logo Hematology/Oncology Research Studies: Open for Enrollment Date: 05/27/20


Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34 Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease

NCT#/Phase: 02140554
Phase 1/2
IRB#: 2015-009
Description: This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in up to 50 adults and adolescents with Severe Sickle Cell Disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using LentiGlobin BB305 Drug Product..
Inclusion: Individuals with severe sickle cell disease who are between 12 - 50 years of age. Individuals must have had one or more of the following: Subject is to have at least 4 episodes in the past 24 months prior to informed consent. Pain episodes consist of severe vaso occlusive crises (VOC), acute Chest Syndrome (ACS), acute hepatic sequestration, acure splenic sequestration and acute priapism.
Enrollment Status: Open for enrollment
Sponsor: bluebird bio, Inc.
Principal Investigator(s): Mark Walters, MD
Contact(s): Marci Moriarty, RN (510-428-3385 x5396)
Cyrus Bascon (510-428-3385 x6953)
Study Link: https://clinicaltrials.gov/ct2/show/NCT02140554

UCSF Benioff Children's Hospital Oakland • 747 52nd Street, Oakland, California 94609 &bull Phone: (510) 428-3000