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Clinical trial

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)

NCT05444894 is registered in ClinicalTrials.gov with Editas Medicine, Inc. as lead sponsor.

NCT NCT05444894
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Registry record

As filed in ClinicalTrials.gov.

Title
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)
Lead sponsor
Editas Medicine, Inc.
Phase
Phase 1/2
Overall status
Active, not recruiting
Start date
April 29, 2022
Completion date
December 31, 2025
Enrollment
9 (estimated)
Sites
8

Conditions

As the registry lists them; a condition with its own page is linked.

hemoglobinopathies, thalassemia intermedia, thalassemia major, transfusion dependent beta thalassemia