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drugset / Trial / NCT04925206

A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia

NCT04925206 ↗

Phase 1 Terminated 8 enrolled EdiGene (GuangZhou) Inc.
NaSingle-groupOpen-labelTreatment

Summary

This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.

Timeline

Start
2021-08-17
Primary completion
2024-01-30
Completion
2025-03-12

Drugs

EvaluationDrugModalityDoseRoute
Subject Botulinum toxin type A Protein / enzyme biologic — Intravenous

Indications

beta thalassemia