|Purpose||This open-label, Phase 1/2 study will determine the safety, efficacy and feasibility of ARU-1801 in patients with severe sickle cell disease (SCD).|
|Investigational Product||ARU-1801 is autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene.|
|Mechanism of Action||ARU-1801 is designed to increase production of fetal hemoglobin (HbF) in order to reduce vaso-occlusive crises, hospitalizations and cumulative organ damage. The modified γ-globin gene contains a point mutation at codon 16 that improves the affinity of γ-globin to α-globin, which promotes the preferential formation of a modified HbF tetramer.|
|Reduced Intensity Conditioning||The proprietary modification of the gamma globin allows engraftment with only reduced intensity conditioning, and does not require fully myeloablative conditioning prior to treatment.|
- Ages 18 to 45 years.
- Confirmed diagnosis of severe SCD (HbSS, HbS-b0 or Hbs-b+ thalassemia).
- Severe SCD is defined as one or more of the following:
- At least two severe acute sickle pain events per year requiring treatment at a medical facility outside of home, or
- Two or more lifetime episodes of acute chest syndrome (ACS), or
- One episode of ACS requiring intensive care unit (ICU) admission, or
- Chronic transfusions required for management of disease symptoms.
- Adequate functional status and organ function.
- Current trial sites
- Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio.
- Atrium Health, Levine Cancer Institute, Charlotte, North Carolina.
- University of the West Indies Hospital, Kingston, Jamaica.
- University of Pennsylvania, Philadelphia, Pennsylvania.
- Study participation requires two trips to Cincinnati (for apheresis and product administration) with all other visits taking place at site.
- All travel expenses for patient and one family member are reimbursed.
Duration and Follow-Up
After ARU-1801 infusion, patients are monitored to evaluate the efficacy, safety and durability of ARU-1801. The follow-up schedule is as follows:
- Once per month for the first year
- Every three months in Year 2
- Every 6 months years 3-5
- Every year in years 6-15
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