Ignite Creation Date:
2024-05-06 @ 11:07 AM
Last Modification Date:
2024-10-26 @ 12:40 PM
Study NCT ID:
NCT03432364
Status:
COMPLETED
Last Update Posted:
2023-12-14
First Post:
2018-02-01
Brief Title:
A Study to Assess the Safety Tolerability and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia TDT
Sponsor:
Sangamo Therapeutics
Organization:
Sangamo Therapeutics
Study Overview
Official Title:
A Phase 12 Open-label Single-arm Study to Assess the Safety Tolerability and Efficacy of ST-400 Autologous Hematopoietic Stem Cell Transplant for Treatment of Transfusion-Dependent Beta-thalassemia TDT
Status:
COMPLETED
Status Verified Date:
2023-11
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 single-arm multi-site single-dose Phase 12 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia TDT who are 18 and 40 years of age ST-400 is a type of investigational therapy that consists of gene edited cells ST-400 is composed of the patients own blood stem cells which are genetically modified in the laboratory using Sangamos zinc finger nuclease ZFN technology to disrupt a precise and specific sequence of the enhancer of the BCL11A gene which normally suppresses fetal hemoglobin production in erythrocytes This process is intended to boost fetal hemoglobin HbF which can substitute for reduced or absent adult defective hemoglobin ST-400 is then infused back into the patient after receiving conditioning chemotherapy to make room for the new cells in the bone marrow with the aim of producing new erythrocytes with increased amounts of HbF The primary objective is to understand safety and tolerability of ST-400 and secondary objectives are to assess the effects on HbF levels and transfusion requirements
Detailed Description:
Once consented study participants will progress through the following stages
Screening in-person visit at the study site to confirm eligibility for proceeding
Collection autologous self blood stem cells are harvested at the study site also known as apheresis
Manufacturing of ST-400 no study participant activities expected
Infusion conditioning chemotherapy followed by infusion of ST-400 occurs at the study site
Follow-up follow up at the study site to monitor for safety and effectiveness of the study
Screening in-person visit at the study site to confirm eligibility for proceeding
Collection autologous self blood stem cells are harvested at the study site also known as apheresis
Manufacturing of ST-400 no study participant activities expected
Infusion conditioning chemotherapy followed by infusion of ST-400 occurs at the study site
Follow-up follow up at the study site to monitor for safety and effectiveness of the study
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