Status:
COMPLETED
Escalation of Plerixafor for Mobilization of CD34+ Hematopoietic Progenitor Cells and Evaluation of Globin Gene Transfer in Patients With Sickle Cell Disease
Lead Sponsor:
Memorial Sloan Kettering Cancer Center
Collaborating Sponsors:
Sanofi
New York Blood Center
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
Brief Summary
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 i...
Eligibility Criteria
Inclusion
- 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
- 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).
Key Trial Info
Start Date :
September 17 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 27 2025
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT02193191
Start Date
September 17 2014
End Date
March 27 2025
Last Update
March 30 2025
Active Locations (2)
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1
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
2
Weill Cornell Medical College
New York, New York, United States, 10065