A 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
A Study to Evaluate the Safety of a Dose of Autologous CRISPR-Cas9 Human Stem Cells in Subjects With Severe Sickle Cell Disease
Sponsor: Vertex Pharmaceuticals & CRISPR Therapeutics AG
Enrolling: Male and Female Patients
IRB Number: AAAR8989
U.S. Govt. ID: NCT03745287
Contact: Desmarie Sherwood: 212-305-7213 / ds3851@cumc.columbia.edu
Additional Study Information: This study is being done to learn more about the safety and effects of CTX001 in patients with severe SCD. CTX001 is created by changing (editing) the DNA in your own blood stem cells near a gene called BCL11A which is stored in a solution called dimethyl sulfoxide (DMSO) to try to prevent cells from dying while they are frozen. BCL11A is the area in the gene that stops the blood cell from making Hemoglobin F, the normal type of hemoglobin that can carry oxygen. Editing this genemay stop it from working, which may lead to your blood cell to produce more Hemoglobin F. This editing is a permanent change to your stem cell DNA and is not reversible, meaning it cannot be changed back once it is made.
This study is closed
Monica Bhatia, MD
Do You Qualify?
Are you between 18 to 21 years of age (inclusive at time of informed consent)? Yes No
Do you have a documented S/S genotype? Yes No
Do you have severe Sickle Cell Disease? Yes No
Are you willing to participate in a long term follow up study? Yes No
You may be eligible for this study

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For more information, please contact:
Desmarie Sherwood