Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy (NCT02633943)
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
🩺 Plain-English Summary (8th-Grade Level)
This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent clinical studies. After completing the parent clinical studies (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in this study.
Who can join: Review the eligibility criteria below.
Location: Clinical Research Facility — Oakland, California
Age range: 0 Years to 50 Years
🧪 Interventions in This Study
- Safety and efficacy assessments (OTHER)
📋 "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 (15 Sites)
- Clinical Research Facility — Oakland, California
- Clinical Research Facility — Chicago, Illinois
- Clinical Research Facility — Bethesda, Maryland
- Clinical Research Facility — New York, New York
- Clinical Research Facility — Philadelphia, Pennsylvania
- Clinical Research Facility — Charleston, South Carolina
- Clinical Research Facility — Sydney,
- Clinical Research Facility — Marseille,
- Clinical Research Facility — Paris,
- Clinical Research Facility — Hanover,
- Clinical Research Facility — Heidelberg,
- Clinical Research Facility — Thessaloniki,
- Clinical Research Facility — Rome,
- Clinical Research Facility — Bangkok,
- Clinical Research Facility — London,
💰 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.