let's recode the science
our clinical trials
bluebird bio is conducting clinical studies of investigational gene therapies in four diseases: cerebral adrenoleukodystrophy, relapsed/refractory multiple myeloma, sickle cell disease, and transfusion-dependent β-thalassemia.
For more information, follow the links below to the study listings on clinicaltrials.gov. Medical professionals may also email us at clinicaltrials@bluebirdbio.com for more information about study participation.
For more information, follow the links below to the study listings on clinicaltrials.gov. Medical professionals may also email us at clinicaltrials@bluebirdbio.com for more information about study participation.
Lenti-D
PHASE 3 STUDY
ALD-104
Evaluating the efficacy and safety of autologous CD34+ hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of CALD
STATUS: RECRUITING
PHASE 2/3 STUDY
Starbeam ALD-102
Evaluating safety and efficacy of Lenti-D gene therapy in patients with CALD
STATUS: ACTIVE, NOT RECRUITING
LONG-TERM FOLLOW-UP
LTF-304
Monitoring long-term safety and efficacy of Lenti-D gene therapy in patients with CALD treated in bluebird-sponsored clinical trials
STATUS: ENROLLING BY INVITATION
OBSERVATIONAL
ALD-103
Evaluating safety and efficacy of allogeneic hematopoietic stem cell transplantation in patients with CALD aged 17 or younger
STATUS: TERMINATED
bb21217
PHASE 1 STUDY
CRB-402
Dose-escalation and dose-expansion study of safety and efficacy of bb21217 CAR T therapy in relapsed/refractory MM
STATUS: ACTIVE, NOT RECRUITING
LONG-TERM FOLLOW-UP
LTF-306
STUDY INITIATION PENDING, ENROLLMENT BY INVITATION
LentiGlobin
PHASE 1/2 STUDIES
HGB-206
Evaluating safety and efficacy of LentiGlobin gene therapy in patients with SCD
STATUS: ACTIVE, NOT RECRUITING
HGB-205
Evaluating safety and efficacy of LentiGlobin gene therapy in patients with TDT or SCD
STATUS: COMPLETE
PHASE 3 STUDY
HGB-210
Evaluating safety and efficacy of LentiGlobin gene therapy in patients with SCD
STATUS: RECRUITING
LONG-TERM FOLLOW-UP
LTF-307
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
STATUS: ENROLLING BY INVITATION
LentiGlobin
PHASE 3 STUDIES
Northstar-2 HGB-207
Evaluating safety and efficacy of LentiGlobin gene therapy in patients with TDT and non-β0/β0 genotypes
STATUS: ACTIVE, NOT RECRUITING
Northstar-3 HGB-212
Evaluating safety and efficacy of LentiGlobin gene therapy in patients with TDT and a β0/β0 genotype
STATUS: ACTIVE, NOT RECRUITING
PHASE 1/2 STUDIES
Northstar HGB-204
Evaluating safety and efficacy of LentiGlobin gene therapy in patients with TDT
STATUS: COMPLETED; RESULTS
LONG-TERM FOLLOW-UP
LTF-303
Monitoring long-term safety and efficacy of LentiGlobin gene therapy in patients with TDT or SCD treated in bluebird-sponsored clinical trials
STATUS: ENROLLING BY INVITATION