Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)

Plain-language summary

This study aims to follow, over the long term, the safety and efficacy in people treated with the gene therapy OTL-300 for transfusion-dependent beta-thalassemia. It was designed for people who had completed the earlier TIGET-BTHAL study, received OTL-300, and been followed for two years after treatment. The study is being conducted without an assigned phase; it is not currently recruiting but is still ongoing (active, not recruiting).

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

Trial details

Phase

Not Applicable

Status

Active, not recruiting

Sponsor

IRCCS San Raffaele

Intervention

Safety and Efficacy assessments

Start date

10/4/2017

Estimated completion

6/1/2026

Countries

Italy

Related genes

Locations

• Milan, Italy

Eligibility criteria

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

Inclusion Criteria:

* Subjects who have completed study TIGET-BTHAL i.e. who have received treatment and been followed for two years post treatment with OTL-300.
* For adults; capable of giving signed informed consent. For children; informed assent and/or consent in writing signed by the subject and/or parent(s) / legal representative (according to local regulations and age of the subject).

Exclusion Criteria:

* None