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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

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

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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
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