NCT04628585

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector

Study 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.

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Interventions

Safety and efficacy assessmentsOTHER
Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant

Study Locations

FacilityCityStateCountry
University of AlabamaBirminghamAlabamaUnited States
UCSF Benioff Children's Hospital OaklandOaklandCaliforniaUnited States
Children's Healthcare of AtlantaAtlantaGeorgiaUnited States
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinoisUnited States
Warren Grant Magnuson Clinical CenterBethesdaMarylandUnited States
University of Minnesota Masonic Children's HospitalMinneapolisMinnesotaUnited States
Hackensack University Medical CenterHackensackNew JerseyUnited States
Cohen Children's Medical CenterNew Hyde ParkNew YorkUnited States
Columbia University Medical CenterNew YorkNew YorkUnited States
The University of North CarolinaChapel HillNorth CarolinaUnited States
Duke University Medical CenterDurhamNorth CarolinaUnited States
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvaniaUnited States
Medical University of South CarolinaCharlestonSouth CarolinaUnited States
Baylor College of MedicineHoustonTexasUnited States
Virginia Commonwealth UniversityRichmondVirginiaUnited States
Hospital NeckerParisFrance

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026