Ignite Creation Date:
2024-05-06 @ 3:57 PM
Last Modification Date:
2024-10-26 @ 2:00 PM
Study NCT ID:
NCT04817345
Status:
WITHDRAWN
Last Update Posted:
2023-04-07
First Post:
2021-03-23
Brief Title:
Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease SCD Using Plerixafor
Sponsor:
St Jude Childrens Research Hospital
Organization:
St Jude Childrens Research Hospital
Study Overview
Official Title:
Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease SCD Using Plerixafor
Status:
WITHDRAWN
Status Verified Date:
2023-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
The safety and efficacy of plerixafor in adult and pediatric SCD patients was an unknown when we started A lot more data from therapeutic trials is available We feel that recruiting patients without a therapeutic option isnt ethically justifiable
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 cellskg 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
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 cellskg 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
Detailed 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 levelsHydroxyurea 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 024 mgkg 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
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 levelsHydroxyurea 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 024 mgkg 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None