Ignite Creation Date:
2024-05-06 @ 12:03 PM
Last Modification Date:
2024-10-26 @ 12:53 PM
Study NCT ID:
NCT03664830
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-02-22
First Post:
2018-09-07
Brief Title:
Safety of Blood Stem Cell Mobilization With Plerixafor in Patients With Sickle Cell Disease
Sponsor:
City of Hope Medical Center
Organization:
City of Hope Medical Center
Study Overview
Official Title:
A Pilot Study To Evaluate The Safety And Feasibility of Hematopoietic Progenitor Cell Mobilization With Plerixafor as Part of a Gene Therapy Strategy in Sickle Cell Disease
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
PISMO
Brief 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
Detailed Description:
Allogeneic hematopoietic cell transplantation HCT is the only curative option for sickle cell disease SCD patients but its use is significantly limited by the lack of compatible donors Gene therapy using autologous hematopoietic stem cell HSC transplantation represents an alternative approach but requires the collection of significant numbers of cells Severe adverse events have been observed in SCD patients after mobilization using the standard agent granulocyte colony-stimulating factor G-CSF and bone marrow harvesting is also associated with side effects The use of a single administration of plerixafor has been suggested as an alternative mobilization strategy in SCD but may not mobilize sufficient number of HSC In this study our primary objective is to assess if up to two injections of plerixafor starting dose level 240 µgkgdose are safe in SCD patients and can recruit enough blood stem cells needed for future gene therapy
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