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Safety and efficacy assessments clinical trials
2 indexed studies · 1 currently recruiting
- Active not recruiting
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
This study is looking at the long-term effects of a gene therapy treatment for people with a condition called beta-thalassemia. It follows patients who have already received the treatment in earlier studies to see how they are doing over many years, checking their safety and how well the treatment continues to work.
For: Transfusion-dependent Beta-ThalassemiaUnited States · Australia · France - Enrolling by invitation
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
This study is checking on people with sickle cell disease or a type of thalassaemia called transfusion-dependent beta-thalassaemia. They all previously received a special treatment called EDIT-301. We want to see how safe and effective this treatment has been for them over a long time.
For: Sickle Cell Disease · Transfusion-dependent Beta-Thalassemia · HemoglobinopathiesUnited States · Canada