EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) (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)
🩺 Plain-English Summary (8th-Grade Level)
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
Who can join: Review the eligibility criteria below.
Location: University of California San Francisco — Oakland, California
Age range: 18 Years to 35 Years
🧪 Interventions in This Study
- EDIT-301 (GENETIC)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (8 Sites)
- University of California San Francisco — Oakland, California
- University of Minnesota — Minneapolis, Minnesota
- Columbia University Medical Center - Department of Pediatrics — New York, New York
- Columbia University Medical Center — New York, New York
- Cleveland Clinic — Cleveland, Ohio
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers — Nashville, Tennessee
- Princess Margaret Cancer Centre-University Health Network — Toronto, Ontario
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
Explore by condition: All Transfusion Dependent Beta Thalassemia clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.