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This Phase 1 study examines the safety and efficacy of a drug called Plerixafor in people with sickle cell disease. Plerixafor is approved by the US FDA to increase blood stem cell counts before collection in cancer patients, but it is not yet approved for sickle cell disease. Researchers want to learn whether it can also raise cell counts in these patients; participants were adults aged 18-65 with SS or Sβ thalassemia confirmed by hemoglobin fractionation.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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).