A Study Evaluating the Safety and Efficacy of the LentiGlobin® BB305 Drug Product in Beta-Thalassemia Major Subjects
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ClinicalTrials.gov Identifier: NCT01745120 |
Recruitment Status
:
Completed
First Posted
: December 7, 2012
Last Update Posted
: March 30, 2018
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Beta-thalassemia Major | Genetic: LentiGlobin® BB305 Drug Product | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 18 participants |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product) |
Study Start Date : | August 2013 |
Actual Primary Completion Date : | February 8, 2018 |
Actual Study Completion Date : | February 21, 2018 |

Arm | Intervention/treatment |
---|---|
Experimental: LentiGlobin® BB305 Drug Product |
Genetic: LentiGlobin® BB305 Drug Product
Transplant of autologous hematopoietic stem cells transduced with LentiGlobin® BB305 lentiviral vector.
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- Evaluate the efficacy of treatment with LentiGlobin® BB305 Drug Product in subjects with β-thalassemia major as measured by the production of hemoglobin containing the therapeutic globin protein [βA-T87Q-globin] [ Time Frame: 18 - 24 months post-transplant ]
- Evaluate the safety of treatment with LentiGlobin® BB305 Drug Product in subjects with β-thalassemia major as measured by the incidence of adverse events [ Time Frame: 0-24 months post-transplant ]
- Hematopoietic stem cell engraftment [ Time Frame: 42 days post-transplant ]
- Assess transgene marking as determined by measurement of the average vector copy number in peripheral blood and bone marrow [ Time Frame: 0 - 24 months post-transplant ]

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Ages Eligible for Study: | 12 Years to 35 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion criteria:
- Subjects between 12 and 35 years of age, inclusive, at the time of consent/assent, and able to provide written consent/assent, if applicable.
- Diagnosis of β-thalassemia major and a history of at least 100 mL/kg/year of pRBCs or ≥8 transfusions of pRBCs per year for the prior 2 years.
- Eligible for allogeneic bone marrow transplant.
- Treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history.
Exclusion criteria:
- Positive for presence of human immunodeficiency virus type 1 or 2 (HIV 1 and HIV 2).
- A white blood cell (WBC) count <3 × 109/L, and / or platelet count <100 × 109/L if not due to hypersplenism.
- Uncorrected bleeding disorder.
- Any prior or current malignancy or myeloproliferative or immunodeficiency disorder.
- Immediate family member with a known or suspected Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome and familial adenomatous polyposis).
- Receipt of an allogeneic transplant.
- Advanced liver disease, including persistent aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin value >3 × the upper limit of normal, liver biopsy demonstrating cirrhosis, extensive bridging fibrosis, or active hepatitis.
- Kidney disease with a calculated creatinine clearance <30% normal value.
- Uncontrolled seizure disorder.
- Diffusion capacity of carbon monoxide (DLco) <50% of predicted (corrected for hemoglobin).
- A cardiac T2* <10 ms by magnetic resonance imaging (MRI).
- Any other evidence of severe iron overload that, in the Investigator's opinion, warrants exclusion.
- Clinically significant pulmonary hypertension, as defined by the requirement for ongoing pharmacologic treatment or the consistent or intermittent use of supplemental home oxygen.
- Participation in another clinical study with an investigational drug within 30 days of Screening.
- Any prior or current malignancy or myeloproliferative disorder.
- Prior receipt of gene therapy.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01745120
United States, California | |
Oakland, California, United States | |
United States, Illinois | |
Chicago, Illinois, United States | |
United States, Pennsylvania | |
Philadelphia, Pennsylvania, United States | |
Australia | |
Sydney, Australia | |
Thailand | |
Bangkok, Thailand |
Study Director: | Mohammed Asmal, MD | bluebird bio |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | bluebird bio |
ClinicalTrials.gov Identifier: | NCT01745120 History of Changes |
Other Study ID Numbers: |
HGB-204 |
First Posted: | December 7, 2012 Key Record Dates |
Last Update Posted: | March 30, 2018 |
Last Verified: | March 2018 |
Keywords provided by bluebird bio:
gene therapy beta thalassemia hemoglobin anemia CD34 |
Additional relevant MeSH terms:
Thalassemia beta-Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic |
Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |