Clinical Research Directory

Inclusion Criteria: 1. Written, informed consent 2. Age 18 to 45 years of age, inclusive at screening 3. Confirmed SS or S-b0-thalassemia SCD 4. Sickle crisis rate of 2-10 within the past year with no crisis in the last 28 days 5. HbF \<8.6% of total Hb at screening 6. Regular compliance with comprehensive care and previous therapy - Exclusion Criteria: 1. Experienced severe sepsis or septic shock within the previous 12 weeks 2. Febrile illness in the 1 week prior to baseline visit 3. Acute complications due to SCD (i.e., hospitalization, acute pain, or acute chest syndrome) in the 28 days prior to screening visit 4. Plans for hospitalization, surgery, or other major procedures during the duration of the study or between screening and baseline 5. ALT ≥2X the upper limit of normal or albumin \<2.0 mg/dL or direct (conjugated) bilirubin ≥ 1.5 mg/dl\* 6. Serum creatinine \>2.9 mg/dL and calculated creatinine clearance \<30 mL/min# \* 7. Platelet count \>800 x 109/L OR \<150 x 109/L\* 8. Absolute neutrophil count \<1.5 x 109/L\* 9. Currently pregnant or breastfeeding 10. Female of active childbearing potential$ who is unwilling or unable to adhere to the contraception requirements specified in the protocol 11. Male with female partner(s) of childbearing potential$ who is unwilling or unable to adhere to the contraception requirements specified in the protocol 12. Altered mental status or recurrent seizures requiring anti-seizure medications 13. Moribund or any concurrent disease (e.g., hepatic, renal, cardiac, metabolic) of such severity that death within 24 weeks is likely 14. Concurrent diagnosis of malignancy including MDS, leukemia, or an abnormal karyotype 15. Known Vitamin-B12, folate, or iron deficiency 16. New York Heart Association (NYHA) class III/IV status 17. Eastern Co-operative Oncology Group (ECOG) performance status ≥3 18. Participant is on chronic transfusion therapy (e.g., for history of TIA or stroke) and medically contraindicated to discontinue transfusions (unless multiple allo-antibodies prevent the patient from getting transfusions as scheduled) 19. Blood transfusion in the 28 days prior to screening visit or between screening and baseline visits 20. Known history of illicit drug or alcohol abuse within the past 12 months. 21. Current treatment with Oxbryta or Adakveo (must be off therapy for 30 days for Oxbryta with no plans to restart and off therapy for 3 months for Adakveo with no plans to restart) 22. Other experimental or investigational drug therapy in the past 28 days -