Ignite Creation Date:
2024-06-16 @ 11:50 AM
Last Modification Date:
2024-10-26 @ 3:31 PM
Study NCT ID:
NCT06442761
Status:
RECRUITING
Last Update Posted:
2024-06-14
First Post:
2024-05-13
Brief Title:
SCD Stem Cell Mobilization and Apheresis Using Motixafortide
Sponsor:
St Jude Childrens Research Hospital
Organization:
St Jude Childrens Research Hospital
Study Overview
Official Title:
Sickle Cell Disease Stem Cell Mobilization and Apheresis Using Motixafortide
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study is being done to see if the study drug motixafortide is safe in participants with sickle cell disease SCD Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use
PRIMARY OBJECTIVE
To characterize the safety and tolerability of motixafortide in participants with SCD as determined by the incidence of adverse events AEs
SECONDARY OBJECTIVES
To characterize the efficacy of a single dose Part A or two doses Part B of motixafortide for hematopoietic stem cell HSC mobilization and apheresis collection in participants with SCD as determined by the yield of CD34 cells CD34 cellskg
To measure the mobilization effects of single-day Part A or daily dosing Part B dosing with motixafortide in the peripheral blood in participants with SCD as determined by peak peripheral blood CD34 counts
To recommend a phase 2 dosing strategy based on safety efficacy and mobilization effects
PRIMARY OBJECTIVE
To characterize the safety and tolerability of motixafortide in participants with SCD as determined by the incidence of adverse events AEs
SECONDARY OBJECTIVES
To characterize the efficacy of a single dose Part A or two doses Part B of motixafortide for hematopoietic stem cell HSC mobilization and apheresis collection in participants with SCD as determined by the yield of CD34 cells CD34 cellskg
To measure the mobilization effects of single-day Part A or daily dosing Part B dosing with motixafortide in the peripheral blood in participants with SCD as determined by peak peripheral blood CD34 counts
To recommend a phase 2 dosing strategy based on safety efficacy and mobilization effects
Detailed Description:
This study is divided into 2 parts Participants will be assigned to a part based on when they enroll Early in the study participants will be assigned to part A Later in the study participants will be assigned to part B In both parts participants will receive the study drug motixafortide by an injection under the skin About 8 hours after the injection stem cells will be collected Participants will follow-up within 7-10 days after the study drug and stem cell collection Study staff will contact participants about 30 days after the last drug dose administration
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