Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

Plain-language summary

This multi-center study provides long-term safety and efficacy follow-up for subjects with transfusion-dependent β-thalassemia (TDT) who were treated with an ex vivo gene therapy drug product in bluebird bio-sponsored parent studies. After the roughly 2-year parent studies, eligible subjects are followed for an additional 13 years, for a total of 15 years post-infusion. No investigational drug product is administered in this study; participation requires prior treatment in a parent study and written informed consent.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Active, not recruiting

Sponsor

Genetix Biotherapeutics Inc.

Intervention

Safety and efficacy assessments

Start date

1/1/2014

Estimated completion

11/1/2035

Countries

United States, Australia, France, Germany, Greece, Italy, Thailand, United Kingdom

Related genes

Locations

• Oakland, United States
• Chicago, United States
• Bethesda, United States
• New York, United States
• Philadelphia, United States
• Charleston, United States
• Sydney, Australia
• Marseille, France
• Paris, France
• Hanover, Germany
• Heidelberg, Germany
• Thessaloniki, Greece
• Rome, Italy
• Bangkok, Thailand
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* Provision of written informed consent for this study by subjects, or as applicable, subject's parent(s)/legal guardian(s)
* Treated with drug product for therapy of transfusion-dependent β-thalassemia in a bluebird bio-sponsored clinical study

Exclusion Criteria:

* There are no exclusion criteria for this study