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Results for “Hemoglobinopathies (Transfusion-dependent β-thalassemia and Sickle Cell Disease)”

5 trials

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Showing 5 of 5 results

Testing effectiveness (Phase 2)Study completedNCT02151526
What this trial is testing

A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease

Who this might be right for
Beta-Thalassemia MajorSickle Cell Disease
Genetix Biotherapeutics Inc. 7
Very early researchNot Yet RecruitingNCT07190001
What this trial is testing

YOLT-204 in Patients With Hemoglobinopathies

Who this might be right for
Hemoglobinopathies (Transfusion-dependent β-thalassemia and Sickle Cell Disease)
Guangzhou Women and Children's Medical Center 18
Early research (Phase 1)Looking for participantsNCT06647979
What this trial is testing

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

Who this might be right for
Sickle Cell DiseaseSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)Beta-Thalassemia+1 more
Daniel Bauer 10
Not applicableEnrolling By InvitationNCT06363760
What this trial is testing

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

Who this might be right for
Sickle Cell DiseaseTransfusion-dependent Beta-ThalassemiaHemoglobinopathies
Editas Medicine, Inc. 54
Large-scale testing (Phase 3)Looking for participantsNCT05477563
What this trial is testing

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Who this might be right for
Beta-ThalassemiaThalassemiaHematologic Diseases+4 more
Vertex Pharmaceuticals Incorporated 26