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

About the study

The purpose of this research study is to test the safety and efficacy of a drug called
Plerixafor. Plerixafor is approved by the US FDA for use in increasing blood stem cell counts
before collection in cancer patients. It is not yet approved for patients with sickle cell
disease. The investigators want to find out if Plerixafor can be used to increase cell counts
in patients with sickle cell disease.

Study point of contact

Susan Prockop, MD
Farid Boulad, MD
212- 639-6684


2 United States sites


18 to 65 Years


Phase 1

Study type








participation requirements

– 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.

participation restrictions

– 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).


  • New York, New York, United States, Memorial Sloan Kettering Cancer Center, 10065 [Recruiting]
  • New York, New York, United States, Weill Cornell Medical College, 10065 [Active, not recruiting]
Last updated 2021-04-09