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

  • End date
    Jul 18, 2023
  • participants needed
  • sponsor
    Memorial Sloan Kettering Cancer Center
Updated on 18 April 2022
absolute neutrophil count
gene therapy


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.

Condition Sickle Cell Disease
Treatment Plerixafor
Clinical Study IdentifierNCT02193191
SponsorMemorial Sloan Kettering Cancer Center
Last Modified on18 April 2022


Yes No Not Sure

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
Each patient must be willing to participate as a research subject and must sign an informed consent form
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

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)
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact



Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider


Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 



Reply by • Private

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note