Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
- Conditions
- Sickle Cell Disease
- Interventions
- Other: Safety and efficacy assessments
- Registration Number
- NCT04628585
- Lead Sponsor
- bluebird bio
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 85
- 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
- There are no exclusion criteria for this study
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Subjects with sickle-cell disease Safety and efficacy assessments 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
- Primary Outcome Measures
Name Time Method 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 malignancies 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
- Secondary Outcome Measures
Name Time Method Annualized number of VOEs over time through Year 15 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 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
Proportion of subjects with complete resolution of severe VOEs (sVOE-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 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
Proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15 Through 15 years post-drug product infusion 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 in annualized number of severe VOEs over time through Year 15 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 Through 15 years post-drug product infusion Non-transfused total Hb refers to the total g/dL of HbS + HbF + HbA2 + HbAT87Q
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
Trial Locations
- Locations (16)
Hospital Necker
🇫🇷Paris, France
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
University of Alabama
🇺🇸Birmingham, Alabama, United States
UCSF Benioff Children's Hospital Oakland
🇺🇸Oakland, California, United States
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
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States