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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

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
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
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
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