Safety of Blood Stem Cell Mobilization With Plerixafor in Patients With Sickle Cell Disease (NCT03664830) | Clinical Trial Compass
Active — Not RecruitingPhase 1
Safety of Blood Stem Cell Mobilization With Plerixafor in Patients With Sickle Cell Disease
United States5 participantsStarted 2018-09-19
Plain-language summary
The objective of this study is to investigate if up to two injections of plerixafor represent a safe and effective strategy to mobilize adequate numbers of CD34+ hematopoietic stem progenitor cells (HSPC) for autologous hematopoietic cell transplantation (HCT) in sickle cell disease (SCD) patients
Who can participate
Age range
18 Years – 40 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:
* Weight between 50 and 120 kg;
* Karnofsky performance status (KPS) ≥70%;
* Confirmed diagnosis of sickle cell disease with βS/βS or βS/β0 or βS/β+ genotype;
* Must have had one or more of the following events in the 2 year period preceding enrollment:
* History of ≥2 severe vaso-occlusive pain crises (VOC) (or at least two episodes in the year preceding the setting up of regular transfusion protocol). A severe VOC is defined as an episode of pain lasting more than 2 hours severe enough to require care at a medical facility.
* History of ≥1 episodes of acute chest syndrome despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea)
* Clinically significant neurological event (stroke) or any neurological deficit lasting 24 hours. A stroke is defined as a sudden neurological change lasting more than 24 hours that is accompanied by cerebral magnetic resonance imaging (MRI) changes.
* Prior treatment with regular RBC transfusion therapy, defined as receiving ≥8 transfusions per year for \>1 year to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and acute chest syndrome)
* Osteonecrosis of two or more joints;
* Anti-erythrocyte alloimmunization (\>2 antibodies);
* Presence of sickle cell cardiomyopathy documented by Doppler echocardiography;
* Presence of any significant cerebral abnormality such as stenosis or occlusions on magnetic resonance imaging (MRA)
* Meet current eligibility require…
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
Toxicities
Timeframe: 120 hours (5 days) from the last injection of plerixafor