Ignite Creation Date:
2024-05-06 @ 3:14 AM
Last Modification Date:
2024-10-26 @ 11:31 AM
Study NCT ID:
NCT02247843
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-05-16
First Post:
2014-09-20
Brief Title:
Stem Cell Gene Therapy for Sickle Cell Disease
Sponsor:
Donald B Kohn MD
Organization:
University of California Los Angeles
Study Overview
Official Title:
Clinical Research Study of Autologous Stem Cell Transplantation for Sickle Cell Disease SCD Using Peripheral Blood CD34 Cells Modified With the LentiG-βAS3-FB Lentiviral Vector
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 Phase I clinical trial will assess the safety and initial evidence for efficacy of an autologous transplant of lentiviral vector modified peripheral blood for adults with severe sickle cell disease
Detailed Description:
Sickle cell disease SCD affects 90000 people in the US who suffer significant neurological lung and kidney damage as well as severe chronic pain episodes that adversely impact on quality of life While current medical therapies for SCD can reduce short-term morbidity the inevitable progressive deterioration in organ function results in a significant decrease in quality of health with early mortality Allogeneic hematopoietic stem cell transplant HSCT can benefit patients with SCD by providing a source for life-long production of normal red blood cells However allogeneic HSCT is limited by the availability of well-matched donors and immunological complications especially for the more than 80 of patients who lack an HLA-identical sibling donor Autologous HSCT using a patients own peripheral blood stem cells that have been corrected by transfer of a modified human beta-globin gene that inhibits polymerization of the HbS stem cell gene therapy may provide a better therapeutic alternative as it would avoid the immunologic complications and donor limitations of allogeneic HSCT
Up to 6 subjects with SCD meeting eligibility criteria for disease severity and adequacy of organ function will be enrolled
Following informed consent enrolled subjects will be screened to confirm full eligibility for participation A chronic red blood cell transfusions regimen will be given prior to stem cell collection and transplant Subjects will undergo peripheral blood stem cell collection using plerixafor mobilization and apheresis A portion of their stem cells will be cryopreserved as back-up with the remaining portion used to prepare the gene-modified Final Cellular Product autologous peripheral blood CD34 cells transduced ex vivo by the LentiG-βAS3-FB lentiviral vector to express an anti-sickling βAS3 gene The subject will receive marrow cytoreduction with busulfan prior to infusion of the gene-modified cells The follow-up period will include an initial 2 years of active follow-up where the subjects will be seen at intervals of no more than 3 months followed by offer for enrollment into a long-term follow-up study during years 3-15
The primary objectives of the Phase I study are to assess safety and feasibility with secondary objectives to assess efficacy engraftment βAS3-globin gene expression and effects on red blood cells function and clinical hematologic and disease parameters
Up to 6 subjects with SCD meeting eligibility criteria for disease severity and adequacy of organ function will be enrolled
Following informed consent enrolled subjects will be screened to confirm full eligibility for participation A chronic red blood cell transfusions regimen will be given prior to stem cell collection and transplant Subjects will undergo peripheral blood stem cell collection using plerixafor mobilization and apheresis A portion of their stem cells will be cryopreserved as back-up with the remaining portion used to prepare the gene-modified Final Cellular Product autologous peripheral blood CD34 cells transduced ex vivo by the LentiG-βAS3-FB lentiviral vector to express an anti-sickling βAS3 gene The subject will receive marrow cytoreduction with busulfan prior to infusion of the gene-modified cells The follow-up period will include an initial 2 years of active follow-up where the subjects will be seen at intervals of no more than 3 months followed by offer for enrollment into a long-term follow-up study during years 3-15
The primary objectives of the Phase I study are to assess safety and feasibility with secondary objectives to assess efficacy engraftment βAS3-globin gene expression and effects on red blood cells function and clinical hematologic and disease parameters
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