National Heart, Lung, and Blood Institute (NHLBI): Anemia, Aplastic trials

The registry lists 39 Anemia, Aplastic studies with National Heart, Lung, and Blood Institute (NHLBI) as lead sponsor. 6 are open to participants now and 18 have results posted.

Phase 1 9Phase 2 27Phase 3 1No phase 9(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research StudyEnrolling by invitationNCT07102849
• Natural History of Acquired and Inherited Bone Marrow Failure SyndromesRecruitingNCT05012111
• Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)RecruitingNCT04304820Phase 2Results posted
• Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD ProphylaxisRecruitingNCT03520647Phase 2
• Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant OutcomeRecruitingNCT03173937Phase 1/2
• Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cellsRecruitingNCT01174108Phase 2
• JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow FailureActive, not recruitingNCT05998408Phase 1/2Results posted
• Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan AnemiaCompletedNCT05828108Phase 1/2Results posted
• Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive TherapyActive, not recruitingNCT02979873Phase 2Results posted
• Extended Dosing With Eltrombopag for Severe Aplastic AnemiaCompletedNCT01891994Phase 2Results posted
• Eltrombopag With Standard Immunosuppression for Severe Aplastic AnemiaActive, not recruitingNCT01623167Phase 1/2Results posted
• Danazol for Genetic Bone Marrow and Lung DisordersCompletedNCT01441037Phase 1/2Results posted
• Eltrombopag for Moderate Aplastic AnemiaCompletedNCT01328587Phase 2Results posted
• Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic AnemiaCompletedNCT01193283Phase 1/2Results posted
• A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic AnemiaCompletedNCT01187017Phase 1/2Results posted
• Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA TreatmentCompletedNCT00944749Phase 2Results posted
• A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia PatientsCompletedNCT00922883Phase 2Results posted
• Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic SyndromeCompletedNCT00604201Phase 2Results posted
• Three Immunosuppressive Treatment Regimens for Severe Aplastic AnemiaCompletedNCT00260689Phase 2Results posted
• Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan AnemiaCompletedNCT00229619Phase 2Results posted
• Alemtuzumab to Treat Severe Aplastic AnemiaCompletedNCT00195624Phase 2Results posted
• Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic AnemiaCompletedNCT00065260Phase 2Results posted
• A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow FailureTerminatedNCT00001962Phase 2Results posted
• Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive TherapyCompletedNCT04436380
• Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chromosome 7 Abnormalities After Immunosuppressive TherapyCompletedNCT04436367
• Low-Dose Danazol for the Treatment of Telomere Related DiseasesCompletedNCT03312400Phase 2
• Neuropsychological Effects of Immunosuppressive Treatment in Subjects With Aplastic AnemiaCompletedNCT00767650
• Improving Immunosuppressive Treatment for Patients With Severe Aplastic AnemiaCompletedNCT00061360Phase 2
• Collection of Biological Samples From Patients With Hepatitis-Associated Aplastic AnemiaCompletedNCT00050115
• Stem Cell Mobilization Potential in Patients With Aplastic Anemia in RemissionCompletedNCT00011830Phase 1
• Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic AnemiaCompletedNCT00005935Phase 2
• Aplastic Anemia Epidemiology: Incidence and Case-controlCompletedNCT00005682
• Etiology of Blood Dyscrasias: Analysis of the International Agranulocytosis and Aplastic Anemia Study DataCompletedNCT00005307
• Drug Etiology of Aplastic Anemia and Related DyscrasiasCompletedNCT00005302
• Combination Therapy of Severe Aplastic AnemiaCompletedNCT00001964Phase 2
• Comparing Therapies for the Treatment of Severe Aplastic AnemiaCompletedNCT00001626Phase 2
• Stem Cell Factor Medication for Aplastic AnemiaCompletedNCT00001398Phase 1
• Cord Blood Stem Cell Transplantation Study (COBLT)CompletedNCT00000603Phase 2
• Multi-Center Trial of Anti-Thymocyte Globulin in Treatment of Aplastic Anemia and Other Hematologic DisordersCompletedNCT00000597Phase 3