Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
Active, not recruiting · NCT02633943
Conditions: Transfusion-dependent Beta-Thalassemia
At a glance
- Research payments reported, 2025
- $1.4M
- U.S. investigators named
- 7
- Sites paid
- 9
- Planned or actual enrollment
- 66
About the trial
- Sponsor
- Genetix Biotherapeutics Inc.
- Tested
- Safety and efficacy assessments (other)
- Dates
- Started 2014-01; completion 2035-11
- Results posted
- Not yet
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.
From ClinicalTrials.gov, last updated 2025-04-09. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genetix Biotherapeutics Inc reported $1.4M in research payments for this study in 2025 (61 payments).
Products named: Zynteglo
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Mark Walters, MO Pediatric Hematology & Oncology · Oakland, CA
- Robert I. Liem VIII, MD Pediatric Hematology & Oncology · Chicago, IL
- David A. Williams, MD Pediatric Hematology & Oncology · Boston, MA
- Paul J. Orchard, MD Pediatric Hematology & Oncology · Minneapolis, MN
- Anjulika Chawla, MD Pediatric Hematology & Oncology · Lebanon, NH
- Janet L. Kwiatkowski, M.D. Pediatric Hematology & Oncology · Philadelphia, PA
- Timothy Olson, M.D., PH.D. Pediatric Hematology & Oncology · Philadelphia, PA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- The Medical College of Wisconsin $685,551
- Cincinnati Children's Hospital Medical Center, OH $209,784
- Ronald Reagan UCLA Medical Center, CA $110,001
- Children's Hospital of Philadelphia, PA $100,389
- Ann & Robert H Lurie Childrens Hospital of Chicago, IL $93,806
- Boston Children's Hospital, MA $69,750
- Stanford University $38,784
- Regents of the University of Minnesota $35,390
- UCSF Benioff Children's Hospital Oakland, CA $11,346
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.