Sickle Cell Disease Clinical Studies

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A Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Subjects With Severe Sickle Cell Disease

The purpose of this research study is to learn more about the safety and effects of CTX001 (the “Study Product”) in patients ages 12 to 35 years who have been diagnosed with severe sickle cell disease. This is an open-label study in which subjects will receive one dose of the Study Product. The goal of is to see if a single “dose” of the Study Product allows the body to increase the amount of hemoglobin F (HbF) while decreasing the effects of SCD. CTX001, which is created by changing (editing) the DNA in the subjects blood stem cells near a gene called BCL11A

BMT CTN 1503 (STRIDE2): BMT for SCD

The purpose of this research study is to compare transplant (HCT/BMT) to standard of care treatment in young adults with SCD who have experienced complications. Once consented we would assess to see if you have a matched donor. Subjects who have a closely matched donor will undergo a bone marrow transplant. This is called the Donor Arm. Subjects who do not have a closely matched donor will receive the current standard of care for their disease. This is called the No Donor Arm. We will follow subjects in both arms for two years to evaluate outcomes over that time period.
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