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

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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
6
sites
Charleston, South Carolina 29412
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Bethesda, MD
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Chicago, IL
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Oakland, CA
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Philadelphia, PA
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Sydney,
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