Condition: Sickle Cell Disease · Sponsor: St. Jude Children's Research Hospital
This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD. Secondary Objective * To assess the efficacy autologous infusion of CRISPR/Cas9 genome-edited CD34+ HSPCs into patients with severe SCD.
This description comes directly from the study's public registry record.
Akshay Sharma, MBBS, MSc · 888-226-4343 · referralinfo@stjude.org
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| St. Jude Children's Research Hospital | Memphis, Tennessee, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06506461