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 | NCT05018936 (ClinicalTrials.gov) | October 1, 2021 | 19/8/2021 | Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia | Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia: a Prospective Single Arm Study | Aplastic Anemia;Drug Effect | Drug: Hetrombopag | Peking Union Medical College Hospital | NULL | Not yet recruiting | 18 Years | 75 Years | All | 40 | Phase 2/Phase 3 | China |
| 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 | ChiCTR2100045322 | 2021-06-01 | 2021-04-12 | Efficacy and safety of umbilical cord mesenchymal stem cells combined with standard immunosuppressive agents in the treatment of children with acquired severe aplastic anemia: a randomized, controlled clinical study | Efficacy and safety of umbilical cord mesenchymal stem cells combined with standard immunosuppressive agents in the treatment of children with acquired severe aplastic anemia: a randomized, controlled clinical study | pediatric acquired severe aplastic anemia | Experimental group:Anti-thymocyte globulin+Ciclosporin A+Mesenchymal stem cell;Control group:Anti-thymocyte globulin+Ciclosporin A; | Institute of Hematology & Blood Disease Hospital Chinese Academy of Medical Sciences | NULL | Recruiting | 1 | 16 | Both | Experimental group:32;Control group:32; | China | |
| 4 | NCT04854889 (ClinicalTrials.gov) | April 22, 2021 | 14/4/2021 | Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia | Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia (LODACA): a Phase 2, Single-arm, Open-label Study | Refractory Aplastic Anemia | Drug: Decitabine | Institute of Hematology & Blood Diseases Hospital | NULL | Recruiting | 12 Years | N/A | All | 45 | Phase 2 | China |
| 5 | ChiCTR2100043229 | 2021-02-22 | 2021-02-09 | A Single-Center, Single-Arm, Phase II Clinical Study of the Efficacy and Safety of Cyclosporine Combined with Eltrombopag Olamine Tablets in Treatment-Naive Transfusion-Dependent Non-Severe Aplastic Anemia | A Single-Center, Single-Arm, Phase II Clinical Study of the Efficacy and Safety of Cyclosporine Combined with Eltrombopag Olamine Tablets in Treatment-Naive Transfusion-Dependent Non-Severe Aplastic Anemia | Aplastic anemia | Durgs Group:Elrombopag combined with Cyclosporine; | Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences | NULL | Recruiting | 18 | 85 | Both | Durgs Group:61; | Phase 2 | China |
| 6 | NCT04728789 (ClinicalTrials.gov) | February 1, 2021 | 1/12/2020 | Avatrombopag Usage in NSAA | Efficacy and Safety of Avatrombopag in Non-severe Aplastic Anemia - a Multicenter Prospective Single Arm Study | Aplastic Anemia;Drug Effect | Drug: Avatrombopag 20 MG | Peking Union Medical College Hospital | NULL | Not yet recruiting | 18 Years | 75 Years | All | 40 | Phase 2/Phase 3 | China |
| 7 | NCT04409080 (ClinicalTrials.gov) | January 13, 2021 | 26/5/2020 | REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy | A Phase 1/2 Study of REGN7257 (Anti-Interleukin 2 Receptor Subunit Gamma [IL2RG] Monoclonal Antibody) in Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy | Severe Aplastic Anemia (SAA) | Drug: REGN7257 | Regeneron Pharmaceuticals | NULL | Recruiting | 18 Years | N/A | All | 48 | Phase 1/Phase 2 | United States;France;Korea, Republic of;United Kingdom |
| 8 | 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 |
| 9 | NCT04439006 (ClinicalTrials.gov) | October 23, 2020 | 17/6/2020 | Ibrutinib for the Treatment of COVID-19 in Patients Requiring Hospitalization | Randomized Double-Blind Phase 2 Trial of Ibrutinib Versus Standard Treatment for COVID-19 Illness Requiring Hospitalization With Safety Lead-In | Aplastic Anemia;Hematopoietic and Lymphoid Cell Neoplasm;Malignant Solid Neoplasm;Monoclonal B-Cell Lymphocytosis;Monoclonal Gammopathy of Undetermined Significance;Myelodysplastic Syndrome;Symptomatic COVID-19 Infection Laboratory-Confirmed | Other: Best Practice;Drug: Ibrutinib | Jennifer Woyach | Janssen Scientific Affairs, LLC | Active, not recruiting | 18 Years | N/A | All | 10 | Phase 2 | United States |
| 10 | NCT05088642 (ClinicalTrials.gov) | September 28, 2020 | 12/10/2021 | A Trial of Hetrombopag in Healthy and Hepatic Impairment Subjects | A Phase I Clinical Study Evaluating the Pharmacokinetics and Safety of Hetrombopag Olamine Tablets in Subjects With Mild Hepatic Impairment (Child-Pugh Class A), Moderate Hepatic Impairment (Child-Pugh Class B), and Normal Hepatic Function | Sever Aplastic Anaemia | Drug: Hetrombopag Olamine Tablet | Jiangsu HengRui Medicine Co., Ltd. | NULL | Completed | 18 Years | 65 Years | All | 24 | Phase 1 | China |
| 11 | NCT04478227 (ClinicalTrials.gov) | August 18, 2020 | 10/7/2020 | TPO-Mimetic Use in Children for Hemotopoietic Failure | Single Arm Prospective Open Label Pilot Study Evaluating Short-Term Safety and Efficacy of Romiplostim in Children With Inherited and Acquired Disorders of Hematopoietic Failure | Bone Marrow Failure Disorders;Aplastic Anemia;Thrombocytopenia;Refractory Cytopenia of Childhood;Myelodysplastic Syndrome(MDS) | Drug: Romiplostim | Anjali Sharathkumar | Amgen | Recruiting | N/A | 21 Years | All | 25 | Early Phase 1 | United States |
| 12 | NCT04350606 (ClinicalTrials.gov) | July 25, 2020 | 1/4/2020 | A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia | A MULTICENTER, OPEN-LABEL, SINGLE-ARM STUDY TO ASSESS THE EFFICACY AND SAFETY OF PF-06462700 ADMINISTERED INTRAVENOUSLY AT 40 MG/KG/DAY FOR 4 DAYS IN JAPANESE PARTICIPANTS WITH MODERATE AND ABOVE APLASTIC ANEMIA | Aplastic Anemia | Biological: PF-06462700 | Pfizer | NULL | Completed | 2 Years | N/A | All | 3 | Phase 3 | Japan |
| 13 | NCT04403321 (ClinicalTrials.gov) | July 1, 2020 | 21/5/2020 | Efficacy and Safety of Eltrombopag + Tacrolimus/Cyclosporin A in Chinese Refractory or Relapsed Aplastic Anemia Patients | Efficacy and Safety of Eltrombopag + Tacrolimus/Cyclosporin A in Chinese Refractory or Relapsed Aplastic Anemia Patients | Aplastic Anemia;Drug Effect | Drug: Tacrolimus;Drug: Placebo (for Tacrolimus);Drug: Cyclosporin A | Peking Union Medical College Hospital | NULL | Not yet recruiting | 16 Years | 80 Years | All | 60 | Phase 2 | China |
| 14 | NCT04304820 (ClinicalTrials.gov) | May 7, 2020 | 10/3/2020 | Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA) | Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA) | Severe Aplastic Anemia | Drug: Eltrombopag;Drug: Cyclosporine;Drug: Horse-Anti-thymocyte-Globulin | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Recruiting | 3 Years | N/A | All | 39 | Phase 2 | United States |
| 15 | NCT03988608 (ClinicalTrials.gov) | December 9, 2019 | 13/6/2019 | Study to Assess the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects. | A Non-randomized, Open-label, Multi-center, Phase II Study to Assess the Safety and Efficacy of Eltrombopag in Chinese Subjects With Refractory or Relapsed Severe Aplastic Anemia | Aplastic Anemia | Drug: Eltrombopag 25 mg | Novartis Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 20 | Phase 2 | China |
| 16 | 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 |
| 17 | NCT03896971 (ClinicalTrials.gov) | November 15, 2019 | 21/3/2019 | Combination of Thrombopoietin Mimetic and Immunosuppressive Therapy in Aplastic Anaemia | Treatment With Thrombopoietin Mimetic Plus Immunosuppressive Therapy in Egyptian Patients With Aplastic Anaemia | Aplastic Anemia | Drug: Combination of thrombopoietin mimetic and cyclosporin A | Safaa AA Khaled | Assiut University | Completed | 16 Years | 65 Years | All | 66 | Phase 4 | Egypt |
| 18 | EUCTR2016-002814-29-IT (EUCTR) | 14/06/2019 | 24/05/2021 | SOAR, Interventional phase II single-arm study to assess efficacy and safety of eltrombopag combined with cyclosporine as first line therapy in adult patients with severe acquired aplastic anemia | SOAR, Interventional phase II single-arm study to assess efficacy and safety of eltrombopag combined with cyclosporine as first line therapy in adult patients with severe acquired aplastic anemia - SOAR | First-line severe aplastic anaemia MedDRA version: 20.0;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: SANDIMMUN NEORAL - 10 MG CAPSULE MOLLI 50 CAPSULE Product Name: Sandimmun Neoral Product Code: [Sandimmun Neoral ] INN or Proposed INN: 00070500 Other descriptive name: CICLOSPORINA Trade Name: SANDIMMUN NEORAL - 25 MG CAPSULE MOLLI 50 CAPSULE Product Name: Sandimmun Neoral Product Code: [Sandimmun Neoral] INN or Proposed INN: 00070500 Other descriptive name: CICLOSPORINA Trade Name: SANDIMMUN NEORAL - 50 MG CAPSULE MOLLI 50 CAPSULE Product Name: Sandimmun Neoral Product Code: [Sandimmun Neoral] INN or Proposed INN: 00070500 Trade Name: SANDIMMUN NEORAL - 100 MG CAPSULE MOLLI 30 CAPSULE Product Name: Sandimmun Neoral Product Code: [Sandimmun Neoral] INN or Proposed INN: 00070500 Trade Name: REVOLADE - 25 MG - COMPRESSA RIVESTITA CON FILM - USO ORALE - BLISTER(PA/ALU/PVC/ALU) 28 COMPRESSE Product Name: Eltrombopag Product Code: [ETB115] INN or Proposed INN: 00319301 Other descriptive name: Eltrombopag Trade Name: REVOLADE - 50 MG - COMPRESSA RIVESTITA CON FILM - USO ORALE - BLISTER(PA/ALU/PVC/ALU | NOVARTIS PHARMA AG | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 2 | Hungary;Hong Kong;Mexico;Spain;Thailand;Brazil;Korea, Democratic People's Republic of;Turkey;Netherlands;Italy;India | ||
| 19 | EUCTR2015-003166-91-GB (EUCTR) | 22/05/2019 | 05/07/2017 | A phase II dose escalation study: use of eltrombopag in pediatric patientswith 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 naïve severe aplastic anemia or recurrent aplastic anemia. | Refractory, relapsed or treatment naïve severe aplastic anemia (SAA) or recurrent aplastic anemia (AA) MedDRA version: 20.0;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 Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | United States;Portugal;Hong Kong;Thailand;Russian Federation;Netherlands;United Kingdom | ||
| 20 | NCT03520647 (ClinicalTrials.gov) | February 19, 2019 | 9/5/2018 | Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis | Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis | Severe Aplastic Anemia (SAA);Hypo-Plastic Myelodysplastic Syndrome (MDS);Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: Cyclophosphamide;Other: Peripheral Blood Stem Cells | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Recruiting | 4 Years | 75 Years | All | 56 | Phase 2 | United States |
| 21 | ChiCTR1800019669 | 2019-01-01 | 2018-11-21 | A randomized, open-label, single-center controlled trial for the efficacy and safety of recombinant human thrombopoietin injection in the treatment of severe Aplastic Anemia with thrombocytopenia | A randomized, open-label, single-center controlled trial for the efficacy and safety of recombinant human thrombopoietin injection in the treatment of severe Aplastic Anemia with thrombocytopenia | Aplastic anemia,Thrombocytopenia | Group 1:Add rhTPO, 300U/kg qd after ATG treatment for 8 weeks;Group 2:Add rhTPO, 300U/kg qod after ATG treatment for 8 weeks; | Institute of Hematology, People's Hospital, Peking University | NULL | Pending | Both | Group 1:30;Group 2:30; | China | |||
| 22 | NCT03821987 (ClinicalTrials.gov) | December 17, 2018 | 23/1/2019 | Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA | Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA | Aplastic Anemia;Stem Cell Transplant Complications;Engraft Failure | Drug: Fludarabine | Peking University People's Hospital | NULL | Recruiting | 3 Years | 55 Years | All | 55 | N/A | China |
| 23 | EUCTR2015-003166-91-NL (EUCTR) | 14/11/2018 | 08/08/2017 | A phase II dose escalation study: use of eltrombopag in pediatric patients with 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 naïve severe aplastic anemia or recurrent aplastic anemia. | Refractory, relapsed or treatment naïve severe aplastic anemia (SAA) or recurrent aplastic anemia (AA) MedDRA version: 20.0;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 Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | Portugal;United States;Hong Kong;Thailand;Russian Federation;Netherlands;United Kingdom | ||
| 24 | NCT03557099 (ClinicalTrials.gov) | June 20, 2018 | 3/6/2018 | A Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia (SAA) Patient | A Multicentre, Open-label, Single-arm, Phase II Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Patients With Severe Aplastic Anemia | Severe Aplastic Anemia | Drug: Hetrombopag Olamine | Jiangsu HengRui Medicine Co., Ltd. | NULL | Recruiting | 18 Years | N/A | All | 55 | Phase 1/Phase 2 | China |
| 25 | 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 |
| 26 | EUCTR2015-003166-91-PT (EUCTR) | 15/12/2017 | 13/07/2017 | 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 naïve severe aplastic anemia or recurrent aplastic anemia. | Refractory, relapsed or treatment naïve severe aplastic anemia (SAA) or recurrent aplastic anemia (AA) MedDRA version: 20.0;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 Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | United States;Portugal;Hong Kong;Thailand;Russian Federation;Netherlands;United Kingdom | ||
| 27 | ChiCTR-IOR-17012957 | 2017-10-31 | 2017-10-12 | A single Central, open, parallel control study to evaluate the safety and effectiveness of Arsenious Acid- Immunological preparationsand Immunological preparations in aplastic anemia (AA) | A single Central, open, parallel control study to evaluate the safety and effectiveness of Arsenious Acid- Immunological preparationsand Immunological preparations in aplastic anemia (AA) | aplastic anemia | A:ATO+CsA;B:CsA; | Henan Province Cancer Hospital | NULL | Pending | 18 | 65 | Both | A:100;B:100; | China | |
| 28 | EUCTR2016-002814-29-HU (EUCTR) | 07/09/2017 | 05/07/2017 | SOAR Trial: Eltrombopag combined with cyclosporine as first line therapy in patients with severe acquired aplastic anemia with follow-up up to 60-months. | SOAR Trial, A two-part study: Interventional phase II single-arm trial to assess efficacy and safety of Eltrombopag combined with cyclosporine as first line therapy in patients with severe acquired aplastic anemia, and an extension with up to 60-months follow-up. - SOAR | First-line severe aplastic anaemia MedDRA version: 20.0;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 Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG | Novartis Pharma AG | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 2 | France;Hong Kong;Hungary;Mexico;Spain;Brazil;Turkey;Netherlands;Italy;Korea, Republic of;India | ||
| 29 | EUCTR2016-002814-29-NL (EUCTR) | 21/07/2017 | 29/03/2017 | SOAR Trial: Eltrombopag combined with cyclosporine as first line therapy in adult patients with severe acquired aplastic anemia with follow-up up to 24-months. | SOAR, Interventional phase II single-arm study to assess efficacy and safety of eltrombopag combined with cyclosporine as first line therapy in adult patients with severe acquired aplastic anemia - SOAR | First-line severe aplastic anaemia MedDRA version: 20.0;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 Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG | Novartis Pharma AG | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 2 | Hungary;Hong Kong;Mexico;Thailand;Spain;Brazil;Turkey;Netherlands;Italy;Korea, Republic of;India | ||
| 30 | ChiCTR-IOR-17011653 | 2017-07-01 | 2017-06-14 | A prospective, randomized, controlled clinical study of non transfusion dependent non severe aplastic anemia treated with cyclosporine, androgen and levamisole hydrochloride | A prospective, randomized, controlled clinical study of non transfusion dependent non severe aplastic anemia treated with cyclosporine, androgen and levamisole hydrochloride | Aplastic anemia | SA group:none;LSA group:levamisole hydrochloride; | The First affiliated Hospital of Zhejiang Chinese Medical University | NULL | Pending | 18 | 70 | Both | SA group:124;LSA group:124; | China | |
| 31 | NCT02998645 (ClinicalTrials.gov) | May 11, 2017 | 4/11/2016 | Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia | SOAR, Interventional Phase II Single-arm Study to Assess Efficacy and Safety of Eltrombopag Combined With Cyclosporine as First Line Therapy in Adult Patients With Severe Acquired Aplastic Anemia | Severe Aplastic Anemia | Drug: eltrombopag;Drug: Cyclosporine | Novartis Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 54 | Phase 2 | Brazil;Hong Kong;India;Italy;Korea, Republic of;Mexico;Spain;Thailand;Turkey;Hungary;Netherlands |
| 32 | EUCTR2016-002814-29-ES (EUCTR) | 21/03/2017 | 16/12/2016 | SOAR Trial: Eltrombopag combined with cyclosporine as first line therapy in patients with severe acquired aplastic anemia with follow-up up to 60-months. | SOAR Trial, A two-part study: Interventional phase II single-arm trial to assess efficacy and safety of Eltrombopag combined with cyclosporine as first line therapy in patients with severe acquired aplastic anemia, and an extension with up to 60-months follow-up. - SOAR | First-line severe aplastic anaemia 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 Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG Trade Name: REVOLADE Product Name: Eltrombopag Product Code: ETB115 INN or Proposed INN: ELTROMBOPAG | Novartis Farmacéutica, S.A | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 2 | Qatar;Hong Kong;Spain;Turkey;Italy;India;France;Hungary;Mexico;Canada;Brazil;Netherlands;Korea, Republic of | ||
| 33 | NCT03055078 (ClinicalTrials.gov) | January 2017 | 4/2/2017 | Umbilical Cord Derived Mesenchymal Stem Cells Therapy in Aplastic Anemia | Evaluation of Safety and Efficacy in Patients With Aplastic Anemia by Transplantation of Umbilical Cord Derived Mesenchymal Stem Cells | Aplastic Anemia | Biological: mesenchymal stem cells | Hebei Medical University | NULL | Recruiting | 14 Years | 60 Years | All | 10 | Phase 1 | China |
| 34 | NCT02857530 (ClinicalTrials.gov) | August 2016 | 26/7/2016 | Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia | Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia | Anemia, Aplastic | Drug: rhTPO;Drug: placebo | Tianjin Medical University General Hospital | NULL | Not yet recruiting | 6 Years | 60 Years | Both | 250 | Phase 2 | China |
| 35 | NCT01861093 (ClinicalTrials.gov) | October 16, 2015 | 21/5/2013 | Safety Study of Cord Blood Units for Stem Cell Transplants | A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units (CBUs) Manufactured by the National Cord Blood Program (NCBP) and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients | Aplastic Anemia;Leukemia;Myelodysplastic Syndrome (MDS);Lymphoma | Biological: Cord Blood Units | National Heart, Lung, and Blood Institute (NHLBI) | National Cancer Institute (NCI);National Institute of Allergy and Infectious Diseases (NIAID);National Cord Blood Program, New York Blood Center | Recruiting | 4 Years | N/A | All | 500 | Phase 2 | United States |
| 36 | NCT02462252 (ClinicalTrials.gov) | October 2015 | 25/5/2015 | Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome | A Phase IIA, Open-label Study Designed to Evaluate Efficacy and Safety of BL-8040 Followed by Anti-Thymocyte Globulin (hATG), Cyclosporine and Methylprednisolone in Adult Subjects With Aplastic Anemia (AA) or Hypoplastic Myelodysplastic Syndrome (MDS) | Aplastic Anemia;Hypoplastic Myelodysplastic Syndrome | Drug: BL-8040;Drug: horse anti-thymocyte globulin (hATG);Drug: Methylprednisolone;Drug: Cyclosporine | BioLineRx, Ltd. | NULL | Recruiting | 18 Years | N/A | All | 25 | Phase 2 | United States |
| 37 | 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 |
| 38 | EUCTR2014-000174-19-DE (EUCTR) | 27/01/2015 | 01/09/2014 | Efficacy and Safety of Eltrombopag in Patients with Acquired Moderate Aplastic Anemia (EMAA) who are treated with Ciclosporin A Prospective Randomized Multicenter Study comparing Thrombopoetin-Receptor agonist Eltrombopag with Placebo in Patients with Acquired Moderate Aplastic Anemia who are treated with Ciclosporin A | Efficacy and Safety of Eltrombopag in Patients with Acquired Moderate Aplastic Anemia (EMAA) who are treated with Ciclosporin A Prospective Randomized Multicenter Study comparing Thrombopoetin-Receptor agonist Eltrombopagwith Placebo in Patients with Acquired Moderate Aplastic Anemia who are treated with Ciclosporin A - EMAA | Moderate Aplastic Anemia (MAA);Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Revolade 75 mg Product Name: Eltrombopag Product Code: SB497115 INN or Proposed INN: Eltrombopag Other descriptive name: ELTROMBOPAG OLAMINE | Universitätsklinikum Ulm | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Phase 2;Phase 3 | France;Netherlands;Germany;Italy;Switzerland | ||
| 39 | 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 |
| 40 | 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 |
| 41 | NCT02148133 (ClinicalTrials.gov) | July 23, 2014 | 22/5/2014 | A Study to Assess the Safety and Efficacy of Eltrombopag in Japanese Subjects With Refractory, Moderate or More Severe Aplastic Anemia | A Non-randomized, Open-label, Phase II Study to Assess the Safety and Efficacy of Eltrombopag in Japanese Subjects With Refractory, Moderate or More Severe Aplastic Anemia | Cytopaenia | Drug: Eltrombopag 12.5 mg;Drug: Eltrombopag 25 mg | Novartis Pharmaceuticals | NULL | Completed | 18 Years | 79 Years | All | 21 | Phase 2 | Japan |
| 42 | NCT01818726 (ClinicalTrials.gov) | June 23, 2014 | 5/3/2013 | Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients | Open-label Study of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients Undergoing Treatment Programs in Comparison With Control Group | Aplastic Anemia | Drug: ICL670;Drug: Chelation;Drug: No chelation | Novartis Pharmaceuticals | NULL | Terminated | 18 Years | N/A | All | 15 | Phase 4 | Russian Federation |
| 43 | NCT02094417 (ClinicalTrials.gov) | April 14, 2014 | 18/3/2014 | A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia | A Phase 2, Randomized, Open-Label, Parallel, Comparative, Dose-Finding Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia Subjects With Thrombocytopenia Refractory to Immunosuppressive Therapy | Aplastic Anemia | Drug: AMG531 | Kyowa Kirin Korea Co., Ltd. | NULL | Completed | 19 Years | N/A | All | 35 | Phase 2 | Korea, Republic of |
| 44 | NCT02055456 (ClinicalTrials.gov) | February 2014 | 3/2/2014 | Nandrolone Decanoate in the Treatment of Telomeropathies | Male Hormones for Telomere Related Diseases | Aplastic Anemia;Bone Marrow Failure Syndromes;Idiopathic Pulmonary Fibrosis;Telomere Shortening | Drug: Nandrolone Decanoate | University of Sao Paulo | Conselho Nacional de Desenvolvimento Científico e Tecnológico | Recruiting | 2 Years | N/A | Both | 20 | Phase 1/Phase 2 | Brazil |
| 45 | NCT02007811 (ClinicalTrials.gov) | November 2013 | 21/11/2013 | Open-label Clinical Trial to Investigate the Safety and Tolerability of Allogeneic B-cell Concentrates for Immune Reconstitution After Allogeneic Stem Cell Transplantation Measured as Response to a Antedated Single Vaccination | Prospective, Open-label, Multicentre Clinical Trial, Phase I/IIa, to Investigate the Safety and Tolerability of Allogeneic B-cell Concentrates CD3+-Depleted, CD19+-Enriched, Cryopreserved (Single Administration After Day 120 Following Allogeneic Stem Cell Transplantation (SCT), Donor-identical) in 4 Groups With Escalating Doses for Immune Response Enhancement, Measured as Response to a Antedated Single Vaccination | Acute Myeloid Leukemia;Acute Lymphoblastic Leukemia;Chronic Myeloid Leukemia;Non Hodgkin's Lymphoma;Hodgkin's Disease;Myelodysplastic Syndrome;Multiple Myeloma;Aplastic Anemia | Biological: allogeneic donor derived B-lymphocytes | University of Erlangen-Nürnberg Medical School | University Hospital Regensburg;Wuerzburg University Hospital;University Hospital, Essen;German Research Foundation | Recruiting | 18 Years | 75 Years | Both | 15 | Phase 1/Phase 2 | Germany |
| 46 | NCT02218437 (ClinicalTrials.gov) | October 2013 | 6/8/2014 | Treatment Protocol of Child SAA With the Injection of Mesenchymal Stem Cells(Umbilical Cord Derived) | Child With Severe Aplastic Anemia (SAA) Therapy: the Injection of Umbilical Cord Derived Mesenchymal Stem Cells. | Complications of Organ Transplant Stem Cells Umbilical Cord | Drug: MSC+ATG | Chinese Academy of Medical Sciences | NULL | Recruiting | 1 Month | 18 Years | Both | 20 | Phase 4 | China |
| 47 | NCT01891994 (ClinicalTrials.gov) | June 28, 2013 | 28/6/2013 | Extended Dosing With Eltrombopag for Severe Aplastic Anemia | Extended Dosing With Eltrombopag in Refractory Severe Aplastic Anemia | Severe Aplastic Anemia (SAA) | Drug: Eltrombopag | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Active, not recruiting | 2 Years | 100 Years | All | 40 | Phase 2 | United States |
| 48 | ChiCTR-PRC-13003130 | 2013-04-01 | 2013-03-30 | Voriconazole tablet for primary prophylaxis of invasive fungal infection in patients with severe aplastic anemia: a prospective, randomized, controlled, multi-center trial | The efficacy and pharmacoeconimics of voriconazole tablet for primary prophylaxis of invasive fungal infection in patients with severe aplastic anemia | Severe Aplastic Anemia (SAA) | Trial group:Vfend? (Voriconazole tablet for oral) should be administered 6mg/kg at the first day for adults, and then 200mg, twice daily; 4-7mg/kg/d for children, divided into twice a day,with administration period no longer than 6 weeks.;control:without anti-fungal drug; | Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC | NULL | Completed | 3 | 65 | Both | Trial group:50;control:50; | China | |
| 49 | NCT01586455 (ClinicalTrials.gov) | April 2013 | 25/4/2012 | Human Placental-Derived Stem Cell Transplantation | A Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells Combined With Unrelated and Related Cord Blood in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders | Mucopolysaccharidosis I;Mucopolysaccharidosis VI;Adrenoleukodystrophy;Niemann-Pick Disease;Metachromatic Leukodystrophy;Wolman Disease;Krabbe's Disease;Gaucher's Disease;Fucosidosis;Batten Disease;Severe Aplastic Anemia;Diamond-Blackfan Anemia;Amegakaryocytic Thrombocytopenia;Myelodysplastic Syndrome;Acute Myelogenous Leukemia;Acute Lymphocytic Leukemia | Drug: Human Placental Derived Stem Cell | New York Medical College | NULL | Active, not recruiting | N/A | 55 Years | All | 43 | Phase 1 | United States |
| 50 | NCT01760096 (ClinicalTrials.gov) | January 2013 | 1/1/2013 | Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013) | Phase II Study of the Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013) | Paroxysmal Nocturnal Hemoglobinuria;Hemoglobinuria;Hemoglobinuria, Paroxysmal;Proteinuria;Urination Disorders;Thrombosis;Bone Marrow Failure;Aplastic Anemia,;Anemia, Hemolytic | Drug: Levamisole+cyclosporin A+Glucocorticoids;Drug: cyclosporin A+Glucocorticoids;Drug: Glucocorticoids | Institute of Hematology & Blood Diseases Hospital | NULL | Active, not recruiting | 18 Years | N/A | Both | 50 | Phase 2 | China |
| 51 | NCT01703169 (ClinicalTrials.gov) | November 2012 | 27/9/2012 | Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia | Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia | Severe Aplastic Anemia;Very Severe Aplastic Anemia;Moderate Aplastic Anemia | Drug: Eltrombopag | University of Utah | Novartis | Completed | 18 Years | N/A | All | 13 | Phase 2 | United States |
| 52 | NCT01642979 (ClinicalTrials.gov) | July 2012 | 14/7/2012 | Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria | Phase II Study of the Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria;Hemoglobinuria;Hemoglobinuria, Paroxysmal;Proteinuria;Urination Disorders;Thrombosis;Bone Marrow Failure;Aplastic Anemia,;Anemia, Hemolytic | Drug: Levamisole+cyclosporin A+Glucocorticoids;Drug: cyclosporin A+Glucocorticoids;Drug: Glucocorticoids | Institute of Hematology & Blood Diseases Hospital | NULL | Active, not recruiting | 18 Years | N/A | Both | 120 | Phase 2 | China |
| 53 | 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 |
| 54 | NCT01546415 (ClinicalTrials.gov) | October 2011 | 10/11/2011 | Safety and Efficacy of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia | A Single Arm, Multicenter, Open Label Study of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia | Iron Overload | Drug: Desferasirox | Novartis Pharmaceuticals | NULL | Completed | 18 Years | N/A | Both | 64 | Phase 4 | China |
| 55 | NCT01472055 (ClinicalTrials.gov) | October 2011 | 6/11/2011 | Pharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell Transplantation | Pharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell Transplantation | Acute Leukemia;Chronic Leukemia;Severe Aplastic Anemia | Drug: Fludarabine | Seoul National University Hospital | Ministry of Food and Drug Safety, Korea | Recruiting | N/A | 19 Years | Both | 46 | Phase 2 | Korea, Republic of |
| 56 | JPRN-UMIN000006071 | 2011/08/01 | 31/08/2011 | Allogeneic hematopoietic stem cell transplantation for aplastic anemia using low-dose anti-thymocyte globulin | SAA | Conditioning regimen Fludarabine 30mg/m2/day iv day-6,-5,-4,-3 Cyclophosphamide 25mg/kg/day iv day-6,-5,-4,-3 ATG(thymoglobulin) 1.25mg/kg iv day-4,-3 In case of HSCT from unrelated donor or HLA-mismatched related donor, TBI 2Gy day-1 will be added. | Kanto Study Group for Cell Therapy | NULL | Complete: follow-up complete | 16years-old | 65years-old | Male and Female | 28 | Phase 2 | Japan | |
| 57 | NCT01343680 (ClinicalTrials.gov) | April 2011 | 27/4/2011 | Trial of Two Central Venous Catheter (CVC) Flushing Schemes in Pediatric Hematology and Oncology Patients | A Randomized Controlled Crossover Trial of Two Different Central Venous Catheter Flushing Schemes in Pediatric Hematology and Oncology Patients in Alberta, Canada | Childhood Cancer;Aplastic Anemia;Metabolic Disorders | Drug: Heparin;Drug: Normal saline | Alberta Children's Hospital | Stollery Children's Hospital | Terminated | N/A | 17 Years | Both | 2 | Phase 3 | Canada |
| 58 | NCT01297972 (ClinicalTrials.gov) | February 2011 | 16/2/2011 | Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia | Bone Marrow Mesenchymal Stem Cells in the Treatment of Refractory Severe Acquired Aplastic Anemia | Aplastic Anemia | Biological: Intravenous bone marrow mesenchymal stem cells infusion | University of Sao Paulo | NULL | Completed | 18 Years | 65 Years | Both | 9 | Phase 1/Phase 2 | Brazil |
| 59 | JPRN-UMIN000004264 | 2010/11/01 | 01/11/2010 | A Pharmacokinetics (PK)/Phase I study of intravenous (i.v.) administration of mycophenolate mofetil (MMF) for graft-versus-host disease (GVHD) prophylaxis after allogeneic hematopoietic stem cell transplantation (allo-SCT) | Refractory hematologic disorders, including1. Acute myelogenous leukemia2. Acute lymphoblastic leukemia3. Myelodysplastic syndrome4. Chronic myelogenous leukemia5. Malignant lymphoma6. Aplastic anemia | For GVHD prophylaxis, MMF is administered 4-6 h after allo-SCT at a dose of 1000 mg i.v. (diluted to a concentration of 6 mg/ml using 5% Dextrose, over 2 h) thrice daily (or twice daily in the case of cord blood transplantation) from day 0 to day 10 (for up to 14 days). Thereafter, patients are changed to p.o. MMF at the same dose and interval. After day 31, the dose tapers depending on individual risk factors for GVHD. Blood samples (2 ml) for PK analysis are collected in EDTA tubes at 0, 0.5, 1, 2, 4, 8, and 12 h after the morning dose on days 2 and 9 during i.v. MMF administration and at 0, 1, 2, 4, 8, and 12 h on day 16 during p.o. MMF administration. Total mycophenolic acid (MPA) levels are quantified by reverse-phase HPLC. After quantification, non-compartmental analyses of total MPA concentration time data are conducted to estimate the AUC. | Kobe University Graduate School of Medicine | School of Pharmacy and Pharmaceutical Science, Mukogawa Women's University | Complete: follow-up complete | 15years-old | 69years-old | Male and Female | 10 | Phase 1 | Japan | |
| 60 | NCT01182662 (ClinicalTrials.gov) | August 2010 | 11/8/2010 | Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia | Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat SAA | Aplastic Anemia | Other: Human umbilical cord-derived MSCs and cyclosporin A;Other: cyclosporin A | Shandong University | National Natural Science Foundation of China | Recruiting | 18 Years | 80 Years | Both | 30 | Phase 2 | China |
| 61 | EUCTR2008-005594-35-BE (EUCTR) | 28/10/2009 | 06/08/2009 | Randomized double-blind study of mesenchymal stem cells (MSC) in patients undergoing matched unrelated allogeneic bone marrow or peripheral blood stem cell transplantation- A European multicentre study. - NA | Randomized double-blind study of mesenchymal stem cells (MSC) in patients undergoing matched unrelated allogeneic bone marrow or peripheral blood stem cell transplantation- A European multicentre study. - NA | Hematological disorders of the following types:ALL: acute lymphoblastic leukemia in complete remission (<5% blasts in marrow) up to and including third remission (excluding relapse).AML: acute myelocytic leukemia in remission (excluding relapse).CML: chronic myelocytic leukemia who are in the chronic phase of the disease.Severe aplastic anemia, hemoglobinopathies (thalassemia major).Inborn errors of metabolism.Myelodysplastic syndromes MedDRA version: 9.1;Level: LLT;Classification code 10027703;Term: Mismatched donor bone marrow transplantation therapy | Product Name: Mesenchymal stem cells Product Code: MSC | CHU Sart-Tilman | NULL | Not Recruiting | Female: yes Male: yes | 172 | Phase 3 | Belgium | ||
| 62 | 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 |
| 63 | 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 |
| 64 | NCT00604201 (ClinicalTrials.gov) | May 21, 2008 | 8/1/2008 | Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic Syndrome | Co-Infusion of Umbilical Cord Blood and Haploidentical CD34+ Cells Following Nonmyeloablative Conditioning as Treatment for Severe Aplastic Anemia and MDS Associated With Severe Neutropenia Refractory to Immunosuppressive Therapy | Myelodysplastic Syndrome (MDS) With Refractory Anemia (RA);Severe Aplastic Anemia (SAA) | Biological: Umbilical Cord Blood | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Active, not recruiting | 4 Years | 75 Years | All | 31 | Phase 2 | United States |
| 65 | NCT00455312 (ClinicalTrials.gov) | August 2007 | 30/3/2007 | Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA | Hematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic Anemia | Dyskeratosis Congenita;Aplastic Anemia | Drug: Campath 1H;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Stem Cell Transplantation;Drug: antithymocyte globulin;Drug: Methylprednisolone | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 70 Years | All | 36 | Phase 2/Phase 3 | United States |
| 66 | NCT00673114 (ClinicalTrials.gov) | August 2007 | 27/12/2007 | Unrelated Cord Blood Transplant Plus a Haplo-Identical (Half-Matched), T-Cell Depleted Stem Transplant From a Related Donor for Subjects With High Risk Malignancies | A Prospective, Phase I/II Trial Determining the Efficacy and Safety of Allogeneic Hematopoietic Stem Cell Transplantation Using Banked Unrelated Umbilical Cord Blood Supplemented With Related, Haplo-Identical T-Cell Depleted Stem Cells in Subjects With High Risk Malignancies | Hematologic Malignancy;Myelodysplastic Syndrome (MDS);Aplastic Anemia | Biological: haplo/cord transplant | Joanne Kurtzberg, MD | Miltenyi Biotec GmbH | Completed | N/A | 55 Years | All | 3 | Phase 1/Phase 2 | United States |
| 67 | 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 | |||
| 68 | EUCTR2008-001151-22-IT (EUCTR) | 06/06/2007 | 14/06/2011 | ALEMTUZUMAB AND LOW-DOSE CYCLOSPORINE-A AS ALTERNATIVE IMMUNOSUPPRESSIVE TREATMENT FOR SEVERE APLASTIC ANEMIA (SAA) AND SINGLE-LINEAGE APLASTIC PATIENTS. - Alesaa | ALEMTUZUMAB AND LOW-DOSE CYCLOSPORINE-A AS ALTERNATIVE IMMUNOSUPPRESSIVE TREATMENT FOR SEVERE APLASTIC ANEMIA (SAA) AND SINGLE-LINEAGE APLASTIC PATIENTS. - Alesaa | Aplastic anemia and single-lineage bone marrow failure MedDRA version: 13.1;Level: PT;Classification code 10002965;Term: Aplasia pure red cell;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 13.1;Level: LLT;Classification code 10047350;Term: Very few granulocyte precursors;Classification code 10002967;Term: Aplastic anaemia;Classification code 10001507;Term: Agranulocytosis;System Organ Class: 10005329 - Blood and lymphatic system disorders | Trade Name: MABCAMPATH INN or Proposed INN: Alemtuzumab | UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Italy | |||
| 69 | NCT00895739 (ClinicalTrials.gov) | June 2006 | 7/5/2009 | Alemtuzumab and Low-Dose Cyclosporine in Treating Patients With Severe Aplastic Anemia or Acquired Marrow Failure | Alemtuzumab and Low-Dose Cyclosporine-A as Alternative Immunosuppressive Treatment for Severe Aplastic Anemia (SAA) and Single-Lineage Aplastic Patients | Nonmalignant Neoplasm | Biological: alemtuzumab;Drug: cyclosporine | Federico II University | NULL | Recruiting | 18 Years | N/A | Both | 50 | Phase 2 | Italy |
| 70 | NCT00737685 (ClinicalTrials.gov) | January 2006 | 18/8/2008 | Flu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blood)in Severe Aplastic Anemia (SAA) | Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Bone Marrow (or Mobilized Peripheral Blood) Transplantation in Severe Aplastic Anemia | Anemia, Aplastic | Drug: cyclophosphamide, fludarabine , thymoglobulin | The Korean Society of Pediatric Hematology Oncology | NULL | Active, not recruiting | N/A | 25 Years | Both | 30 | Phase 2 | Korea, Republic of |
| 71 | NCT00438178 (ClinicalTrials.gov) | October 2005 | 21/2/2007 | Safety and Efficacy of Obatoclax Mesylate (GX15-070MS) for the Treatment of Hematological Malignancies | A Phase I Study of Obatoclax Mesylate (GX15-070MS) in Patients With Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), Chronic Myeloid Leukemia (CML)in Myeloid Blast Phase, Myelofibrosis, Previously-Treated Chronic Lymphocytic Leukemia (CLL), or Aplastic Anemia | Hematological Malignancies | Drug: Obatoclax mesylate (GX15-070MS) | Gemin X | NULL | Completed | 18 Years | N/A | Both | 44 | Phase 1 | United States;Canada |
| 72 | NCT00295997 (ClinicalTrials.gov) | May 2005 | 23/2/2006 | Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer, Metastatic Kidney Cancer, or Aplastic Anemia | Non-myeloablative Allogeneic Stem Cell Transplantation With Match Unrelated Donors for Treatment of Hematologic Malignancies and Renal Cell Carcinoma and Aplastic Anemia | Chronic Myeloproliferative Disorders;Kidney Cancer;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Neoplasms | Biological: anti-thymocyte globulin;Biological: filgrastim;Biological: graft-versus-tumor induction therapy;Biological: therapeutic allogeneic lymphocytes;Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methotrexate;Drug: mycophenolate mofetil;Drug: tacrolimus;Procedure: allogeneic bone marrow transplantation;Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation | University of California, San Francisco | National Cancer Institute (NCI) | Active, not recruiting | N/A | 74 Years | Both | 35 | N/A | United States |
| 73 | NCT00806598 (ClinicalTrials.gov) | May 2005 | 9/12/2008 | Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome | Phase II Study of Combination of Thymoglobulin, Cyclosporine, Methylprednisone, and Granulocyte Colony-stimulating Factor (GCSF) in Patients With Newly Diagnosed Aplastic Anemia or With Hypoplastic or Low/Intermediate-1 Risk Myelodysplastic Syndrome | Myelodysplastic Syndrome;Aplastic Anemia | Drug: Thymoglobulin;Drug: Cyclosporine;Drug: Methylprednisolone;Drug: G-CSF | M.D. Anderson Cancer Center | Genzyme, a Sanofi Company | Completed | 15 Years | N/A | All | 53 | Phase 2 | United States |
| 74 | 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 |
| 75 | JPRN-C000000356 | 2004/12/01 | 20/03/2006 | Evaluation of the safety and efficacy of allogeneic hematopoietic stem cell transplantation (HSCT) using alemtuzumab for patients with aplastic anemia | Aplastic anemia | Alemtuzumab is added to the fludarabine-based regimen at 0.16 – 0.25 mg/kg per day for 6 days (days -10 to -5). | GCP-ISS HE0403 group | NULL | Complete: follow-up complete | 20years-old | 65years-old | Male and Female | 38 | Phase 1,2 | Japan | |
| 76 | NCT00516152 (ClinicalTrials.gov) | November 2002 | 13/8/2007 | Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing MUD SCT | Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing Matched Unrelated Donor Stem Cell Transplantation | Chronic Myeloid Leukemia;Acute Myelogenous Leukemia;Myelodysplasia;Acute Lymphocytic Leukemia;Severe Aplastic Anemia;Non-Hodgkin's Lymphoma;Lymphoproliferative Disease;Multiple Myeloma;Advanced Myeloproliferative Disease | Drug: Busulfan/Fludarabine phosphate/Tacrolimus/Methotrexate/G-CSF | University of California, San Francisco | NULL | Completed | 15 Years | 61 Years | Both | 36 | Phase 2 | United States |
| 77 | NCT00399971 (ClinicalTrials.gov) | November 2002 | 14/11/2006 | Safety and Efficacy Study of Ex Vivo Immunotherapy for Treatment of Aplastic Anemia | Phase I/II Study of An Ex Vivo Immunotherapy for Treatment of Idiopathic Aplastic Anemia | Anemia, Aplastic | Procedure: Ex Vivo Immunotherapy;Drug: Ex vivo immunotherapy | Shenzhen Zhongxing Yangfan Biotech Co. Ltd. | The 12th Guangzhou Municipal Hospital;Liu Hua Qiao Hospital | Recruiting | 10 Years | 80 Years | Both | 100 | Phase 1/Phase 2 | China |
| 78 | NCT00053157 (ClinicalTrials.gov) | June 2002 | 27/1/2003 | Sargramostim in Reducing Graft-Versus-Host Disease in Patients Who Are Undergoing Donor Stem Cell Transplantation for Hematologic Cancer or Aplastic Anemia | Use Of Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) To Mobilize Donor Peripheral Blood Stem Cells Along With GM-CSF Administration Post Allogeneic Transplant - A Pilot Study | Chronic Myeloproliferative Disorders;Graft Versus Host Disease;Leukemia;Lymphoma;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Neoplasms | Biological: sargramostim | Roswell Park Cancer Institute | National Cancer Institute (NCI) | Completed | 5 Years | 60 Years | Both | 10 | N/A | United States |
| 79 | NCT00578903 (ClinicalTrials.gov) | February 2002 | 19/12/2007 | Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia | Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors (SAA MUD) | Aplastic Anemia | Drug: Cytoxan;Drug: Campath;Radiation: Total Body Irradiation (TBI);Drug: FK-506;Drug: Methotrexate;Procedure: Stem cell infusion | Baylor College of Medicine | The Methodist Hospital System;Texas Children's Hospital;Center for Cell and Gene Therapy, Baylor College of Medicine | Terminated | N/A | 60 Years | All | 22 | Phase 2 | United States |
| 80 | NCT00053989 (ClinicalTrials.gov) | January 29, 2002 | 5/2/2003 | NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders | Non-Myeloablative Allogeneic Hematopoietic Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies and Disorders | Chronic Myeloproliferative Disorders;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Fanconi Anemia;Aplastic Anemia | Biological: anti-thymocyte globulin;Biological: graft-versus-tumor induction therapy;Biological: sargramostim;Biological: therapeutic allogeneic lymphocytes;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methylprednisolone;Drug: mycophenolate mofetil;Drug: tacrolimus;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation | Roswell Park Cancer Institute | NULL | Completed | 4 Years | 75 Years | All | 41 | Phase 2 | United States |
| 81 | NCT01163942 (ClinicalTrials.gov) | March 2001 | 14/7/2010 | Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF) | A RANDOMIZED CONTROLLED STUDY IN NEWLY DIAGNOSED SEVERE APLASTIC ANEMIA PATIENTS RECEIVING ANTITHYMOCYTE GLOBULIN (ATG), CYCLOSPORIN A, WITH OR WITHOUT G-CSF | Aplastic Anaemia | Drug: G-CSF;Drug: Early retreatment with ATG | European Group for Blood and Marrow Transplantation | CHUGAI sanofi-aventis | Terminated | N/A | N/A | Both | 205 | Phase 3 | Czech Republic;France;Germany;Greece;Italy;Netherlands;Sweden;Switzerland;United Kingdom |
| 82 | NCT00006379 (ClinicalTrials.gov) | June 2000 | 4/10/2000 | Non-Ablative Allo HSCT For Hematologic Malignancies or SAA | Purine-Analog-Containing Non-Myeloablative Allogeneic Stem Cell Transplantation for Treatment of Hematologic Malignancies and Severe Aplastic Anemia | Chronic Myeloproliferative Disorders;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Precancerous/Nonmalignant Condition;Small Intestine Cancer | Biological: anti-thymocyte globulin;Biological: graft-versus-tumor induction therapy;Drug: cyclophosphamide;Drug: fludarabine phosphate;Procedure: peripheral blood stem cell transplantation | Case Comprehensive Cancer Center | National Cancer Institute (NCI) | Completed | N/A | 70 Years | Both | 58 | Phase 2 | United States |
| 83 | NCT00001626 (ClinicalTrials.gov) | June 2, 1997 | 3/11/1999 | Comparing Therapies for the Treatment of Severe Aplastic Anemia | A Randomized Trial of Antithymocyte Globulin and Cyclosporine Versus Cyclophosphamide and Cyclosporine in the Treatment of Severe Aplastic Anemia | Severe Aplastic Anemia (SAA) | Drug: antithymocyte globulin;Drug: cyclophosphamide;Drug: cyclosporine | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Completed | 15 Years | 110 Years | All | 33 | Phase 2 | United States |
| 84 | EUCTR2021-002155-11-Outside-EU/EEA (EUCTR) | 09/06/2021 | Open-label, single-arm, multicenter study in Japanese patients with aplastic anemia | A MULTICENTER, OPEN-LABEL, SINGLE-ARM STUDY TO ACCESS THE EFFICACY AND SAFETY OF PF-06462700 ADMINISTERED INTRAVENOUSLY AT 40 MG/KG/DAY FOR 4 DAYS IN JAPANESE PARTICIPANTS WITH MODERATE AND ABOVE APLASTIC ANEMIA | Moderate and above aplastic anemia;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Atgam Product Name: Atgam INN or Proposed INN: Anti-human T-lymphocyte immunoglobulin from horse Other descriptive name: anti-human immunoglobulin, equine | Pfizer Inc. | NULL | NA | Female: yes Male: yes | Phase 3 | Japan |