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Results for “Non-transfusion Dependent Thalassemia”

32 trials

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Showing 20 of 32 results

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 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)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
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
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
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)Looking for participantsNCT05664737
What this trial is testing

Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia

Who this might be right for
Anemia
Bristol-Myers Squibb 189
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 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)Looking for participantsNCT06302491
What this trial is testing

Safety and Efficiency of AND017 in Patients With β-thalassemia

Who this might be right for
β -Thalassemia
Kind Pharmaceuticals LLC 64
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)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
Early research (Phase 1)Study completedNCT02950857
What this trial is testing

EPEG in Beta Thalassemia Patients

Who this might be right for
Beta-Thalassemia
Prolong Pharmaceuticals 6
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
Early research (Phase 1)Looking for participantsNCT06772766
What this trial is testing

A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia

Who this might be right for
Beta-Thalassemia
Mabwell (Shanghai) Bioscience Co., Ltd. 40
Testing effectiveness (Phase 2)Ended earlyNCT04059406
What this trial is testing

Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)

Who this might be right for
Beta Thalassemia Intermedia
Ionis Pharmaceuticals, Inc. 29
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
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
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
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
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