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Clinical Trial Report
Gene Therapy Trial Report
Summary
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
NCTID
NCT03432364
(View at clinicaltrials.gov)
Description
This is a single-arm, multi-site, single-dose, Phase 1/2 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 patient's own blood stem cells which are genetically modified in the laboratory using Sangamo's 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.
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Development Status
Inactive
Indication
Beta-Thalassemia Major
Disease Ontology Term
DOID:0080771
Compound Name
ST-400
Sponsor
Sangamo Therapeutics
Funder Type
Industry
Recruitment Status
Completed
Enrollment Count
5
Results Posted
View Results
Therapy Information
Target Gene/Variant
BCL11A
Therapy Type
Gene editing
Therapy Route
Ex-vivo
Mechanism of Action
Overexpression of protective allele/gene
Route of Administration
Intravenous
Drug Product Type
Autologous cells
Target Tissue/Cell
CD34+ cells
Delivery System
Electroporation
Vector Type
Editor Type
ZFN mRNA
Dose 1
Minimum dose: 4.5E6 cells/kg
Dose 2
Max dose: 11.4E6 CD34+ cells/kg
Dose 3
Average dose: 7.3E6 CD34+ cells/kg
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2018-02-01
Completion Date
2022-11-17
Last Update
2023-12-14
Participation Criteria
Eligible Age
18 Years - 40 Years
Standard Ages
Adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
6
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Development discontinued by Sponsor November 2021
Resources/Links
Clinical Publications
(Poster) Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia - ASH 2021
News and Press Releases
Sangamo Therapeutics Reports Recent Business and Clinical Highlights and Third Quarter 2021 Financial Results
Preclinical Publications
Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major
Protocol
Statistical Analysis Plan
Clinical Trial Protocol
Related NCTID
Long Term Follow-Up: NCT05145062