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

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia

In brief

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

Key facts

Study ID

NCT05444894

Run by

Editas Medicine, Inc.

People needed

9

Starts

2022-04-29

Expected to finish

2025-12-01

Last updated by the study team

2025-04-02

Who can join

Age: 18 and older, up to 35. Sex: any. Healthy volunteers: not accepted.

Where it is running

• University of California San Francisco — Oakland, California, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Columbia University Medical Center - Department of Pediatrics — New York, New York, United States
• Columbia University Medical Center — New York, New York, United States
• Cleveland Clinic — Cleveland, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers — Nashville, Tennessee, United States
• Princess Margaret Cancer Centre-University Health Network — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

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