Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
3 other identifiers
observational
85
2 countries
16
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Oct 2020
Longer than P75 for all trials
16 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 21, 2020
CompletedFirst Submitted
Initial submission to the registry
November 5, 2020
CompletedFirst Posted
Study publicly available on registry
November 13, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2038
ExpectedStudy Completion
Last participant's last visit for all outcomes
January 1, 2038
March 20, 2025
March 1, 2025
17.2 years
November 5, 2020
March 19, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (4)
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 Outcomes (12)
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
- +7 more secondary outcomes
Study Arms (1)
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
Interventions
Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant
Eligibility Criteria
Subjects with sickle cell disease treated with ex vivo gene therapy product in bluebird bio-sponsored clinical studies
You may qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (16)
University of Alabama
Birmingham, Alabama, 35294, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611-2991, United States
Warren Grant Magnuson Clinical Center
Bethesda, Maryland, 20892, United States
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, 55454, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
Cohen Children's Medical Center
New Hyde Park, New York, 11040, United States
Columbia University Medical Center
New York, New York, 10032, United States
The University of North Carolina
Chapel Hill, North Carolina, 27514, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
Hospital Necker
Paris, 75015, France
Related Publications (1)
Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Treluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poiree M, Monpoux F, Touraine P, Brouzes C, de Montalembert M, Payen E, Six E, Ribeil JA, Miccio A, Bartolucci P, Leboulch P, Cavazzana M. Long-term outcomes of lentiviral gene therapy for the beta-hemoglobinopathies: the HGB-205 trial. Nat Med. 2022 Jan;28(1):81-88. doi: 10.1038/s41591-021-01650-w. Epub 2022 Jan 24.
PMID: 35075288DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Anjulika Chawla, MD
bluebird bio, Inc.
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 5, 2020
First Posted
November 13, 2020
Study Start
October 21, 2020
Primary Completion (Estimated)
January 1, 2038
Study Completion (Estimated)
January 1, 2038
Last Updated
March 20, 2025
Record last verified: 2025-03
Data Sharing
- IPD Sharing
- Will share
Bluebird bio is committed to transparency. Appropriately de-identified patient-level datasets and supporting documents may be shared (if contractually or otherwise legally permitted) following study completion and once all applicable regulatory submissions based on this study have been performed. Sharing of individual patient data will be done according to criteria established by bluebird bio and/or industry best practices to protect confidential information and maintain the privacy of study participants. For inquiries, please contact us at datasharing@bluebirdbio.com.