Diamond-blackfan Anemia
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-06-30Clinical Trial Results: Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia [Phase 1, Phase 2]
Phase 1/2 study results of bitopertin, a selective GlyT1 inhibitor, in patients with steroid-refractory Diamond-Blackfan Anemia have been co…
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CLINICAL_TRIALS trial_result · 2026-04-20Clinical Trial Update: Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia [Phase 1]
Phase 1 clinical trial evaluating the safety, tolerability, and preliminary efficacy of APR-2020 in children and adolescents with RPS19-defi…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: Diamond Blackfan Anemia Registry (DBAR)
The Diamond Blackfan Anemia Registry (DBAR) is an active recruiting clinical registry. It is sponsored by Northwell Health.
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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 · 2026-03-09Clinical Trial Update: Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) [Phase 2]
This update reports the completion of a Phase 2 study evaluating a treosulfan-based conditioning regimen before bone marrow or blood stem ce…
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CLINICAL_TRIALS trial_result · 2026-02-17Clinical Trial Update: Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating bitopertin, a selective GlyT1 inhibitor, for steroid-refractory Diamond-Blackfan Anemia has completed.
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CLINICAL_TRIALS research · 2025-11-04Clinical Trial Update: Investigation of the Genetics of Hematologic Diseases
A St. Jude-sponsored clinical trial update indicates a study on the genetics of hematologic diseases is currently recruiting, covering sever…
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CLINICAL_TRIALS research · 2025-09-22Clinical Trial Update: Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)
This clinical trial update reports on an ongoing study assessing mobilization of CD34+ peripheral blood stem cells in patients with Diamond-…
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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 · 2025-08-12Clinical Trial Update: T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias [Phase 1, Phase 2]
This clinical trials update notes that a T-cell depleted alternative donor bone marrow transplant for sickle cell disease and other anemias …
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CLINICAL_TRIALS research · 2025-08-08Clinical Trial Update: AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion [Phase 2]
A Phase 2 clinical trial evaluating allogeneic stem cell transplantation with alpha/beta T cell and CD19+ B cell depletion for malignant and…
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CLINICAL_TRIALS trial_result · 2025-03-03Clinical Trial Update: Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies [NA]
A hematopoietic stem cell transplant trial for high-risk hemoglobinopathies has been terminated; the conditions studied include sickle cell …
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CLINICAL_TRIALS trial_result · 2023-09-26Clinical Trial Update: Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study [Phase 1, Phase 2]
Long-term follow-up update from BP-004 study (Phase 1/Phase 2) by Bellicum Pharmaceuticals; study status TERMINATED with results posted in 2…
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CLINICAL_TRIALS trial_result · 2023-09-15Clinical Trial Update: Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating conditioning regimens for allogeneic hematopoietic stem-cell transplantation has completed recruitment…
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CLINICAL_TRIALS trial_result · 2023-09-13Clinical Trial Update: Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluated eltrombopag for refractory Diamond-Blackfan anemia; results from the completed study were posted in 202…
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CLINICAL_TRIALS trial_result · 2023-01-04Clinical Trial Update: Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant [Phase 1, Phase 2]
A phase 1/2 safety study evaluating gene-modified donor T-cells after TCRαβ+ depleted stem cell transplant was terminated; results were post…
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CLINICAL_TRIALS trial_result · 2022-12-14Clinical Trial Update: The Use of Trifluoperazine in Transfusion Dependent DBA [Phase 1, Phase 2]
Phase 1/2 trial of trifluoperazine for transfusion-dependent Diamond Blackfan Anemia and Pure Red Cell Aplasia was terminated; results poste…
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CLINICAL_TRIALS trial_result · 2022-12-02Clinical Trial Update: Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia [Phase 1, Phase 2]
Pilot Phase I/II study evaluating amino acid leucine for transfusion-dependent Diamond Blackfan Anemia has completed with results posted.
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CLINICAL_TRIALS trial_result · 2021-12-21Clinical Trial Update: Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia [Phase 1, Phase 2]
A phase 1/2 clinical trial of sotatercept in adults with transfusion-dependent Diamond Blackfan anemia was terminated with posted results.
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CLINICAL_TRIALS trial_result · 2018-08-06Clinical Trial Update: Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs [Phase 2]
Phase 2 trial evaluating allogeneic hematopoietic cell transplantation from matched unrelated donor with reduced-intensity conditioning and …
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CLINICAL_TRIALS trial_result · 2017-11-29Clinical Trial Update: Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer [NA]
ClinicalTrials.gov entry describes termination of a study evaluating donor umbilical cord blood transplantation for various hematologic canc…
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CLINICAL_TRIALS trial_result · 2017-08-22Clinical Trial Update: Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias [Phase 2]
Phase 2 study of deferasirox for iron overload in beta-thalassemia patients who cannot be treated with deferoxamine, as well as related chro…
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CLINICAL_TRIALS trial_result · 2017-02-23Clinical Trial Update: Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload [Phase 4]
Novartis-sponsored Phase 4 trial assessing heart and liver iron load by MRI in patients with transfusion-related iron overload; the study st…
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CLINICAL_TRIALS trial_result · 2017-02-07Clinical Trial Update: Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia [Phase 1]
Phase 1 pilot trial of lenalidomide in adult Diamond-Blackfan anemia patients with transfusion-dependent anemia. The study was terminated an…
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CLINICAL_TRIALS trial_result · 2016-06-17Clinical Trial Update: Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells [Phase 2]
Phase 2 study evaluating related hematopoietic stem cell transplantation for several genetic blood disorders; the trial was terminated and r…