- enrolling by invitationPHASE3
A Long-term Follow-up Study in Participants Who Received CTX001
This study is looking at the long-term safety and how well a treatment called CTX001 works for children and adults with serious blood conditions like beta-thalassemia and sickle cell disease. It's for people who have already had CTX001 in a previous study.
United States · Belgium · Canada - active not recruitingPHASE3
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
This study is testing a new 'gene-editing' treatment called CTX001 for children aged 2-11 with a blood condition called beta-thalassemia. It aims to see how safe and effective this single treatment is, hoping to reduce their need for regular blood transfusions.
United States · Canada · Germany - recruitingPHASE3
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
This study is testing a new medicine, Etavopivat, for sickle cell disease and thalassaemia. It looks at how safe and effective the treatment is over a long period for people already benefiting from it in previous studies. The goal is to improve oxygen delivery in the body.
United States · Canada · Egypt - active not recruitingPHASE2
A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia
This study looks at a new medicine called AG-348 for adults with a type of thalassaemia that doesn't need regular blood transfusions. It checks if the medicine is safe and effective in improving their condition over time.
United States · Canada · United Kingdom - active not recruitingPHASE4
CINC424A2X01B Rollover Protocol
This study is for patients already taking ruxolitinib, or ruxolitinib with panobinostat, in a previous trial, and who are still benefiting. It lets them continue their treatment and helps researchers learn more about the long-term safety of these medications.
Australia · Belgium · Bulgaria