Escalation of Plerixafor for Mobilization of CD34+ Hematopoietic Progenitor Cells and Evaluation … (NCT02193191) | Clinical Trial Compass
CompletedPhase 1
Escalation of Plerixafor for Mobilization of CD34+ Hematopoietic Progenitor Cells and Evaluation of Globin Gene Transfer in Patients With Sickle Cell Disease
United States25 participantsStarted 2014-09-17
Plain-language 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 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.
Who can participate
Age range18 Years – 65 Years
SexALL
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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 illn…
What they're measuring
1
safety
Timeframe: up to 30 days
2
efficacy
Timeframe: ≥ 30/ul at either 6-12 hours or 24-48 hours post plerixafor.