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

This is an interventional treatment trial for Sickle Cell Disease focused on measuring Plerixafor, 13-229 Read More

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).