Aplastic Anemia
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure [Phase 1, Phase 2]
A NHLBI-sponsored Phase 1/2 trial is evaluating ruxolitinib, a JAK1/2 inhibitor, for relapsed/refractory immune bone marrow failure. The stu…
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis [Phase 2]
Phase 2 clinical trial evaluating haplo-identical transplantation with post-transplant cyclophosphamide for GVHD prophylaxis in severe aplas…
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: Enarodustat + CsA vs CsA in Newly Diagnosed TD-NSAA [Phase 3]
A Phase 3 trial is being planned to compare Enarodustat plus cyclosporine against cyclosporine alone in newly diagnosed transfusion-dependen…
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia [Phase 2]
A Phase 2 clinical trial evaluating haploidentical bone marrow transplant with post-transplant cyclophosphamide for severe aplastic anemia, …
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
This ClinicalTrials.gov entry describes a study on the natural history of acquired and inherited bone marrow failure syndromes and is curren…
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA [Phase 2]
A Phase 2 clinical trial compares romiplostim to recombinant human thrombopoietin (rhTPO) for platelet engraftment after transplantation in …
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT [Phase 1, Phase 2]
A Phase 1/2 clinical trial is evaluating CD45RA-depleted stem cell addback to prevent viral or fungal infections following TCRab/CD19-deplet…
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: 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 [Phase 2]
Phase 2 clinical trial evaluating a combination of methylprednisolone, horse anti-thymocyte globulin, cyclosporine, and growth factors (filg…
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced AA [Phase 2]
This entry notes a Phase 2 clinical trial of Romiplostim N01 for refractory aplastic anemia induced by chemoradiotherapy, with status not ye…
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
A multicenter protocol to improve access and distribution of unlicensed cryopreserved cord blood units, with the study currently recruiting.…
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: Enarodustat+CsA vs CsA in the Treatment of Newly-diagnosed TD-NSAA [Phase 3]
Phase 3 trial evaluating Enarodustat plus cyclosporine versus cyclosporine alone for newly diagnosed transfusion-dependent non-severe aplast…
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: 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 Outcome [Phase 1, Phase 2]
Phase 1/2 clinical trial evaluating unrelated umbilical cord blood transplantation using CordIn ex vivo expanded cells to accelerate engraft…
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: CK0801 for Treatment of Aplastic Anemia [Phase 2]
This clinical trial update indicates CK0801 for the treatment of aplastic anemia is in Phase 2 and not yet recruiting.
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CLINICAL_TRIALS research · 2026-03-30Clinical Trial Update: A Multicenter, Randomized, Open-Label Study of Haplo-Cord HSCT for the Treatment of Aplastic Anemia [NA]
A multicenter randomized open-label study to evaluate Haplo-Cord hematopoietic stem cell transplantation for aplastic anemia; the trial is c…
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CLINICAL_TRIALS research · 2026-03-12Clinical Trial Update: Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia [Phase 2]
Phase 2 clinical trial evaluating a ruxolitinib-based graft-versus-host disease prophylaxis regimen before, during, and after hematopoietic …
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CLINICAL_TRIALS research · 2026-03-10Clinical Trial Update: Adverse Effects of ATG/ALG Therapy in Aplastic Anemia
An ongoing clinical trial in China is evaluating the adverse effects of anti-thymocyte globulin and anti-lymphocyte globulin therapy for apl…
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CLINICAL_TRIALS research · 2026-02-19Clinical Trial Update: Revolade Tablets Specified Drug-use Survey
A clinical trial update indicates a Revolade (eltrombopag) tablet study for aplastic anemia is currently recruiting.
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202) [Phase 3]
Phase 3 TransIT trial comparing unrelated donor bone marrow transplant with immunosuppressive therapy for pediatric and young adult patients…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia [Phase 2]
A Phase 2 clinical trial evaluating upfront haploidentical or unrelated donor bone marrow transplantation to restore normal hematopoiesis in…
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CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: Phase I Clinical Study of Haplo-HSCT Combined With Hypoxic 3D-Cultured Umbilical Cord MSC for the Treatment of SAA [Phase 1, Phase 2]
Phase 1/2 clinical trial enrolling by invitation to evaluate haploidentical HSCT combined with hypoxic 3D-cultured umbilical cord MSCs for t…
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CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: National Longitudinal Cohort of Hematological Diseases
Recruiting for a national longitudinal cohort study of hematological diseases sponsored by the Institute of Hematology & Blood Diseases Hosp…
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CLINICAL_TRIALS research · 2026-01-30Clinical Trial Update: Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders [Phase 2]
Phase 2 clinical trial update investigating alpha/beta T cell receptor alpha/beta-depleted hematopoietic cell transplantation in inherited b…
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CLINICAL_TRIALS research · 2026-01-29Clinical Trial Update: The HOPE Biobank Resource (BMT CTN 2402 HOPE)
This update announces the HOPE Biobank Resource for BMT CTN 2402, which is currently not yet recruiting and will support research in aplasti…
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CLINICAL_TRIALS research · 2026-01-09Clinical Trial Update: Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA) [Phase 2]
Phase 2 trial evaluating early initiation of oral cyclosporine and eltrombopag in treatment-naive severe aplastic anemia; recruitment is ong…
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CLINICAL_TRIALS research · 2026-01-08Clinical Trial Update: 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-cells [Phase 2]
This Phase 2 clinical trial evaluates allogeneic hematopoietic stem cell transplantation using G-CSF mobilized CD34+ selected cells co-infus…