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 range18 Years – 40 Years
SexALL
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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…
What they're measuring
1
Toxicities
Timeframe: 120 hours (5 days) from the last injection of plerixafor