Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy

Status:	Enrolling by invitation
Conditions:	Anemia, Hematology
Therapuetic Areas:	Hematology
Healthy:	No
Age Range:	Any - 50
Updated:	2/2/2019
Start Date:	September 2013
End Date:	March 2031

Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

This is a multi-center, long-term safety and efficacy follow-up study for subjects with
hemoglobinopathies (β-thalassemia or severe sickle cell disease) who have been treated with
ex vivo gene therapy drug product in bluebird bio-sponsored 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 the study.

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 a hemoglobinopathy in a bluebird
bio-sponsored clinical study

- Able to comply with study requirements

Exclusion Criteria:

- Met the VCN discontinuation criterion in the parent study

We found this trial at

sites

Clinical Trial Facility in Sydney

Sydney,

from

Sydney,