Ignite Creation Date:
2024-05-06 @ 10:38 AM
Last Modification Date:
2024-10-26 @ 12:33 PM
Study NCT ID:
NCT03311503
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-05-06
First Post:
2017-10-12
Brief Title:
Phase III Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
Sponsor:
David Williams
Organization:
Boston Childrens Hospital
Study Overview
Official Title:
Phase III Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-05
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:
This is a phase III open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34 selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector Subjects will be enrolled over 3 years and be followed for 2 years post-infusion on this protocol then followed long-term on a separate long-term follow-up protocol
Enrollment of subjects will be agreed upon by representatives of both sites Data will be collected uniformly from both sites through an electronic capture system and key laboratory studies will be centralized
Harvest cellular manufacturing and infusion will occur at each site using the same SOPs Key aspects of cellular product characterization will be centralized
Enrollment of subjects will be agreed upon by representatives of both sites Data will be collected uniformly from both sites through an electronic capture system and key laboratory studies will be centralized
Harvest cellular manufacturing and infusion will occur at each site using the same SOPs Key aspects of cellular product characterization will be centralized
Detailed Description:
This is an open labeled multi-center phase III cohort study involving a single infusion of autologous CD34 cells transduced with the self-inactivating SIN lentiviral vector G2SCID in up to 10 patients with X-linked SCID SCID-X1 at Boston Childrens Hospital and UCLA Mattel Childrens Hospital Patients will receive transduced cells after low dose targeted busulfan pre-conditioning n10
Enrolled subjects will be followed for 2 years after infusion on this protocol Required long-term monitoring for a total of 15 years after infusion will be performed on a separate protocol
Single infusion of autologous CD34 cells transduced with the SIN lentiviral vector rHIV_IL2RGcoG2SCID hereafter G2SCID The primary objective is to measure event free survival and T cell immune reconstitution at 1 year post-infusion
Secondary objectives are to measure overall survival event-free survival safety related to the procedure and clinical and laboratory measures of efficacy including humoral immune reconstitution and gene marking after gene transfer
Exploratory objectives include molecular characterization of gene transfer detailed assessment of biomarkers of T and B cell development and function assessment of infections nutritional status growth and development post gene therapy assessment of T cell receptor and B cell receptor repertoire by next generation sequencing correlation of busulfan levels with immune outcome and molecular measurements of gene transfer
Enrolled subjects will be followed for 2 years after infusion on this protocol Required long-term monitoring for a total of 15 years after infusion will be performed on a separate protocol
Single infusion of autologous CD34 cells transduced with the SIN lentiviral vector rHIV_IL2RGcoG2SCID hereafter G2SCID The primary objective is to measure event free survival and T cell immune reconstitution at 1 year post-infusion
Secondary objectives are to measure overall survival event-free survival safety related to the procedure and clinical and laboratory measures of efficacy including humoral immune reconstitution and gene marking after gene transfer
Exploratory objectives include molecular characterization of gene transfer detailed assessment of biomarkers of T and B cell development and function assessment of infections nutritional status growth and development post gene therapy assessment of T cell receptor and B cell receptor repertoire by next generation sequencing correlation of busulfan levels with immune outcome and molecular measurements of gene transfer
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