Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease (SCD) Using Plerixafor
Status:
Recruiting
Trial end date:
2023-04-01
Target enrollment:
Participant gender:
Summary
With recent advances in gene editing, gene therapy is becoming a viable curative treatment
option for sickle cell disease. In order to do genetic manipulation, investigators need to
collect hematopoietic stem cells from patients with sickle cell disease. In this study,
investigators want to study the safety and feasibility of collecting peripheral blood stem
cells from pediatric and young adult patients with sickle cell disease after administering
plerixafor. Studying these peripheral blood stem cells will help in optimizing the yield of
peripheral CD34+ cells from pediatric and young adult patients with sickle cell disease,
which in turn will help to develop better gene therapies for these patients.
Primary Objectives
- Determine the safety profile associated with administration of plerixafor in pediatric
and young adult patients with sickle cell disease (SCD).
- To estimate the number of CD34+ cells/kg of body weight that can be collected with
peripheral apheresis after administration of plerixafor in pediatric and young adult
patients with SCD.
Exploratory Objectives
- To describe the kinetics of CD34+ cell mobilization in peripheral blood after - + cells
obtained from pediatric and young adult patients with SCD.
- To study the effect of hydroxyurea therapy on senescence in plerixafor-mobilized CD34+
cells obtained from pediatric and young adult patients with SCD.