Escalation of Plerixafor for Mobilization of CD34+ Hematopoietic Progenitor Cells and Evaluation of Globin Gene Transfer in Patients With Sickle Cell Disease

Prior treatment

• Patients who are:
  • Receiving or received treatment with an investigational agent within 4 weeks prior to entering the study OR
  • have not recovered from adverse events due to agents administered more than 4 weeks earlier as determined by the treating physician.

Other illness

• Patients who have uncontrolled illness including, but not limited to:
  • Ongoing or active infection
  • Emergency room admission or hospitalization in the past 14 days
  • Major surgery in the past 30 days
  • Medical/psychiatric illness/social situations that would limit compliance with study requirements as determined by the treating physician.
• Patients with active hepatitis B, hepatitis C, or HIV infection

Lab values

• Patients with ALT(SGPT) \> 2.5 X upper limit of normal
• Patients with a creatinine clearance of \< 60 ml/min
• Patients with poor cardiac function as defined by an ejection fraction \< 40% are excluded due to potential poor tolerance of the fluid shifts with leukapheresis (only for patients enrolled on second phase of protocol for Leukapheresis).

Other

• Female patients who are pregnant or breast-feeding

• Patients must have confirmed and measurable Sickle Cell Disease, defined by SS or Sβ thalassemia confirmed by hemoglobin fractionation.
• ≥ 18 to 65 years of age
• Patient must have a ECOG performance status ≤2 or Karnofsky score \> 70%
• Patients must have acceptable organ and marrow function as defined below:
  • WBC ≥ 3,000/μL
  • ANC ≥ 1,500/μL
  • platelets ≥150,000//μL
  • Hemoglobin ≥ 6 gm/dL
  • Calculated creatinine clearance ≥ 60ml/min \* \*Using the Cockcroft-gault equation \[140 - Age(yrs)\] \[Weight(kg)\] x 0.85 if Female 72 \[Serum Creatinine (mg/dL\]
• Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
• Each patient must be willing to participate as a research subject and must sign an informed consent form.