Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

• Conditions: Sickle Cell Disease

• 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

Study Timeline

• Study Start: 2020-10-21
• Study First Submitted: 2020-11-05
• Study First Submitted QC: 2020-11-12
• Study First Posted: 2020-11-13
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-19
• Last Update Posted: 2025-03-20
• Primary Completion: 2038-01
• Study Completion: 2038-01

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 85

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

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)

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

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