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A Study Evaluating the Safety and Efficacy of the LentiGlobin® BB305 Drug Product in Beta-Thalassemia Major Subjects

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01745120
Recruitment Status : Completed
First Posted : December 7, 2012
Last Update Posted : March 30, 2018
Information provided by (Responsible Party):
bluebird bio

Brief Summary:
This is a non-randomized, open label, multi-site, single-dose, Phase 1/2 study in up to 18 subjects (including at least 3 adolescents between 12 and 17 years of age, inclusive) with beta-thalassemia major. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin® BB305 Drug Product [autologous CD34+ hematopoietic stem cells transduced with LentiGlobin® BB305 Lentiviral Vector encoding the human βA-T87Q-globin gene].

Condition or disease Intervention/treatment Phase
Beta-thalassemia Major Genetic: LentiGlobin® BB305 Drug Product Phase 1 Phase 2

Detailed Description:
Subject participation for this study will be 2 years. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 13 years post-transplant.

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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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

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.

Primary Outcome Measures :
  1. 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 ]
  2. 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 ]

Secondary Outcome Measures :
  1. Hematopoietic stem cell engraftment [ Time Frame: 42 days post-transplant ]
  2. 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 ]

Information from the National Library of Medicine

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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.

Information from the National Library of Medicine

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 identifier (NCT number): NCT01745120

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United States, California
Oakland, California, United States
United States, Illinois
Chicago, Illinois, United States
United States, Pennsylvania
Philadelphia, Pennsylvania, United States
Sydney, Australia
Bangkok, Thailand
Sponsors and Collaborators
bluebird bio
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Study Director: Mohammed Asmal, MD bluebird bio

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: bluebird bio 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

Additional relevant MeSH terms:
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Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn