aplastic anemia

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Information
Disease name
aplastic anemia
Disease ID
DOID:12449
Description
"A normocytic anemia that is characterized by a deficiency of red blood cells, white blood cells and platelets produced by bone marrow." [url:http\://en.wikipedia.org/wiki/Aplastic_anemia, url:http\://www.nhlbi.nih.gov/health/health-topics/topics/aplastic/]
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
Genes Mutation Description Source Links
NCT ID Status Phase Summary Start date Completion date
NCT06039020 Active, not recruiting ATGAM General Investigation February 21, 2024 February 13, 2026
NCT03025698 Active, not recruiting Phase 2 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 September 30, 2017 January 29, 2025
NCT01659606 Active, not recruiting Phase 2 Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita July 2012 December 2034
NCT04558736 Active, not recruiting Phase 2 Haploidentical HCT for Severe Aplastic Anemia January 21, 2021 July 1, 2026
NCT03333486 Active, not recruiting Phase 2 Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer December 7, 2017 August 28, 2024
NCT04478227 Active, not recruiting Early Phase 1 TPO-Mimetic Use in Children for Hematopoietic Failure August 18, 2020 June 2024
NCT02566304 Active, not recruiting Phase 2 Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies November 13, 2015 February 13, 2024
NCT05972577 Active, not recruiting N/A Geriatric Optimization Plan to Improve Survival in Older Adult Allogeneic Hematopoietic Cell Transplant Candidates, OTIS Study June 2, 2021 December 31, 2024
NCT00053989 Completed Phase 2 NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders January 29, 2002 July 19, 2018
NCT00065260 Completed Phase 2 Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia November 6, 2003 February 5, 2016
NCT00186797 Completed N/A Purified CD34+ Hematopoietic Stem Cell Transplantation From Alternate Donors for Patients With Severe Aplastic Anemia December 2002 May 2007
NCT00343785 Completed Phase 2 Cyclophosphamide and Anti-thymocyte Globulin Followed By Methotrexate and Cyclosporine in Preventing Chronic Graft-Versus-Host Disease in Patients With Severe Aplastic Anemia Undergoing Donor Bone Marrow Transplant February 2006 August 2012
NCT00427336 Completed N/A Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia December 2000 August 2009
NCT00455312 Completed Phase 2/Phase 3 Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA August 2007 June 2016
NCT00471848 Completed Phase 2 Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia August 2008 February 2013
NCT00513175 Completed Non-Myeloablative Allogeneic Stem Cell Transplantation With Matched Unrelated Donors for Treatment of Hematologic Malignancies, Renal Cell Carcinoma, and Aplastic Anemia October 2001 November 2007
NCT00533923 Completed Phase 2 Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic Disorders December 2002 January 2007
NCT00569842 Completed Investigation of the Cylex® ImmuKnow® Assay November 2007 December 2012
NCT00636909 Completed Phase 2 Nonmyeloablative Allo SCT for the Treatment of Hematologic Disorders July 1999
NCT00673114 Completed Phase 1/Phase 2 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 August 2007 April 2012
NCT00767650 Completed Neuropsychological Effects of Immunosuppressive Treatment in Subjects With Aplastic Anemia September 23, 2008 June 28, 2011
NCT00806598 Completed Phase 2 Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome May 2005 June 2012
NCT00856388 Completed N/A Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders January 14, 2009 March 13, 2019
NCT02875743 Completed Phase 4 King's Invasive Aspergillosis Study II December 7, 2016 October 31, 2019
NCT00004474 Completed Phase 3 Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia September 1998 August 2007
NCT03208647 Completed Multi Center Case Control Study on Multiple Risk Factors of Aplastic Anemia January 1, 2003 January 1, 2014
NCT03176849 Completed Phase 4 A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT November 1, 2017 July 1, 2019
NCT00005935 Completed Phase 2 Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic Anemia June 2000 March 2002
NCT00006300 Completed Oral Manifestations of Aplastic Anemia April 2000 April 2002
NCT00011453 Completed Protection Against Benzene Toxicity July 1998 June 2001
NCT00011830 Completed Phase 1 Stem Cell Mobilization Potential in Patients With Aplastic Anemia in Remission February 2001 February 2006
NCT02094417 Completed Phase 2 A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia April 14, 2014 November 7, 2017
NCT00004464 Completed Phase 2 Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria February 1996 November 11, 2008
NCT03896971 Completed Phase 4 Combination of Thrombopoietin Mimetic and Immunosuppressive Therapy in Aplastic Anaemia November 15, 2019 June 1, 2021
NCT02225145 Completed Fertility Preservation in Women Who Will Have Gonadotoxic Therapy or Hematopoetic Stem Cell Transplantation, and in Women With Sickle Cell Disease August 15, 2014 September 20, 2017
NCT02404025 Completed Phase 2 Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A in Naive Aplastic Anemia (AA) Subjects May 12, 2015 September 6, 2017
NCT02462252 Completed Phase 2 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 October 2015 November 2020
NCT02773290 Completed Phase 2/Phase 3 Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia May 2016 July 28, 2020
NCT02833805 Completed Phase 2 NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia September 2016 July 2021
NCT03318159 Completed Phase 2 Posaconazole Prophylaxis During ATG Treatment for hMDS/AA Patients April 20, 2018 July 15, 2023
NCT02960646 Completed Phase 1 Engineered Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies January 18, 2017 February 13, 2023
NCT00987480 Completed Phase 2 Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine September 25, 2009 July 10, 2017
NCT01105273 Completed Phase 1/Phase 2 Human Leukocyte Antigen (HLA)-Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Aplastic Anemia July 2009 July 2012
NCT01187017 Completed Phase 1/Phase 2 A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia August 2010 July 2012
NCT01193283 Completed Phase 1/Phase 2 Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia August 2010 September 2014
NCT01199562 Completed Infection Prophylaxis and Management in Treating Cytomegalovirus (CMV) Infection in Patients With Hematologic Malignancies Previously Treated With Donor Stem Cell Transplant December 2010 December 2013
NCT01231841 Completed Phase 2 Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia March 2005 December 2010
NCT01241357 Completed High-Tc Susceptometer to Monitor Transfusional Iron Overload March 2011 May 2016
NCT01272817 Completed N/A Nonmyeloablative Allogeneic Transplant October 2001 October 2015
NCT01297972 Completed Phase 1/Phase 2 Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia February 2011 November 2013
NCT01350245 Completed Phase 2 Bone Marrow Transplantation of Patients in Remission Using Partially Matched Relative Donor July 2010 May 2014
NCT01384513 Completed Phase 2 A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies August 4, 2011 November 16, 2022
NCT04439006 Completed Phase 1 Ibrutinib for the Treatment of COVID-19 in Patients Requiring Hospitalization October 23, 2020 September 20, 2022
NCT01441037 Completed Phase 1/Phase 2 Danazol for Genetic Bone Marrow and Lung Disorders July 19, 2011 November 14, 2016
NCT01499147 Completed N/A Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies February 2000 May 2013
NCT04403321 Completed Phase 2 Efficacy and Safety of Eltrombopag + Tacrolimus in Chinese Refractory or Relapsed Aplastic Anemia Patients July 1, 2020 July 31, 2022
NCT01529827 Completed Phase 2 Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies February 28, 2012 August 29, 2019
NCT00001398 Completed Phase 1 Stem Cell Factor Medication for Aplastic Anemia October 1993 June 2002
NCT04350606 Completed Phase 3 A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia July 25, 2020 April 19, 2021
NCT00004323 Completed Phase 2 Phase II Study of Bone Marrow Transplantation Using Related Donors in Patients With Aplastic Anemia February 1995
NCT01760655 Completed Phase 2 Reduced-Intensity Conditioning Before Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic Malignancies December 24, 2012 December 5, 2022
NCT04128943 Completed Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria November 1, 2019 July 6, 2020
NCT04095936 Completed Phase 2/Phase 3 Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia December 3, 2019 October 29, 2021
NCT01956799 Completed Identification of Mechanism in the Erythroid Response in Patients With Myelodysplasia Undergoing Chelation Therapy May 2013 December 2019
NCT03988608 Completed Phase 2 Study to Assess the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects. December 9, 2019 May 17, 2023
NCT03957694 Completed Phase 2/Phase 3 Study of AMG531(Romiplostim) in Patients With Aplastic Anemia April 25, 2019 May 26, 2021
NCT02055456 Completed Phase 1/Phase 2 Nandrolone Decanoate in the Treatment of Telomeropathies February 1, 2014 February 1, 2017
NCT04870346 Enrolling by invitation Long Term Follow-up Observational Study After Clinical Trials of AMG531 (Romiplostim) in Patients With Untreated Aplastic Anemia May 10, 2021 June 30, 2024
NCT03906318 No longer available Expanded Access Use of Itacitinib to Treat a Single Patient With Aplastic Anemia
NCT05399732 Not yet recruiting Phase 2 Efficacy and Safety in Transfusion Independent Non-severe Aplastic Anemia July 2022 July 2024
NCT05333861 Not yet recruiting An Observational, Multicenter Study to Evaluate the Safety and Effectiveness of Hetrombopag in Patients With ITP or AA May 1, 2022 May 1, 2026
NCT06004791 Not yet recruiting Phase 4 A Prospective, Randomized, Controlled Study of rhTPO in Combination With Herombopag + CsA vs Herombopag + CsA for the Treatment of Primary TD-NSAA August 2023 August 31, 2025
NCT06287268 Not yet recruiting Revolade Tablets Specified Drug-use Survey June 28, 2024 October 31, 2028
NCT06398457 Not yet recruiting Early Phase 1 Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies June 2024 December 2026
NCT05996393 Not yet recruiting Phase 4 CsA+ATG+AVA vs. CsA+AVA for the Treatment of Newly-diagnosed SAA in the Elderly October 2023 October 2026
NCT06426043 Not yet recruiting Phase 4 A Prospective Study on the Treatment of Recurrent/Refractory/Intolerable NSAA With Lusutrombopag June 2024 August 2025
NCT06424639 Not yet recruiting Phase 4 Luspatercept Plus CsA vs CsA for the Treatment of Newly Diagnosed Non-Transfusion-Dependent NSAA May 2024 December 2025
NCT06430788 Recruiting Phase 2 A Study of Emapalumab for Pediatric Aplastic Anemia May 21, 2024 May 21, 2029
NCT01624805 Recruiting Phase 2 Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome June 25, 2012 June 30, 2026
NCT01758042 Recruiting N/A Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders November 2012 July 2027
NCT02162420 Recruiting N/A Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia January 10, 2015 July 2026
NCT03016806 Recruiting Phase 1 Umbilical Cord Blood Transplantation From Unrelated Donors June 2015 June 2026
NCT03622788 Recruiting Phase 1/Phase 2 Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant August 8, 2019 December 1, 2025
NCT04060485 Recruiting Screening Gene Mutations in Myeloid Cancers by Next Generation Sequencing to Improve Treatment Results August 19, 2019 December 31, 2024
NCT04645199 Recruiting National Longitudinal Cohort of Hematological Diseases December 1, 2020 December 1, 2030
NCT04761965 Recruiting N/A Quantitative MRI of Bone Marrow Fat Fraction in Patients With Trepanobiopsy February 16, 2021 January 1, 2024
NCT05031897 Recruiting Phase 2 Reduced-Intensity Conditioning for the Prevention of Treatment-Related Mortality in Patients Who Undergo a Hematopoietic Stem Cell Transplant October 25, 2021 October 1, 2024
NCT05194397 Recruiting Phase 2 Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors March 21, 2023 June 30, 2026
NCT05386264 Recruiting Phase 1 Autologous Tregs for Aplastic Anaemia July 14, 2022 April 30, 2025
NCT05510505 Recruiting Phase 2 GVHD Prophylaxis by Addition of CD20 Monoclonal Antibody to the Conditioning Regimen in SAA With Treatment of Allo-HSCT December 30, 2021 December 2025
NCT05571332 Recruiting N/A Efficacy and Safety of Avatrombopag Combined With IST for the Treatment of HAAA and SAA With Abnormal Liver Function June 28, 2022 June 28, 2024
NCT05832216 Recruiting Phase 1 Efficacy of Daratumumab to Overcome Platelet Transfusion Refractoriness in Patients With Aplastic Anemia April 17, 2023 December 31, 2026
NCT06004752 Recruiting Phase 2 Efficacy and Safety of CsA+AVA in the Treatment of NSAA in the Elderly August 17, 2023 August 2025
NCT06009965 Recruiting Phase 4 Efficacy of IST Combined With TPO-RA in the Treatment of AA and Establishment of a Recurrence Prediction System January 20, 2023 January 31, 2025
NCT06335277 Recruiting Total Body Irradiation Treatments for Patients Treated at European Institute of Oncology February 22, 2018 December 31, 2024
NCT00038779 Terminated N/A Blood Transplantation for Patients With Hematologic Malignancies or Bone Marrow Failure States August 14, 1996 September 20, 2004
NCT00001962 Terminated Phase 2 A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure November 1999 September 2010
NCT01267643 Terminated Phase 1 Alefacept in Patients With Relapsed/Refractory Aplastic Anemia May 2011 April 16, 2013
NCT01343680 Terminated Phase 3 Trial of Two Central Venous Catheter (CVC) Flushing Schemes in Pediatric Hematology and Oncology Patients April 2011 May 2012
NCT00676806 Terminated Phase 2 A Phase II Study of Umbilical Cord Blood Transplantation July 2005 July 2014
NCT01818726 Terminated Phase 4 Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients June 23, 2014 October 17, 2016
NCT00354419 Terminated Phase 1 Cyclophosphamide, Antithymocyte Globulin, and Total-Body Irradiation in Treating Patients With Severe Aplastic Anemia Undergoing Umbilical Cord Blood Transplant February 2006
NCT00578903 Terminated Phase 2 Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia February 2002 July 2012
NCT01500161 Terminated Phase 2 Pooled Unrelated Donor Umbilical Cord Blood Transplant For Hematologic Malignancy Needing Allogeneic Stem Cell Transplant Without Related HLA-Match November 2011 November 2013
NCT00556400 Terminated Phase 1/Phase 2 Treatment of Menorrhagia in Women With Thrombocytopenia Using Platelets or Platelets and Hormones November 2007 June 2012
NCT04001686 Unknown status Ambispective Observational Study to Evaluate the Incidence and Management of Aplastic Anemia in Spain January 31, 2018 September 30, 2022
NCT01919866 Unknown status Phase 1/Phase 2 Safety Study of CD3/CD19 Depleted Haploidentical Stem Cells March 2004 December 2015
NCT01305694 Unknown status Phase 1/Phase 2 Mesenchymal Stem Cells Transplantation to Patients With Relapsed/Refractory Aplastic Anemia. February 2011 December 2012
NCT00881933 Unknown status Phase 1/Phase 2 Study of Fludarabine + Cyclophosphamide + TBI Conditioning Regimen for Double Units Cord Blood Transplantation(CBT)in Severe Aplastic Anemia(SAA) November 2008 September 2012
NCT04728789 Unknown status Phase 2/Phase 3 Avatrombopag Usage in NSAA February 1, 2021 June 30, 2023
NCT04819607 Unknown status The Evaluating Multidisciplinary Bone Marrow Failure Care in Bone Marrow Failure and Related Disorders. June 9, 2021 April 2024
NCT05018936 Unknown status Phase 2/Phase 3 Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia October 1, 2021 December 1, 2023
NCT00017654 Unknown status N/A Study of Allogeneic Bone Marrow and T-Cell Depleted, CD34+ Peripheral Blood Stem Cell Transplantation in Patients With Aplastic Anemia April 2001
NCT01182662 Unknown status Phase 2 Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia August 2010 August 2013
NCT01145976 Unknown status Phase 3 Comparison of Cy-Atg vs Flu-Atg for the Conditioning Therapy in Allo-HCT for Adult Aplastic Anemia March 2010 February 2016
NCT02007811 Unknown status Phase 1/Phase 2 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 November 2013 December 2015
NCT01995331 Unknown status Phase 4 Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia March 2012 March 2015
NCT00882323 Unknown status Phase 2 Reduced Toxicity Fludarabine (Flu) + Cyclophosphamide (CPM) + Rabbit Antithymocyte Globulin (rATG) Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia (SAA) November 2008 October 2012
NCT03055078 Unknown status Phase 1 Umbilical Cord Derived Mesenchymal Stem Cells Therapy in Aplastic Anemia January 2017 December 2019
NCT03218657 Unknown status N/A Clinical Study of Non Severe Aplastic Anemia Treated With Cyclosporine, Androgen and Levamisole January 1, 2018 July 1, 2022
NCT02838992 Unknown status Phase 4 ATG Combined With Cyclophosphamide And Cord Blood Transfusion in Treating Patients With Severe Aplastic Anemia February 2017 July 2019
NCT02833493 Unknown status Study of MRI Monitoring in Patients With Aplastic Anemia and Low or Int-1 Risk of MDS Complicated With Iron Overload September 2016 October 2019
NCT03373526 Unknown status N/A Effects of Aerobic Training and Inspiratory Muscle Training in Patients During Hematopoietic Stem Cell Transplantation November 11, 2017 December 31, 2019
NCT03821987 Unknown status N/A Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA December 17, 2018 March 30, 2022
NCT02203396 Unknown status Phase 2 A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia August 2014 September 2017
NCT02028416 Unknown status N/A Comparison of Two Different Doses of Rabbit ATG-Fresenius With Cyclosporin in the Treatment of Acquired Aplastic Anaemia September 2013 December 2017
NCT01408342 Withdrawn N/A Alemtuzumab and Rituximab in Aplastic Anemia July 2011 January 2014
NCT03086252 Withdrawn N/A Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study July 24, 2017 May 31, 2018
NCT01680055 Withdrawn Collection of Blood and Bone Marrow From Patients With Aplastic Anemia for Analysis of Adhesion Molecules, Chemokines and Their Receptors February 2009 November 2012
Disase is a (Disease Ontology)
DOID:720
Cross Reference ID (Disease Ontology)
GARD:5836
Cross Reference ID (Disease Ontology)
ICD10CM:D61.9
Cross Reference ID (Disease Ontology)
ICD9CM:284.9
Cross Reference ID (Disease Ontology)
MESH:D000741
Cross Reference ID (Disease Ontology)
MIM:609135
Cross Reference ID (Disease Ontology)
NCI:C2870
Cross Reference ID (Disease Ontology)
SNOMEDCT_US_2023_03_01:154807001
Cross Reference ID (Disease Ontology)
UMLS_CUI:C0002874
HPO Human Phenotype ID (Human Phenotype Ontology)
HP:0001915