Aplastic Anemia
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CLINICAL_TRIALS trial_result · 2026-08-10Clinical Trial Results: 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 [Phase 2]
Phase II dose-escalation study evaluating the pharmacokinetics of eltrombopag in pediatric patients with severe or recurrent aplastic anemia…
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CLINICAL_TRIALS trial_result · 2026-04-24Clinical Trial Update: Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF) [Phase 3]
Phase 3 randomized trial evaluating ATG with or without G-CSF in severe aplastic anemia was terminated.
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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 trial_result · 2026-04-24Clinical Trial Update: hATG+CsA vs hATG+CsA+Eltrombopag for SAA [Phase 3]
A Phase 3 clinical trial comparing horse anti-thymocyte globulin plus cyclosporine with and without eltrombopag for severe aplastic anemia h…
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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 trial_result · 2026-04-14Clinical Trial Update: Cancer in Inherited Bone Marrow Failure Syndromes
This clinical trial is currently recruiting to study cancer in inherited bone marrow failure syndromes, including Diamond Blackfan Anemia, D…
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CLINICAL_TRIALS trial_result · 2026-04-13Clinical Trial Update: CsA+EPAG/HPAG+Romiplostim N01 in the Treatment of Newly-diagnosed SAA/TD-NSAA [Phase 2]
This Phase 2 clinical trial update describes a regimen of cyclosporine with EPAG/HPAG and romiplostim for newly diagnosed severe aplastic an…
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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 trial_result · 2026-04-06Clinical Trial Update: Geriatric Optimization Plan to Improve Survival in Older Adult Allogeneic Hematopoietic Cell Transplant Candidates, OTIS Study [NA]
Completed clinical trial evaluating a geriatric optimization plan to improve survival in older adults undergoing allogeneic hematopoietic ce…
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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 trial_result · 2026-03-27Clinical Trial Update: Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita [Phase 2]
Phase 2 clinical trial update describing a radiation- and alkylator-free bone marrow transplantation regimen for patients with Dyskeratosis …
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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 trial_result · 2026-02-19Clinical Trial Update: A Study of the Safety and Effectiveness of Eltrombopag in Combination With Immunosuppressive Therapy (IST) in Korean Pediatric Patients With Treatment Naïve Severe Aplastic Anemia
This completed pediatric trial in Korea studied eltrombopag in combination with immunosuppressive therapy for treatment-naïve severe aplasti…
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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.