This is a multi-center, long-term safety and efficacy follow-up study for subjects with 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.
Study Type
OBSERVATIONAL
Enrollment
85
Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant
University of Alabama
Birmingham, Alabama, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV)
Time frame: Through 15 years post-drug product infusion
Number of subjects with new or worsening hematologic disorders
Time frame: Through 15 years post-drug product infusion
Number of subjects with new or worsening neurologic disorders
Time frame: Through 15 years post-drug product infusion
Number of subjects with malignancies
Time frame: Through 15 years post-drug product infusion
Proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15
Time frame: Through 15 years post-drug product infusion
Proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15
Time frame: Through 15 years post-drug product infusion
Annualized number of severe VOEs over time through Year 15
Time frame: Through 15 years post-drug product infusion
Annualized number of VOEs over time through Year 15
Time frame: Through 15 years post-drug product infusion
Change from parent study baseline in annualized number of severe VOEs over time through Year 15
Time frame: Through 15 years post-drug product infusion
Assessment of total Hb over time post-drug product infusion through Year 15
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Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Warren Grant Magnuson Clinical Center
Bethesda, Maryland, United States
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Cohen Children's Medical Center
New Hyde Park, New York, United States
Columbia University Medical Center
New York, New York, United States
The University of North Carolina
Chapel Hill, North Carolina, United States
...and 6 more locations
Time frame: Through 15 years post-drug product infusion
Assessment of non-transfused total Hb over time post-drug product infusion through Year 15
Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q
Time frame: Through 15 years post-drug product infusion
Assessment of HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15
Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q
Time frame: Through 15 years post-drug product infusion
Assessment of HbAT87Q percentage of non-transfused total Hb over time post-drug product infusion through Year 15
Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q
Time frame: Through 15 years post-drug product infusion
Assessment of non-HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15
Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q. Non-HbS is the total g/dL of HbF + HbA2 + HbAT87Q
Time frame: Through 15 years post-drug product infusion
Change from parent study baseline through Year 15 in hemolysis markers
Change from parent study baseline through Year 15 in absolute reticulocyte count, percent reticulocytes/erythrocytes, total bilirubin, indirect bilirubin, haptoglobin, and lactate dehydrogenase hemolysis markers
Time frame: Through 15 years post-drug product infusion
Change from parent study baseline through Year 15 in markers of iron stores
Change from parent study baseline through Year 15 in serum ferritin and liver iron content markers of iron stores
Time frame: 15 years post-drug product infusion