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Results for “Alpha and Beta Thalassemia”

25 trials

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Showing 20 of 25 results

Large-scale testing (Phase 3)Not Yet RecruitingNCT07517133
What this trial is testing

Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia

Who this might be right for
Non-Transfusion-dependent Alpha-ThalassemiaNon-Transfusion-dependent Beta-Thalassemia
Agios Pharmaceuticals, Inc. 45
Testing effectiveness (Phase 2)Looking for participantsNCT01962415
What this trial is testing

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more
Paul Szabolcs 100
Testing effectiveness (Phase 2)WithdrawnNCT01419704
What this trial is testing

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

Who this might be right for
Anemia, Sickle CellComplex and Transfusion-dependent HemoglobinopathiesThalassemia+4 more
Talaris Therapeutics Inc.
Early research (Phase 1)Study completedNCT04718844
What this trial is testing

A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome

Who this might be right for
Non-transfusion-dependent ThalassemiaLow Risk Myelodysplastic SyndromeVery-Low Risk Myelodysplastic Syndrome
Silence Therapeutics plc 44
Not applicableLooking for participantsNCT06213402
What this trial is testing

RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)

Who this might be right for
Sickle Cell DiseaseThalassemiaHemolytic; Anemia, Hereditary, Due to Enzyme Disorder+6 more
Hospital Universitari Vall d'Hebron Research Institute 32,564
Large-scale testing (Phase 3)Not Yet RecruitingNCT07157722
What this trial is testing

Evaluating the Effect of N-Acetyl Cysteine and Alpha Lipoic Acid in Patients With Beta Thalassemia

Who this might be right for
Beta Thalassemia
Tanta University 66
Not applicableLooking for participantsNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Large-scale testing (Phase 3)Active Not RecruitingNCT04770779
What this trial is testing

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

Who this might be right for
Transfusion-dependent Alpha-ThalassemiaTransfusion-dependent Beta-Thalassemia
Agios Pharmaceuticals, Inc. 258
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Not applicableStudy completedNCT06831799
What this trial is testing

ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19

Who this might be right for
HemoglobinopathiesSickle Cell DiseaseSickle Cell Anemia+21 more
Hospital Universitari Vall d'Hebron Research Institute 684
Not applicableStudy completedNCT03948737
What this trial is testing

The Effect of Alpha-tocopherol in Hemolysis and Oxidative Stress Marker on the Red Cell Membrane Beta-thalassemia Major

Who this might be right for
Beta Thalassemia Major AnemiaHemolysisOxidative Stress
Indonesia University 40
Testing effectiveness (Phase 2)Study completedNCT00873041
What this trial is testing

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study

Who this might be right for
Non-transfusion Dependent Thalassemia
Novartis Pharmaceuticals 166
Not applicableEnded earlyNCT02179359
What this trial is testing

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Who this might be right for
Sickle Cell DiseaseTransfusion Dependent Alpha- or Beta- ThalassemiaDiamond Blackfan Anemia+5 more
Masonic Cancer Center, University of Minnesota 38
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableLooking for participantsNCT04872179
What this trial is testing

International Registry of Patients With Alpha Thalassemia

Who this might be right for
Alpha-ThalassemiaAlpha Thalassemia MajorAlpha Thalassemia Minor
University of California, San Francisco 500
Testing effectiveness (Phase 2)Study completedNCT04987489
What this trial is testing

Etavopivat in Patients With Thalassemia or Sickle Cell Disease

Who this might be right for
Sickle Cell DiseaseThalassemia
Forma Therapeutics, Inc. 53
Not applicableLooking for participantsNCT05635266
What this trial is testing

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

Who this might be right for
Age-Related Macular DegenerationAllergiesAlpha-Gal Syndrome+44 more
Sanguine Biosciences 20,000
Large-scale testing (Phase 3)Active Not RecruitingNCT04770753
What this trial is testing

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

Who this might be right for
Non-Transfusion-dependent Alpha-ThalassemiaNon-Transfusion-dependent Beta-Thalassemia
Agios Pharmaceuticals, Inc. 194
Not applicableLooking for participantsNCT03937817
What this trial is testing

Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

Who this might be right for
Alpha and Beta ThalassemiaSickle Cell DiseaseMalaria+1 more
National Institute of Allergy and Infectious Diseases (NIAID) 300
Not applicableLooking for participantsNCT05799118
What this trial is testing

Study of the Role of Genetic Modifiers in Hemoglobinopathies

Who this might be right for
Sickle Cell DiseaseThalassemia, BetaThalassemia Alpha+1 more
Cyprus Institute of Neurology and Genetics 30,000
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