Ignite Creation Date:
2024-05-06 @ 6:18 PM
Last Modification Date:
2024-10-26 @ 2:45 PM
Study NCT ID:
NCT05618301
Status:
RECRUITING
Last Update Posted:
2024-05-29
First Post:
2022-11-08
Brief Title:
Motixafortide and Natalizumab to Mobilize CD34 Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease SCD
Sponsor:
Washington University School of Medicine
Organization:
Washington University School of Medicine
Study Overview
Official Title:
A Pilot Safety and Feasibility Study to Evaluate Motixafortide CXCR4SDF-1 Inhibition and Natalizumab VLA-4VCAM-1 Inhibition as a Novel Regimen to Mobilize CD34 Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease SCD
Status:
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:
None
Brief Summary:
Hematopoietic stem cell HSC-based gene therapies now offer curative potential for patients with sickle cell disease SCD with decreased toxicity compared to allogeneic hematopoietic cell transplantation However effective HSC-based gene therapy depends on collecting sufficient HSCs to generate the therapeutic product and currently available mobilization regimens carry unacceptable risk for patients with SCD or do not reliably yield optimal numbers of HSCs for gene therapy
The investigators hypothesize that HSC mobilization with motixafortide CXCR4i alone and the combination of motixafortide plus natalizumab VLA-4i will be safe and tolerable in SCD patients In addition the investigators hypothesize that combined CXCR4 and VLA-4 blockade with motixafortide plus natalizumab will result in a rapid robust and synergistic increase in HSC mobilization to peripheral blood PB in patients with SCD when compared to motixafortide alone
The investigators hypothesize that HSC mobilization with motixafortide CXCR4i alone and the combination of motixafortide plus natalizumab VLA-4i will be safe and tolerable in SCD patients In addition the investigators hypothesize that combined CXCR4 and VLA-4 blockade with motixafortide plus natalizumab will result in a rapid robust and synergistic increase in HSC mobilization to peripheral blood PB in patients with SCD when compared to motixafortide alone
Detailed Description:
None
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?:
False
Is an FDA AA801 Violation?:
None