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

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: 2024-12
• Last Update Submitted: 2024-12-20
• Last Update Posted: 2024-12-27
• 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

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Exclusion Criteria

• There are no exclusion criteria for this study

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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 (15)

University of Minnesota Masonic Children's Hospital; 🇺🇸 Minneapolis, Minnesota, 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

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

Hospital Necker; 🇫🇷 Paris, France