A

Phase 1

• Conditions: Subjects treated with gene therapy drug products in a bluebird bio-sponsored study will be invited to participate in this long-term follow-up study to monitor the safety and efficacy of the drug products. To date, subjects in this study have been treated with bluebird bio gene therapy drug products developed to treat beta-thalassaemia major; MedDRA version: 23.1Level: LLTClassification code 10082045Term: Beta-thalassemia traitSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2013-002245-11-GR
• Lead Sponsor: bluebird bio, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-05-12
• Last Update Posted: 7 February 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 94

Inclusion Criteria

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

Are the trial subjects under 18? yes
Number of subjects for this age range: 14
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 80
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

There are no exclusion criteria for this study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: • Monitor for long-term safety of the gene therapy drug product (i.e., the drug product) used in bluebird bio-sponsored clinical studies (i.e., the parent studies) in treated subjects with transfusion-dependent ß-<br>thalassemia (TDT)<br>• Monitor for long-term efficacy of the drug product;Secondary Objective: Not Applicable;Primary end point(s): • Immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV)<br>• New or worsening hematologic disorders<br>• New or worsening neurologic disorders<br>• Malignancies;Timepoint(s) of evaluation of this end point: Follow-up visits are scheduled every 6 months through Year 5 (Month 60) post-transplant, and then annually thereafter through Year 15 post-transplant.