logo Hematology/Oncology Research Studies: Open for Enrollment Date: 09/20/19


Phase 1 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
IRB#: 2015-009
Description: This is a non-randomized, open label, multi-site, single dose, Phase 1 study in up to 29 adults 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 18 years of age and older. Individuals must have had one or more of the following: Recurrent severe vaso occlusive crises (VOC) (at least 2 episodes per year in the preceding 2 years or in the 2 years prior to initiation of a regular transfusion program). Acute Chest Syndrome (ACS) (at least 2 total episodes in the prior 2 years, with at least one episode in the past year or in the year prior to the initiation of a regular transfusion program), defined as an acute event with pneumonia-like symptoms and the presence of a new pulmonary infiltrate. History of an overt stroke, defined as a sudden neurologic change lasting more than 24 hours that is accompanied by cerebral MRI changes. Echocardiographic evidence of a tricuspid regurgitant jet velocity (TRJV) of > 2.5 m/s.
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