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

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

Contact

 If you are interested in participating in the study or want to learn more, please get in touch. Contact us
This study is no longer recruiting.

Description
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

Eligibility and criteria
IRB Number:
18-015389
Eligible age range:
12 years - 35 years
Clinical trial phase:
Phase I
Phase II
Official title:
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
Smiling CHOP patient holding shopping bag

We need families like you

Your time and participation make a difference in supporting the work of the Research Institute and children in our community. Learn more about opportunities to participate in our research.