Hemoglobinopathy
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-03-25Clinical Trial Update: Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases [Phase 1, Phase 2]
Clinical trial update for a conditioning regimen using Campath, fludarabine, and melphalan for non-malignant diseases; currently recruiting …
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CLINICAL_TRIALS research · 2026-02-24Clinical Trial Update: Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling [EARLY_Phase 1]
This early-phase clinical trial aims to determine red blood cell survival in sickle cell disease and other hemoglobinopathies using biotin l…
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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 · 2026-01-28Clinical Trial Update: A Long-term Follow-up Study in Patients Who Received BEAM-101
A long-term follow-up study of patients who received BEAM-101 is ongoing, with the trial currently recruiting to assess long-term outcomes i…
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CLINICAL_TRIALS trial_result · 2025-12-26Clinical Trial Update: Prevalence of Abnormal Haemoglobin Variants in Metropolitan Chattogram, Bangladesh
A completed clinical trial from Bangladesh assesses how common abnormal haemoglobin variants are in metropolitan Chattogram, focusing on HbE…
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CLINICAL_TRIALS research · 2025-11-10Clinical Trial Update: Discarded Bone Marrow for Hematology Research
Clinical trial update notes that discarded bone marrow is being used for hematology research under St. Jude Children's Research Hospital, in…
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CLINICAL_TRIALS trial_result · 2025-10-06Clinical Trial Update: Prevalence of Hemoglobinopathies Associated With Significant Hb Variants in the Chattogram Region of Bangladesh
A completed clinical trial in the Chattogram region of Bangladesh assessed the prevalence of hemoglobinopathies and their association with s…
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CLINICAL_TRIALS research · 2025-10-03Clinical Trial Update: Integrative Diagnosis for SCD and Other RADs
An ongoing clinical trial focused on integrative diagnosis for sickle cell disease and related red blood cell disorders, currently recruitin…
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CLINICAL_TRIALS research · 2025-08-07Clinical Trial Update: Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Cell Transplant or Gene Therapy
Long-term follow-up study to assess outcomes in individuals with hemoglobin disorders after hematopoietic cell transplantation or gene thera…
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CLINICAL_TRIALS research · 2025-07-25Clinical Trial Update: A Phase I Study of SNH-119014 in Healthy Volunteers [Phase 1]
Phase I clinical trial update for SNH-119014 in healthy volunteers recruiting for a study on hemoglobinopathy.
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CLINICAL_TRIALS research · 2025-06-24Clinical Trial Update: TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children [Phase 2]
A Phase 2 clinical trial at Johns Hopkins All Children's Hospital is evaluating TCR alpha-beta T-cell-depleted haploidentical hematopoietic …
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CLINICAL_TRIALS research · 2025-02-18Clinical Trial Update: ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19
This clinical trials registry update reports completion of the ERN-EuroBloodNet registry documenting patients with rare red blood cell defec…
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CLINICAL_TRIALS trial_result · 2025-01-31Clinical Trial Update: A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) [Phase 1, Phase 2]
This document provides an update on RUBY, a Phase 1/2 clinical trial evaluating EDIT-301 for severe sickle cell disease, focusing on safety …
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CLINICAL_TRIALS trial_result · 2025-01-27Clinical Trial Update: In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM) [Phase 1]
Phase 1 in utero hematopoietic stem cell transplantation for Alpha-thalassemia major has been terminated.
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CLINICAL_TRIALS research · 2024-08-21Clinical Trial Update: Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation [NA]
This ClinicalTrials.gov update reports a completed feasibility study evaluating an adapted physical activity program for children, adolescen…
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CLINICAL_TRIALS research · 2024-06-03Clinical Trial Update: Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease [EARLY_Phase 1]
An early Phase 1 trial evaluating the safety and potential efficacy of RM-004 cell therapy for Alpha-thalassemia with Hemoglobin H disease d…
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CLINICAL_TRIALS trial_result · 2018-03-16Clinical Trial Update: Lucky Iron Fish Home Fortification of Iron [NA]
A clinical trial evaluated home fortification of iron with the Lucky Iron Fish for anemia and iron-deficiency anemia, and the study status i…
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CLINICAL_TRIALS withdrawal · 2017-07-26Clinical Trial Update: Comparing CGM and OGTT in Relation to Iron Overload Detected by Pancreas T2* MRI in High-Risk Hematology Group
A clinical trial comparing continuous glucose monitoring (CGM) and oral glucose tolerance testing (OGTT) in relation to pancreas T2* MRI-det…
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CLINICAL_TRIALS trial_result · 2016-08-18Clinical Trial Update: A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen [NA]
A pilot hematopoietic stem cell transplant study using a nonmyeloablative preparative regimen in patients with high-risk hemoglobinopathies,…
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CLINICAL_TRIALS trial_result · 2013-11-26Clinical Trial Update: Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis [Phase 2]
Phase 2 trial evaluating subcutaneous desferrioxamine for transfusional iron overload has completed, including patients with beta-thalassemi…
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CLINICAL_TRIALS trial_result · 2008-03-04Clinical Trial Update: Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia) [Phase 2]
A Phase 2 clinical trial evaluating hydroxyurea for beta-thalassemia (Cooley's anemia) has completed.