St. Jude Autologous Genome Edited Stem Cells For Sickle Cell Disease-1
St. Jude Children's Research Hospital
Summary
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.
Description
Participants will receive a daily subcutaneous (under the skin) dose of motixafortide for up to 3 consecutive days to mobilize their hematopoietic stem and progenitor cells (HSPCs) into peripheral blood. Participants who cannot tolerate motixafortide may receive a daily subcutaneous dose of plerixafor as an alternative for 3-5 consecutive days. The collection of HSPCs will be done via apheresis. The collected HSPCs will be sent to a lab to genetically modify them using CRISPR/Cas9. In the lab, the researchers will take the stem cells and purify them. The stem cells will then be mixed with the…
Eligibility
- Age range
- 18–24 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Age ≥18 years and ≤24.9 years. * Patients with SCD (Hb SS, Hb SB0 and Hb SB+ genotype) who have experienced EITHER (a) 2 or more SCD-related vaso-occlusive events (acute pain events, acute chest syndrome, priapism and splenic sequestration) per year in the 2-year period before screening, OR (b) administration of regular red blood cell (RBC) transfusions (≥8 transfusions in the 12 months preceding enrollment) EXCEPT if the RBC transfusions are being administered for primary or secondary stroke prevention and, in the opinion of the treating hematologist, cannot be safely d…
Interventions
- DrugPlerixafor
Given Subcutaneous (under the skin)
- DrugBusulfan
Given Intravenous (IV)
- BiologicalGene-modified CD34+ cells
Given Intravenous (IV)
- DrugMotixafortide
Given Subcutaneous (under the skin)
Location
- St. Jude Children's Research HospitalMemphis, Tennessee