Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Subjects with sickle-cell disease Subjects treated with ex vivo gene therapy drug product for sickle cell disease in a bluebird bio-sponsored study who agree to participate in this long-term follow-up study |
Other: Safety and efficacy assessments
Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant
|
Outcome Measures
Primary Outcome Measures
- 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 Outcome Measures
- Proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15 [Through 15 years post-drug product infusion]
- 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]
- 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
- 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
- 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 markers of iron stores [15 years post-drug product infusion]
Eligibility Criteria
Criteria
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 exclusion criteria for this study
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of Alabama | Birmingham | Alabama | United States | 35233 |
| 2 | UCSF Benioff Children's Hospital Oakland | Oakland | California | United States | 94609 |
| 3 | Children's Healthcare of Atlanta | Atlanta | Georgia | United States | 30322 |
| 4 | Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | United States | 60611-2991 |
| 5 | Warren Grant Magnuson Clinical Center | Bethesda | Maryland | United States | 20892 |
| 6 | Hackensack University Medical Center | Hackensack | New Jersey | United States | 07601 |
| 7 | Cohen Children's Medical Center | New Hyde Park | New York | United States | 11040 |
| 8 | Columbia University Medical Center | New York | New York | United States | 10032 |
| 9 | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | United States | 19104 |
| 10 | Medical University of South Carolina | Charleston | South Carolina | United States | 29425 |
| 11 | Hospital Necker | Paris | France | 75015 |
Sponsors and Collaborators
- bluebird bio
Investigators
- Study Director: Anjulika Chawla, MD, FAAP, bluebird bio, Inc.
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- LTF-307
- 2019-004266-18