Home » Clinical Trials » Beta thalassemia » NCT05444894
NCT05444894

A Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited Cluster of Differentiation 34 (CD34+) Human Hematopoietic Stem and Progenitor Cells (HSPC) (EDIT-301) in Transfusion-Dependent Beta Thalassemia (TDT)

Study Summary

The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia

Want to learn more about this trial?

Request More Info

Interventions

EDIT-301GENETIC
Administered by intravenous infusion after myeloablative conditioning with busulfan.

Study Locations

FacilityCityStateCountry
University of California San FranciscoOaklandCaliforniaUnited States
University of MinnesotaMinneapolisMinnesotaUnited States
Columbia University Medical Center - Department of PediatricsNew YorkNew YorkUnited States
Columbia University Medical CenterNew YorkNew YorkUnited States
Cleveland ClinicClevelandOhioUnited States
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvaniaUnited States
Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood CancersNashvilleTennesseeUnited States
Princess Margaret Cancer Centre-University Health NetworkTorontoOntarioCanada

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026