Trial record 13 of 58 for:
Open Studies | "Thalassemia"
A Study Evaluating the Safety and Efficacy of the LentiGlobin® BB305 Drug Product in Beta-Thalassemia Major Subjects
This study is not yet open for participant recruitment.
Verified May 2013 by bluebird bio
Sponsor:
bluebird bio
Information provided by (Responsible Party):
bluebird bio
ClinicalTrials.gov Identifier:
NCT01745120
First received: December 6, 2012
Last updated: May 13, 2013
Last verified: May 2013
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
Purpose
This is a non randomized, open label, multi-site, single-dose, Phase 1/2 study in up to 15 adults 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 β-A(T87Q)-globin gene].
| Condition | Intervention | Phase |
|---|---|---|
|
Beta-thalassemia Major |
Genetic: LentiGlobin® BB305 Drug Product |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: 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 β-A(T87Q)-Globin Vector (LentiGlobin® BB305 Drug Product) |
Resource links provided by NLM:
Further study details as provided by bluebird bio:
Primary Outcome Measures:
- 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 [β-A(T87Q)-globin] [ Time Frame: 18 - 24 months post-transplant ] [ Designated as safety issue: No ]
- 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 ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- Hematopoietic stem cell engraftment [ Time Frame: 42 days post-transplant ] [ Designated as safety issue: Yes ]
- 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 ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 15 |
| Study Start Date: | July 2013 |
| Estimated Primary Completion Date: | July 2017 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: LentiGlobin® BB305 Drug Product |
Genetic: LentiGlobin® BB305 Drug Product
Transplant of autologous hematopoietic stem cells transduced with LentiGlobin® BB305 lentiviral vector.
|
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.
Eligibility| Ages Eligible for Study: | 18 Years to 35 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion criteria:
- Adult subjects between 18 and 35 years of age, inclusive, at the time of consent, and able to provide written consent.
- 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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01745120
Contacts
| Contact: Alexandria Petrusich | clinicaltrials@bluebirdbio.com |
Sponsors and Collaborators
bluebird bio
Investigators
| Study Director: | Rob Ross, MD | bluebird bio |
More Information
No publications provided
| Responsible Party: | bluebird bio |
| ClinicalTrials.gov Identifier: | NCT01745120 History of Changes |
| Other Study ID Numbers: | HGB-204 |
| Study First Received: | December 6, 2012 |
| Last Updated: | May 13, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by bluebird bio:
|
gene therapy beta thalassemia hemoglobin anemia CD34 |
Additional relevant MeSH terms:
|
Beta-Thalassemia Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic |
Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |
ClinicalTrials.gov processed this record on May 16, 2013