2 United States sites
18 to 65 Years
– 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
– Each patient must be willing to participate as a research subject and must sign an
informed consent form.
– 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).