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.
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
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