Sickle Cell Disease
Conditions
Keywords
Hematopoietic Stem Cells
Brief summary
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.
Interventions
Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant
Sponsors
Genetix Biotherapeutics Inc.
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to 53 Years
Healthy volunteers
No
Inclusion criteria
* Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s) * Treated with drug product for therapy of sickle cell disease in a bluebird bio-sponsored clinical study
Exclusion criteria
* There are no
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) | Through 15 years post-drug product infusion |
| Number of subjects with new or worsening hematologic disorders | Through 15 years post-drug product infusion |
| Number of subjects with new or worsening neurologic disorders | Through 15 years post-drug product infusion |
| Number of subjects with malignancies | Through 15 years post-drug product infusion |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from parent study baseline in annualized number of severe VOEs over time through Year 15 | Through 15 years post-drug product infusion | — |
| Assessment of total Hb over time post-drug product infusion through Year 15 | Through 15 years post-drug product infusion | — |
| Assessment of non-transfused total Hb over time post-drug product infusion through Year 15 | Through 15 years post-drug product infusion | Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q |
| Assessment of HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15 | Through 15 years post-drug product infusion | Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q |
| Proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15 | 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 | Through 15 years post-drug product infusion | 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 |
| Change from parent study baseline through Year 15 in hemolysis markers | Through 15 years post-drug product infusion | 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 |
| Change from parent study baseline through Year 15 in markers of iron stores | 15 years post-drug product infusion | Change from parent study baseline through Year 15 in serum ferritin and liver iron content markers of iron stores |
| Assessment of HbAT87Q percentage of non-transfused total Hb over time post-drug product infusion through Year 15 | Through 15 years post-drug product infusion | Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q |
| Proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15 | Through 15 years post-drug product infusion | — |
| Annualized number of severe VOEs over time through Year 15 | Through 15 years post-drug product infusion | — |
| Annualized number of VOEs over time through Year 15 | Through 15 years post-drug product infusion | — |
Countries
France, United States
Outcome results
None listed