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 range
18 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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.