CPM) + Rabbit Antithymocyte Globulin (rATG) Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia (SAA) | Reduced Toxicity Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia | Aplastic Anemia | Drug: Cyclophosphamide, Fludarabine, Thymoglobulin | The Korean Society of Pediatric Hematology Oncology | NULL | Recruiting | 1 Year | 21 Years | Both | 33 | Phase 2 | Korea, Republic of |
| 18 | NCT00471848 (ClinicalTrials.gov) | August 2008 | 9/5/2007 | Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia | Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia and Comparison With Matched Historical Patients Treated With Horse ATG and Ciclosporin | Aplastic Anemia | Drug: rabbit antithymocyte globulin | European Group for Blood and Marrow Transplantation | Genzyme, a Sanofi Company | Active, not recruiting | 16 Years | N/A | Both | 35 | Phase 2 | France;Germany;Italy;Saudi Arabia;Switzerland;United Kingdom |
| 19 | EUCTR2007-000902-55-FR (EUCTR) | 23/04/2008 | 04/10/2007 | Prospective Phase II Pilot study of Rabbit Antithymocyte globulin (ATG, Thymoglobuline®, Genzyme) with Ciclosporin for Patients with Acquired Aplastic Anaemia and comparison with matched historical patients treated with horse ATG and Ciclosporin | Prospective Phase II Pilot study of Rabbit Antithymocyte globulin (ATG, Thymoglobuline®, Genzyme) with Ciclosporin for Patients with Acquired Aplastic Anaemia and comparison with matched historical patients treated with horse ATG and Ciclosporin | Aquired aplastic anaemia and transfusion dependent non-severe aplastic anaemia MedDRA version: 9.1;Level: LLT;Classification code 10002274;Term: Anemia aplastic | Trade Name: Thymoglobuline Product Name: Thymoglobuline®/Thymoglobulin® Product Code: anti-thymocyte globulin (rabbit) Other descriptive name: GLYCINE Other descriptive name: SODIUM CHLORIDE SOLUTION 0.9% Other descriptive name: Mannitol Trade Name: Neoral Product Name: Ciclosporine INN or Proposed INN: Cyclosporin A Other descriptive name: Ciclosporine | EBMT (European group for Blood and Marrow Transplantation) | NULL | Not Recruiting | Female: yes Male: yes | 35 | Phase 2 | France;Germany;United Kingdom | ||
| 20 | NCT01530555 (ClinicalTrials.gov) | April 2008 | 24/1/2012 | Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia | Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia | Acquired Aplastic Anaemia | Drug: Rabbit ATG, Thymoglobuline (Genzyme) | King Faisal Specialist Hospital & Research Center | NULL | Completed | 16 Years | 80 Years | Both | 35 | Phase 2 | NULL |
| 21 | EUCTR2007-001657-26-GB (EUCTR) | 29/02/2008 | 07/11/2007 | Single Centre Phase II Pilot study of Unrelated Cord Blood Transplantation in Patients with Poor Risk Haematological Malignancies. | Single Centre Phase II Pilot study of Unrelated Cord Blood Transplantation in Patients with Poor Risk Haematological Malignancies. | 1.Acute, chronic leukaemia or myelodysplastic syndrome for which allogeneic transplantation is considered as the best treatment option. 2.Acute lymphoblastic leukaemia (ALL)3.Non-Hodgkin’s lymphoma4.Hodgkin’s disease5.Chronic lymphocytic leukaemia. 6.Acquired bone marrow failure syndromes7.Other haematological malignancies for which UD bone marrow transplantation is indicated MedDRA version: 9.1;Level: LLT;Classification code 10000880;Term: Acute myeloid leukaemia MedDRA version: 9.1;Classification code 10028533;Term: Myelodysplastic syndrome MedDRA version: 9.1;Classification code 10009013;Term: Chronic myeloid leukaemia MedDRA version: 9.1;Classification code 10000844;Term: Acute lymphoblastic leukaemia MedDRA version: 9.1;Classification code 10020328;Term: Hodgkin's lymphoma MedDRA version: 9.1;Classification code 10029593;Term: Non-Hodgkin's lymphoma NOS MedDRA version: 9.1;Classification code 10003892;Term: B-cell chronic lymphocytic leukaemia/prolymphocytic leukaemia/small lymphocytic lymphoma MedDRA version: 9.1;Classification code 10002968;Term: Aplastic anaemia, unspecified | Trade Name: Fludarabine INN or Proposed INN: FLUDARABINE PHOSPHATE Trade Name: Busilvex INN or Proposed INN: BUSULFAN Trade Name: Thymoglobulin Other descriptive name: RABBIT HUMAN T LYMPHOCYTE IMMUNOGLOBULIN Trade Name: Thiotepa INN or Proposed INN: THIOTEPA Trade Name: Cyclophosphamide Other descriptive name: CYCLOPHOSPHAMIDE MONOHYDRATE Trade Name: Alkeran INN or Proposed INN: MELPHALAN | King's College Hospital NHS Foundation Trust | NULL | Not Recruiting | Female: yes Male: yes | 27 | Phase 2 | United Kingdom | ||
| 22 | EUCTR2007-000902-55-GB (EUCTR) | 19/09/2007 | 26/06/2007 | Prospective Phase II study of Rabbit Antithymocyte globulin (ATG, Thymoglobuline®, Genzyme) with Ciclosporin for Patients with Acquired Aplastic Anaemia and comparison with matched historical patients treated with horse ATG and Ciclosporin | Prospective Phase II study of Rabbit Antithymocyte globulin (ATG, Thymoglobuline®, Genzyme) with Ciclosporin for Patients with Acquired Aplastic Anaemia and comparison with matched historical patients treated with horse ATG and Ciclosporin | Acquired severe aplastic anaemia and transfusion dependent non-severe aplastic anaemia MedDRA version: 14.0;Level: LLT;Classification code 10002274;Term: Anemia aplastic;System Organ Class: 10005329 - Blood and lymphatic system disorders | Trade Name: Thymoglobuline Product Name: Thymoglobuline®/Thymoglobulin® Product Code: anti-thymocyte globulin (rabbit) INN or Proposed INN: GLYCINE INN or Proposed INN: Sodium chloride INN or Proposed INN: Mannitol | EBMT (European group for Blood and Marrow Transplantation) | NULL | Not Recruiting | Female: yes Male: yes | 35 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Germany;United Kingdom | ||
| 23 | NCT00260689 (ClinicalTrials.gov) | November 28, 2005 | 1/12/2005 | Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia | A Randomized Study of Three Immunosuppressive Regimens in Treatment Naive Patients With Severe Aplastic Anemia: Horse ATG/CsA Taper vs Rabbit-ATG/CsA vs Alemtuzumab | Immunosuppresion;Thrombocytopenia;Pancytopenia;Neutropenia | Biological: Anti-thymocyte globulin (rabbit);Biological: Anti-thymocyte globulin (horse);Drug: Cyclosporine;Drug: Alemtuzumab | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Completed | 2 Years | N/A | All | 136 | Phase 2 | United States |
| 24 | 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 |
| 25 | 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 |
65. 原発性免疫不全症候群
臨床試験数 : 500 / 薬物数 : 614 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 217
| 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 | NCT03597594 (ClinicalTrials.gov) | September 8, 2021 | 19/6/2018 | Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) | Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) | Severe Combined Immunodeficiency | Drug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa;Device: CliniMACS;Other: Donor Lymphocyte Infusion | St. Jude Children's Research Hospital | NULL | Recruiting | 2 Months | N/A | All | 42 | Phase 1/Phase 2 | United States |
| 2 | NCT01220531 (ClinicalTrials.gov) | November 8, 2010 | 22/9/2010 | Thymus Transplantation Safety-Efficacy | Safety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836 | Complete DiGeorge Anomaly;DiGeorge Syndrome;DiGeorge Anomaly;Complete DiGeorge Syndrome | Biological: Cultured Thymus Tissue;Procedure: Blood Draw;Drug: Rabbit anti-thymocyte globulin;Drug: Cyclosporine;Drug: Tacrolimus;Drug: Methylprednisolone or Prednisolone;Drug: Mycophenolate mofetil | M. Louise Markert | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Enzyvant Therapeutics GmbH | Available | N/A | N/A | All | United States | ||
| 3 | NCT00579527 (ClinicalTrials.gov) | December 19, 2005 | 20/12/2007 | Phase I/II Thymus Transplantation With Immunosuppression #950 | Phase I/II Trial of Thymus Transplantation With Immunosuppression, #950 | DiGeorge Anomaly;Complete DiGeorge Anomaly;Complete Atypical DiGeorge Anomaly;Complete DiGeorge Syndrome;Complete Atypical DiGeorge Syndrome | Biological: Cultured Thymus Tissue for Implantation (CTTI);Other: Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation;Procedure: Blood Draw;Drug: Rabbit anti-thymocyte globulin;Drug: Cyclosporine;Drug: Tacrolimus;Drug: Methylprednisolone or Prednisolone;Drug: Daclizumab;Drug: Mycophenolate mofetil | Enzyvant Therapeutics GmBH | National Institutes of Health (NIH);National Institute of Allergy and Infectious Diseases (NIAID);Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Completed | N/A | N/A | All | 14 | Phase 1/Phase 2 | United States |
96. クローン病
臨床試験数 : 2,442 / 薬物数 : 1,278 - (DrugBank : 248) / 標的遺伝子数 : 142 - 標的パスウェイ数 : 209
| 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 | NCT04224558 (ClinicalTrials.gov) | December 15, 2020 | 4/4/2018 | Stem Cell Transplantation in Crohn's Disease | Autologous Hematopoietic Stem Cell Transplantation for Refractory Crohn's Disease | Crohn Disease | Drug: Mesna;Drug: Cyclophosphamide;Drug: Filgrastim;Procedure: Apheresis catheter placement;Procedure: Leukapheresis;Drug: Fludarabine;Drug: Methylprednisolone;Drug: Diphenhydramine;Drug: Acetaminophen;Drug: anti-thymocyte globulin (rabbit);Drug: lymphocyte immune globulin;Biological: Peripheral Blood Stem Cell Infusion;Drug: Cytoxan | Cedars-Sinai Medical Center | NULL | Recruiting | 13 Years | 28 Years | All | 15 | Phase 1/Phase 2 | United States |
| 2 | EUCTR2017-002545-30-GB (EUCTR) | 06/11/2017 | 07/06/2018 | Controlled trial to assess low intensity autologous stem cell transplant in refractory Crohn's Disease | Autologous Stem cell Transplantation In refractory Crohn's disease - Low Intensity Therapy Evaluation - ASTIClite | Refractory Crohn's Disease MedDRA version: 20.0;Level: PT;Classification code 10011401;Term: Crohn's disease;System Organ Class: 10017947 - Gastrointestinal disorders;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Trade Name: Cyclophosphamide Product Name: Cyclophosphamide INN or Proposed INN: Cyclophosphamide monohydrate INN or Proposed INN: Cyclophosphamide monohydrate Trade Name: Filgrastim Product Name: Filgrastim INN or Proposed INN: Filgrastim Trade Name: Fludarabine Product Name: Fludarabine INN or Proposed INN: Fludarabine phosphate Trade Name: Thymoglobuline Product Name: Thymoglobuline INN or Proposed INN: Rabbit anti-human thymocyte immunoglobulin | Barts Health NHS Trust | NULL | Not Recruiting | Female: yes Male: yes | 99 | Phase 3 | United Kingdom | ||
| 3 | EUCTR2005-003337-40-IT (EUCTR) | 19/11/2007 | 30/12/2008 | Autologous Stem Cell Transplantation for Crohn?s Disease: Autologous Stem Cell Transplantation International Crohn?s Disease Trial A multicentre, prospective, randomised phase III study conducted by the European Crohn?s and Colitis Organisation (ECCO), sponsored by the Autoimmune Disease Working Party of the European Group for Blood and Marrow Transplantation (EBMT) - ASTIC | Autologous Stem Cell Transplantation for Crohn?s Disease: Autologous Stem Cell Transplantation International Crohn?s Disease Trial A multicentre, prospective, randomised phase III study conducted by the European Crohn?s and Colitis Organisation (ECCO), sponsored by the Autoimmune Disease Working Party of the European Group for Blood and Marrow Transplantation (EBMT) - ASTIC | Crohn`s disease MedDRA version: 9.1;Level: LLT;Classification code 10013099;Term: Disease Crohns | Trade Name: ENDOXAN BAXTER INN or Proposed INN: Cyclophosphamide Trade Name: THYMOGLOBULINE INN or Proposed INN: Antithymocyte immunoglobulin (rabbit) | WOLFSON DIGESTIVE DISEASES CENTRE | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 3 | United Kingdom;Italy | ||
| 4 | EUCTR2005-003337-40-GB (EUCTR) | 13/02/2006 | 13/01/2006 | Autologous Stem Cell Transplantation for Crohn's Disease ('ASTIC'-Trial: Autologous Stem Cell Transplantation International Crohn's Disease Trial) - ASTIC | Autologous Stem Cell Transplantation for Crohn's Disease ('ASTIC'-Trial: Autologous Stem Cell Transplantation International Crohn's Disease Trial) - ASTIC | Patients with Crohn's disease who have not responded to immunosupressant medication | Product Name: Cyclophosphamide Product Code: N/A INN or Proposed INN: cyclophosphamide Other descriptive name: N/A INN or Proposed INN: cyclophosphamide Other descriptive name: N/A Product Name: Filgrastim Product Code: N/A INN or Proposed INN: Filgrastim Other descriptive name: N/A Product Name: rabbit antithymocyte globulin Product Code: rbATG INN or Proposed INN: rabbit antithymocyte globulin Other descriptive name: N/A Product Name: Mesna Product Code: N/A INN or Proposed INN: mensa Other descriptive name: N/A Product Name: methyl prednisolone Product Code: N/A INN or Proposed INN: methyl prednisolone Other descriptive name: N/A Product Name: ciprofloxacin Product Code: N/A INN or Proposed INN: ciprofloxacin Other descriptive name: N/A | European Group for Blood and Marrow Transplantation (EMBT) | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | United Kingdom |
285. ファンコニ貧血
臨床試験数 : 62 / 薬物数 : 93 - (DrugBank : 30) / 標的遺伝子数 : 30 - 標的パスウェイ数 : 144
| 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 | NCT04784052 (ClinicalTrials.gov) | December 7, 2021 | 2/3/2021 | Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing JSP191 Antibody | TCRaß+ T-cell/CD19+ B-cell Depleted Hematopoietic Grafts and a Reduced Intensity Preparative Conditioning Regimen Containing JSP191 to Achieve Engraftment and Blood Reconstitution in Patients With Fanconi Anemia | Fanconi Anemia | Drug: JSP191;Device: CliniMACS Prodigy System;Biological: Depleted Stem Cell Transplant;Biological: Rabbit Anti-Thymoglobulin (rATG);Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Rituximab | Rajni Agarwal | NULL | Recruiting | 2 Years | N/A | All | 12 | Phase 1/Phase 2 | United States |
| 2 | NCT03600909 (ClinicalTrials.gov) | May 15, 2018 | 17/7/2018 | A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia | A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | Fanconi Anemia;Myelodysplastic Syndrome (MDS);Acute Myelogenous Leukemia (AML) | Drug: Busulfan;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Anti-Thymocyte Globulin (Rabbit);Device: The CliniMACS device;Drug: G-CSF | Memorial Sloan Kettering Cancer Center | Pediatric Brain Tumor Consortium | Completed | 1 Month | N/A | All | 3 | Phase 2 | United States |
| 3 | NCT02143830 (ClinicalTrials.gov) | April 2014 | 28/4/2014 | HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy | A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | Fanconi Anemia;Severe Marrow Failure;Myelodysplastic Syndrome (MDS);Acute Myelogenous Leukemia (AML) | Drug: Busulfan;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: rabbit ATG;Drug: G-CSF;Biological: Peripheral blood stem cell | Children's Hospital Medical Center, Cincinnati | Fred Hutchinson Cancer Center;Memorial Sloan Kettering Cancer Center | Recruiting | 3 Months | N/A | All | 70 | Phase 2 | United States |