Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
Study of CTX001 in Patients with Severe Sickle Cell Disease (SCD)
Sponsor: CRISPR Therapeutics AG
Enrolling: Male and Female Patients
IRB Number: AAAR9004
U.S. Govt. ID: NCT03745287
Contact: Research Nurse Navigator: 212-342-5162 /
Additional Study Information: This study is being done to learn more about the safety and effects of CTX001 (the Study Product) in patients with severe Sickle Cell Disease (SCD). The Study Product is considered investigational; investigational means the Study Product is not approved by the United States Food and Drug Administration (FDA). The goal of this study is to see if a single dose of the Study Product allows your body to increase the amount of a certain kind of hemoglobin (called hemoglobin F or abbreviated as HbF) while decreasing the effects of SCD.
This study is closed
Markus Mapara, MD
Do You Qualify?
Are you 18 to 35 years old? Yes No
Do you have severe sickle cell disease? Yes No
Have you been hospitalized or gone to the emergency room for your pain? Yes No
Have you been told you were eligible for a stem cell transplant? Yes No
You may be eligible for this study

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