Ignite Creation Date:
2024-05-06 @ 7:36 PM
Last Modification Date:
2024-10-26 @ 3:10 PM
Study NCT ID:
NCT06078696
Status:
RECRUITING
Last Update Posted:
2023-10-12
First Post:
2023-08-30
Brief Title:
Siplizumab for Sickle Cell Disease Transplant
Sponsor:
Markus Mapara
Organization:
Columbia University
Study Overview
Official Title:
Siplizumab-based Conditioning for Hematopoietic Stem Cell Transplantation in Patients With Advanced Sickle Cell Disease CD2 SCD
Status:
RECRUITING
Status Verified Date:
2023-10
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:
CD2 SCD
Brief Summary:
The purpose of this study is to find out whether siplizumab is safe and effective for patients with SCD undergoing an allogeneic transplant and to prevent development of Graft versus Host Disease GVHD and graft failure The main goals of this study are
To determine if acute GVHD occurs and how severe the acute GVHD is in subjects receiving the study drug
To determine if graft failure occurs in subjects receiving the study drugs
In this study participants will receive 5 infusions of the study drug siplizumab while getting a stem cell transplant for SCD Before siplizumab infusion participants will be given medications to reduce the risks of allergic reaction to the drug
To determine if acute GVHD occurs and how severe the acute GVHD is in subjects receiving the study drug
To determine if graft failure occurs in subjects receiving the study drugs
In this study participants will receive 5 infusions of the study drug siplizumab while getting a stem cell transplant for SCD Before siplizumab infusion participants will be given medications to reduce the risks of allergic reaction to the drug
Detailed Description:
Phase III label single arm safety assessment study of Siplizumab-based conditioning It is designed to determine the safety and feasibility of hematopoietic stem cell transplantation for patients with advanced sickle cell disease using in vivo T cell depletion with Siplizumab
It is a three-stage design with a target sample size of at most 18 patients At the first stage a total of 6 patients will be recruited the study will be stopped if 2 or more of patients experience any of the following events graft failureGrade 3-4 acute GVHDdeath in the first 100 days If not the study will proceed to the second stage and recruit additional 6 patients The study will be stopped if 4 or more patients experience an event as specified above among the 12 patients If not the study will proceed to the third stage and recruit additional 6 patients the study will be stopped any time if 6 or more patients experience an event among the 18 patients The study will be stopped at any time if a patient death occurs
It is a three-stage design with a target sample size of at most 18 patients At the first stage a total of 6 patients will be recruited the study will be stopped if 2 or more of patients experience any of the following events graft failureGrade 3-4 acute GVHDdeath in the first 100 days If not the study will proceed to the second stage and recruit additional 6 patients The study will be stopped if 4 or more patients experience an event as specified above among the 12 patients If not the study will proceed to the third stage and recruit additional 6 patients the study will be stopped any time if 6 or more patients experience an event among the 18 patients The study will be stopped at any time if a patient death occurs
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