Aplastic Anemia
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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 trial_result · 2026-02-11Clinical Trial Update: A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia [Phase 2]
Completed Phase 2 trial evaluating an optimized standard protocol for severe aplastic anemia.
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CLINICAL_TRIALS trial_result · 2026-02-11Clinical Trial Update: Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia [Phase 2]
A Phase 2 trial evaluating low-dose decitabine for refractory aplastic anemia has been terminated.
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CLINICAL_TRIALS trial_result · 2026-02-11Clinical Trial Update: In Vitro Effect Study of Interleukin-2 Muteins on Regulatory T Cells of Patients With Different Autoimmune, Allo-immune or Inflammatory Diseases [NA]
A completed clinical trial update reporting an in vitro study of interleukin-2 muteins on regulatory T cells in patients with autoimmune, al…
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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 trial_result · 2026-01-16Clinical Trial Update: Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy [Phase 2]
Phase 2 trial evaluating sirolimus (Rapamune) for relapse prevention in people with severe aplastic anemia who previously responded to immun…
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FDA approval · 2026-01-15FDA Approved: ALVAIZ — Supplemental Application (Teva Pharmaceuticals, Inc.)
The FDA approved Teva's supplemental application for ALVAIZ (eltrombopag).
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CLINICAL_TRIALS trial_result · 2026-01-15Clinical Trial Update: A Study of Romiplostim N01 Plus IST vs. Placebo Plus IST for Treatment-Naive Severe Aplastic Anemia [Phase 3]
Phase 3 study comparing Romiplostim N01 plus immunosuppressive therapy versus placebo plus IST in treatment-naive severe aplastic anemia. Th…
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FDA approval · 2026-01-14FDA Approved: ELTROMBOPAG — Original Application (Zydus Pharmaceuticals USA Inc.)
Zydus Pharmaceuticals USA Inc. received FDA approval for an original application of eltrombopag (generic), a thrombopoietin receptor agonist…
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FDA approval · 2026-01-14FDA Approved: ELTROMBOPAG OLAMINE — Original Application (Amneal Pharmaceuticals NY LLC)
FDA approved Amneal Pharmaceuticals NY LLC's eltrombopag olamine as an Original Application (ANDA), making a generic version of eltrombopag …
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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…
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CLINICAL_TRIALS trial_result · 2026-01-05Clinical Trial Update: Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation (BMT CTN 1702) [NA]
A completed clinical trial update reporting long-term outcomes of alternative donor allogeneic transplantation across multiple hematologic d…
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CLINICAL_TRIALS research · 2026-01-05Clinical Trial Update: A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia [Phase 1]
This Phase 1 trial at City of Hope is evaluating a reduced-intensity conditioning regimen of cyclophosphamide, pentostatin, and anti-thymocy…
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CLINICAL_TRIALS trial_result · 2025-12-30Clinical Trial Update: Tolerance of Teeth Brushing During Prolonged Aplasia [Phase 3]
Phase 3 trial completed evaluating tolerability of teeth brushing in patients with prolonged aplasia.
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CLINICAL_TRIALS trial_result · 2025-12-23Clinical Trial Update: Impact of Supportive Care on the Experience of Hospitalization of Patients Staying in the Protected Area of the Department of Blood Diseases [NA]
Completed clinical trial assessing the impact of supportive care on the hospitalization experience for patients with acute leukemia undergoi…
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CLINICAL_TRIALS research · 2025-12-22Clinical Trial Update: Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia [Phase 1, Phase 2]
A Phase 1/2 clinical trial is evaluating the safety and efficacy of a JAK1 inhibitor combined with intensive immunosuppressive therapy for s…
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CLINICAL_TRIALS research · 2025-12-19Clinical Trial Update: UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia [Phase 2]
Phase 2 trial UI-Romi-02 investigates adding romiplostim to standard of care for treatment-naive and relapsed or refractory severe aplastic …
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CLINICAL_TRIALS research · 2025-12-17Clinical Trial Update: A Study of Emapalumab for Pediatric Aplastic Anemia [Phase 2]
A Phase 2 pediatric study evaluating emapalumab for aplastic anemia is currently recruiting, aiming to assess safety and potential efficacy …
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CLINICAL_TRIALS research · 2025-12-17Clinical Trial Update: The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia [Phase 1, Phase 2]
This clinical trial update describes a Phase 1/2 study of daratumumab for patients with refractory aplastic anemia, focusing on safety and e…
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CLINICAL_TRIALS research · 2025-12-04Clinical Trial Update: Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors [Phase 2]
Phase 2 study evaluating exercise training with nicotinamide riboside (NR) supplementation to improve fitness in adolescent/young adult hema…