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Results for “Non-transfusion Dependent Thalassemia”

33 trials

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Showing 20 of 33 results

Early research (Phase 1)WithdrawnNCT04176653
What this trial is testing

Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124

Who this might be right for
Non-transfusion-dependent ThalassemiaLow Risk Myelodysplastic Syndrome
Silence Therapeutics plc
Large-scale testing (Phase 3)Study completedNCT02906202
What this trial is testing

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

Who this might be right for
Beta-Thalassemia
Genetix Biotherapeutics Inc. 24
Large-scale testing (Phase 3)Study completedNCT07210450
What this trial is testing

Effect of L-Glutamine on Pulmonary Artery Pressure in Patients With Non-Transfusion-Dependent Thalassemia

Who this might be right for
Pulmonary Artery PressureThalassemiaNon-transfusion Dependent Thalassemia
Mazandaran University of Medical Sciences 8
Testing effectiveness (Phase 2)Enrolling By InvitationNCT04432623
What this trial is testing

The BENeFiTS Trial in Beta Thalassemia Intermedia

Who this might be right for
Beta Thalassemia IntermediaSickle Cell Disease
Phoenicia BioScience 36
Not applicableStudy completedNCT00872339
What this trial is testing

Assessment of Pain in People With Thalassemia

Who this might be right for
Thalassemia
Carelon Research 252
Testing effectiveness (Phase 2)Study completedNCT04364269
What this trial is testing

Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia

Who this might be right for
Beta-ThalassemiaNon-transfusion-dependent Thalassemia
Vifor (International) Inc. 35
Testing effectiveness (Phase 2)Study completedNCT02993224
What this trial is testing

Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet

Who this might be right for
Transfusion-dependent ThalassemiaNon-transfusion-dependent Thalassemia
Novartis Pharmaceuticals 148
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
Not applicableEnded earlyNCT00578292
What this trial is testing

Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO

Who this might be right for
Thalassemia
Baylor College of Medicine 10
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
Testing effectiveness (Phase 2)Study completedNCT03342404
What this trial is testing

Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia

Who this might be right for
Thalassemia
Celgene 145
Not applicableStudy completedNCT02626689
What this trial is testing

To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care

Who this might be right for
Beta-Thalassemia
Celgene 100
Testing effectiveness (Phase 2)Study completedNCT04054921
What this trial is testing

Safety Study for Beta Thalassemia Subjects on PTG-300

Who this might be right for
β-thalassemiaIneffective Erythropoiesis
Protagonist Therapeutics, Inc. 34
Large-scale testing (Phase 3)Study completedNCT01825512
What this trial is testing

Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients

Who this might be right for
Chronic Iron Overload
Consorzio per Valutazioni Biologiche e Farmacologiche 435
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
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
Testing effectiveness (Phase 2)Study completedNCT03271541
What this trial is testing

Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia

Who this might be right for
Beta-Thalassemia
Hoffmann-La Roche 12
Testing effectiveness (Phase 2)Looking for participantsNCT06421636
What this trial is testing

Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body

Who this might be right for
Non-Transfusion Dependent Beta-Thalassemia (NTDT)
Regeneron Pharmaceuticals 95
Large-scale testing (Phase 3)Study completedNCT00176852
What this trial is testing

Stem Cell Transplant for Hemoglobinopathy

Who this might be right for
Sickle Cell DiseaseThalassemiaSevere Congenital Neutropenia+2 more
Masonic Cancer Center, University of Minnesota 22
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
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