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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
