Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease (SCD) Using Plerixafor

Sickle Cell Disease Clinical Trial

Official title: Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease (SCD) Using Plerixafor

Status Withdrawn

Clinical Trial 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.

Clinical Trial Description

Participants will be enrolled sequentially, and no two participants will undergo drug administration, mobilization or apheresis at the same time. A subsequent participant can only receive the study drug when the previous participant has been safely apheresed and discharged from the hospital. In the first stratum of the study only adult participants (18-25 years of age) will be enrolled. Once plerixafor and apheresis has been shown to be safe and acceptable in at least 5 adult participants, the study will enroll participants in the pediatric stratum. Pediatric stratum will not be activated until all the patients in the adult stratum have been evaluated and completed participation with acceptable results. In the pediatric stratum, older children (14 years old and above) will be enrolled before younger children (10-14 years old). After 10 participants, 14 years and older, have safely completed the study participation, younger children 10-14 years old will be allowed to participate.

Prophylactic red blood cell exchange or simple red cell transfusions will be given within 7 days prior to plerixafor administration to participants targeting HbS <30% to reduce the incidence of vaso-occlusive crisis and other events that may be associated with high hemoglobin S levels.Hydroxyurea treatment should be stopped 4 weeks before mobilization. Plerixafor administration and apheresis will be timed for participants already receiving chronic transfusion therapy such that the plerixafor administration and apheresis coincides with regularly timed transfusion.

Participants undergoing hematopoietic stem cell (HSC) mobilization will receive a daily-dose subcutaneous administration of plerixafor (Mozobil®) at 0.24 mg/kg on up to 2 consecutive days. Leukapheresis will start approximately 4 hours after each dose of plerixafor is given. This process lasts 4-10 hours.

Participants will be followed for 30 days after the last dose of plerixafor and then taken off study. ;

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

• NCT number: NCT04817345
• Study type: Interventional
• Source: St. Jude Children's Research Hospital
• Status: Withdrawn
• Phase: Phase 2
• Start date: April 2023
• Completion date: April 2023