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 the following indications:beta-thalassaemia majorSickle cell disease; MedDRA version: 20.1Level: LLTClassification code 10055579Term: Sickle-cell beta thalassemiaSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

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

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-12-17
• Last Update Posted: 30 November 2020

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. Previously treated with drug product for therapy of a hemoglobinopathy in a bluebird bio-sponsored clinical study.
3. Able to comply with study requirements

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