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

Inclusion Criteria:

- 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.

Exclusion Criteria:

- 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.

- Patients with ALT(SGPT) > 2.5 X upper limit of normal

- Patients with a creatinine clearance of < 60 ml/min

- 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.

- Female patients who are pregnant or breast-feeding

- Patients with active hepatitis B, hepatitis C, or HIV infection

- 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).