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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
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