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Clinical Trial Summary

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 study (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.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT02633943
Study type Observational
Source bluebird bio
Contact
Status Active, not recruiting
Phase
Start date January 2014
Completion date November 2035

See also
  Status Clinical Trial Phase
Active, not recruiting NCT04770779 - A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) Phase 3
Recruiting NCT05860595 - Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia. N/A
Recruiting NCT05991336 - Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Not yet recruiting NCT06363760 - A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
Recruiting NCT06219239 - Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients N/A
Completed NCT06146478 - Deciphering Effects of Thalidomide on Red Blood Cells in Transfusion Dependents Beta Thalassemia Patients Phase 3
Not yet recruiting NCT06280378 - A Phase I/II Clinical Study of the KL003 Cell Injection in β-Thalassemia Major Participants Phase 1/Phase 2