Bone Marrow Failure
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-02-06Clinical Trial Update: Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure [Phase 1]
Clinical trial update announcing a Phase 1 study of EXG34217 in patients with telomere biology disorders and bone marrow failure, currently …
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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 · 2025-12-22Clinical Trial Update: Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study
A clinical trial update describing a secondary research study that analyzes molecular and clinical aspects of bone marrow failure and VEXAS …
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CLINICAL_TRIALS trial_result · 2025-12-03Clinical Trial Update: The Use of Eltrombopag Post HSCT in BMFD [Phase 2, Phase 3]
A Phase 2/3 clinical trial evaluating eltrombopag after hematopoietic stem cell transplantation for bone marrow failure has completed.
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CLINICAL_TRIALS research · 2025-11-28Clinical Trial Update: Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures [Phase 2]
Phase 2 clinical trial update on regenerative medicine to restore hematopoiesis and immune function in immunodeficiencies and inherited bone…
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CLINICAL_TRIALS research · 2025-08-14Clinical Trial Update: CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation [Phase 1, Phase 2]
This document provides an update on a phase 1/2 clinical trial evaluating CD34+ stem cell selection for patients receiving allogeneic stem c…
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CLINICAL_TRIALS research · 2025-05-22Clinical Trial Update: Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC
This clinical trial update describes an expanded access protocol using alpha/beta T and CD19+ depleted peripheral blood stem cells. It targe…
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CLINICAL_TRIALS trial_result · 2025-04-13Clinical Trial Update: A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF) [Phase 1]
Phase 1 clinical trial update for CK0801 in patients with bone marrow failure; the study is currently active but not recruiting.
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CLINICAL_TRIALS research · 2024-12-31Clinical Trial Update: Avatrombopag for Platelet Recovery Post-UCBT in Patients With Bone Marrow Failure Disease
This clinical trial is recruiting to evaluate avatrombopag for promoting platelet recovery after umbilical cord blood transplantation in pat…
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CLINICAL_TRIALS trial_result · 2024-10-31Clinical Trial Update: Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases [Phase 2, Phase 3]
Clinical trial update reports Phase 2 and Phase 3 results for risk-adapted allogeneic stem cell transplantation in non-malignant diseases wi…
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CLINICAL_TRIALS trial_result · 2024-07-30Clinical Trial Update: TPO-Mimetic Use in Children for Hematopoietic Failure [EARLY_Phase 1]
An early-phase 1 pediatric trial evaluating thrombopoietin mimetics for hematopoietic failure has completed. The study enrolled children wit…
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CLINICAL_TRIALS trial_result · 2019-10-22Clinical Trial Update: CD34+ Stem Cell Infusion to Augment Graft Function [Phase 2]
Phase 2 trial investigating CD34+ stem cell infusion to augment graft function; the study was terminated with results posted in 2019.
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CLINICAL_TRIALS trial_result · 2018-08-21Clinical Trial Update: CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant [Phase 2]
Phase 2 clinical trial update on CD34+ selection for partially matched family or matched unrelated adult donor transplants in leukemia and l…
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CLINICAL_TRIALS withdrawal · 2018-07-26Clinical Trial Update: Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study [NA]
A pilot clinical trial on patient-driven transfusion thresholds in hematological disorders sponsored by Emory University has been withdrawn.
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CLINICAL_TRIALS trial_result · 2013-08-12Clinical Trial Update: Alemtuzumab and Low-Dose Cyclosporine in Treating Patients With Severe Aplastic Anemia or Acquired Marrow Failure [Phase 2]
Phase 2 clinical trial update evaluating alemtuzumab with low-dose cyclosporine in patients with severe aplastic anemia or acquired marrow f…
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CLINICAL_TRIALS trial_result · 2013-01-03Clinical Trial Update: Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013) [Phase 2]
Phase 2 trial evaluating the safety and efficacy of combining levamisole with cyclosporine A in patients with paroxysmal nocturnal hemoglobi…