Alpha-thalassemia
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Screening for Alpha Thalassemia in Healthy Volunteers
Clinical trial update indicating alpha thalassemia screening in healthy volunteers; the study is currently active but not recruiting.
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
NIAID-sponsored clinical trial update indicates recruitment is ongoing to collect human biospecimens for basic and clinical research into gl…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) [Phase 3]
A Phase 3 pediatric study evaluating mitapivat for transfusion-dependent alpha- and beta-thalassemia to assess efficacy, pharmacokinetics, a…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia [Phase 3]
This clinical trial update describes a Phase 3 study to evaluate the efficacy, pharmacokinetics, and safety of mitapivat in pediatric partic…
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CLINICAL_TRIALS research · 2025-04-04Clinical Trial Update: International Registry of Patients With Alpha Thalassemia
University of California, San Francisco is sponsoring an international registry (NCT04872179) to collect data from patients with alpha-thala…
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CLINICAL_TRIALS research · 2024-11-14Clinical Trial Update: FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a longitudinal epidemiology study by xCures to follow outcomes across multiple rare diseases, and the study is currently recruitin…
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CLINICAL_TRIALS research · 2024-09-19Clinical Trial Update: Genetic Profile of Alpha Thalassemia Children at Sohag University Hospital .
Clinical trial investigating the genetic profile of Alpha-thalassemia in children at Sohag University Hospital; currently recruiting.
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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…