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NCT04293185

A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the BB305 Lentiviral Vector in Subjects With Sickle Cell Disease

Study Summary

This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD).

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Interventions

bb1111GENETIC
Drug Product is administered by IV infusion following myeloablative conditioning with busulfan.

Study Locations

FacilityCityStateCountry
University of AlabamaBirminghamAlabamaUnited States
Children's National HospitalWashington D.C.District of ColumbiaUnited States
Tufts Medical CenterBostonMassachusettsUnited States
University of MinnesotaMinneapolisMinnesotaUnited States
Hackensack University Medical CenterHackensackNew JerseyUnited States
Montefiore Medical CenterThe BronxNew YorkUnited States
Duke UniversityDurhamNorth CarolinaUnited States
Baylor College of Medicine/Texas Children's HospitalHoustonTexasUnited States
Virginia Commonwealth University (VCU)RichmondVirginiaUnited States

Official Trial Information

View on ClinicalTrials.gov

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