Phase 3 Sickle Cell Disease Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• You have been diagnosed with transfusion-dependent beta-thalassemia (TDT) or severe sickle cell disease (SCD), and your doctor believes you are a suitable candidate for a stem cell transplant using your own stem cells.
• (For TDT patients) You have a confirmed diagnosis of a specific type of beta-thalassemia (including beta-thalassemia/hemoglobin E), supported by genetic testing.
• (For TDT patients) You have needed large amounts of red blood cell transfusions — at least 100 mL per kg of body weight per year, or at least 10 units per year — during the two years before joining the trial.
• (For SCD patients) You have a confirmed diagnosis of sickle cell disease based on genetic testing.
• (For SCD patients) You have had at least two serious pain crises (called vaso-occlusive crises) per year over the two years before joining the trial.

Who may not be able to join:

• You have a close family member who is a healthy, well-matched stem cell donor (a 10 out of 10 HLA-matched related donor) and is willing to donate.
• You have previously had a stem cell transplant.
• You currently have a serious active infection, whether bacterial, viral, fungal, or parasitic, as judged by your doctor.
• (For TDT patients) You have a related condition called alpha-thalassemia with more than one alpha gene deletion, or extra copies of the alpha gene.
• (For TDT patients) You have a specific variant called sickle cell beta-thalassemia.
• (For SCD patients) You have a brain blood vessel condition called moyamoya syndrome that has not been treated, or it is found at your initial screening (confirm with trial site).

Important: Always verify eligibility with the trial site directly before applying.