- 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 - 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 - 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 - enrolling by invitationPHASE4
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
This study allows patients who are already benefiting from a drug called mitapivat in a previous trial to continue taking it. It's for those with conditions like Pyruvate Kinase Deficiency, Sickle Cell Disease, or Thalassemia, to ensure they can keep receiving the treatment if their doctor believes it's helping them.
Belgium · Brazil · Canada - 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 - 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