transfusion-dependent beta-thalassemia research hub
4 indexed studies · 1 currently recruiting
- authorisedTherapeutic exploratory (Phase II)
A single arm, open label, multicenter, single-dose, phase 2b clinical study evaluating efficacy and safety of gene therapy using autologous CD34+ hematopoietic stem cells transduced with the GLOBE lentiviral vector using an improved transduction protocol in subjects with transfusion-dependent beta-thalassemia.
This study is testing a new gene therapy for adults with beta-thalassemia who need regular blood transfusions. The main goal is to see if this treatment can help people stop needing transfusions for at least a year.
Italy - 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.
United States · Canada - active not recruitingPHASE3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
This study looks at a new medicine called mitapivat for people with severe types of thalassemia who need regular blood transfusions. It aims to see if mitapivat can reduce how often they need transfusions compared to a dummy treatment.
United States · Brazil · Bulgaria - 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.
United States · Australia · France