Ignite Creation Date:
2024-05-06 @ 8:24 PM
Last Modification Date:
2024-10-26 @ 3:26 PM
Study NCT ID:
NCT06364774
Status:
RECRUITING
Last Update Posted:
2024-06-24
First Post:
2024-03-26
Brief Title:
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
Sponsor:
Childrens Hospital of Philadelphia
Organization:
Childrens Hospital of Philadelphia
Study Overview
Official Title:
Phase 12 Study Evaluating the Safety and Efficacy of Gene Therapy Employing Lentiviral Vector ALS20-transduced Hematopoietic Progenitor Cells in Subjects With Transfusion-dependent-thalassemia
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:
The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells This is done by collecting blood stem cells from the subject modifying those cells adding a healthy beta globin gene and then giving them back to the subject It is hoped that these modified cells will decrease the need for blood transfusions The gene modified blood stem cells are called CHOP-ALS20 study drug This experimental gene therapy has not been tried on human beings before and is not FDA approved
Detailed Description:
Beta thalassemia major is a hereditary blood disorder that requires lifelong regular transfusions and is associated with significant morbidity early mortality and decreased quality of life Allogeneic hematopoietic stem cell transplantation is potentially curative but limited availability of suitable donors as well as risks of graft versus host disease limit its applicability Gene addition of a functional beta globin gene may be an alternative treatment option
The primary objective is to assess the safety of treatment with autologous hematopoietic stem cells transduced with a novel lentiviral vector ALS20 in subjects 18 to 35 years old with transfusion dependent beta thalassemia
The secondary objective is to evaluate the efficacy of treatment with autologous hematopoietic stem cells transduced with a novel lentiviral vector ALS20 in subjects 18 to 35 years old with transfusion dependent beta thalassemia
Study Design This is a single arm pilot phase 12 study of up to 12 subjects ages 18 to 35 years who have transfusion-dependent beta thalassemia genotypes β0β0 ββ0 ββ βEβ0 βEβ dominant β thalassemia The study will evaluate the safety and efficacy of infusing autologous hematopoietic stem and progenitor cells HSPC transduced with the novel lentiviral vector ALS20 that encodes the human βA-T87Q-globin following myeloablative conditioning with busulfan
The main risks of this study involve risks of the genetic modification of the stem cells and the busulfan chemotherapy conditioning Genetic modification of blood stem cells may increase the risk of blood cancer The main risks of busulfan conditioning include prolonged low blood counts liver injury infertility and cancer There also is a risk of failure of the modified blood stem cells to grow
The primary objective is to assess the safety of treatment with autologous hematopoietic stem cells transduced with a novel lentiviral vector ALS20 in subjects 18 to 35 years old with transfusion dependent beta thalassemia
The secondary objective is to evaluate the efficacy of treatment with autologous hematopoietic stem cells transduced with a novel lentiviral vector ALS20 in subjects 18 to 35 years old with transfusion dependent beta thalassemia
Study Design This is a single arm pilot phase 12 study of up to 12 subjects ages 18 to 35 years who have transfusion-dependent beta thalassemia genotypes β0β0 ββ0 ββ βEβ0 βEβ dominant β thalassemia The study will evaluate the safety and efficacy of infusing autologous hematopoietic stem and progenitor cells HSPC transduced with the novel lentiviral vector ALS20 that encodes the human βA-T87Q-globin following myeloablative conditioning with busulfan
The main risks of this study involve risks of the genetic modification of the stem cells and the busulfan chemotherapy conditioning Genetic modification of blood stem cells may increase the risk of blood cancer The main risks of busulfan conditioning include prolonged low blood counts liver injury infertility and cancer There also is a risk of failure of the modified blood stem cells to grow
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