NCT05444894

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

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)

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Editas Medicine, Inc. · Industry
• Sex: All
• Age: 18 Years – 35 Years
• Healthy volunteers: Not accepted

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

Detailed description

This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant in adult participants with TDT, age 18 to 35 years, inclusive

Conditions

• Transfusion Dependent Beta Thalassemia
• Hemoglobinopathies
• Thalassemia Major
• Thalassemia Intermedia

Interventions

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

Timeline

• Start date: 2022-04-29
• Primary completion: 2025-09-01
• Completion: 2025-12-01
• First posted: 2022-07-06
• Last updated: 2025-04-02

Locations

8 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study