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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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 & Criteria

IRB #:
18-015389
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
Study Phase:
Phase I
Phase II
Eligible Age Range:
12 - 35 Years
Gender:
All
Study Categories:
Leader
Stephan A. Grupp

Stephan A. Grupp, MD, PhD