Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
NCT ID: NCT04628585
Last Updated: 2025-03-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ENROLLING_BY_INVITATION
85 participants
OBSERVATIONAL
2020-10-21
2038-01-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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CASE_ONLY
PROSPECTIVE
Study Groups
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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
Safety and efficacy assessments
Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant
Interventions
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Safety and efficacy assessments
Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant
Eligibility Criteria
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Inclusion Criteria
* Treated with drug product for therapy of sickle cell disease in a bluebird bio-sponsored clinical study
2 Years
53 Years
ALL
No
Sponsors
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Genetix Biotherapeutics Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Anjulika Chawla, MD
Role: STUDY_DIRECTOR
bluebird bio, Inc.
Locations
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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
Virginia Commonwealth University
Richmond, Virginia, United States
Hospital Necker
Paris, , France
Countries
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References
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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.
Related Links
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HGB-205 Clinicaltrials.gov
HGB-206 Clinicaltrials.gov
HGB-210 Clinicaltrials.gov
Other Identifiers
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2019-004266-18
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2024-513901-30-00
Identifier Type: CTIS
Identifier Source: secondary_id
LTF-307
Identifier Type: -
Identifier Source: org_study_id
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