Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Center for International Blood and Marrow Transplant Research | NETWORK |
Not provided
Not provided
Not provided
The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All Participants | Participants with β-thalassemia treated with beti-cel in the post-marketing setting will be followed in this registry study for up to 15 years after infusion with beti-cel to collect real-world longitudinal data. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No Intervention | Other | This is non-interventional study. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs) | The Sponsor considers the following events to be AEIs (which should be reported as a medically significant Serious Adverse Event [SAE]):
| Through 15 years post-beti-cel infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Serious Adverse Events (SAEs) | Through 15 years post-beti-cel infusion | |
| Number of Participants with beti-cel related AEs | Through 15 years post-beti-cel infusion | |
Not provided
Inclusion Criteria:
Exclusion Criteria:
- There are no exclusion criteria for Registry participation.
Not provided
Not provided
Not provided
Not provided
Participants with β-thalassemia treated with beti-cel in the post marketing setting at a center in the United States (US) that participates in the Registry.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| bluebird bio | Contact | +1-833-999-6378 | clinicaltrials@bluebirdbio.com |
| Name | Affiliation | Role |
|---|---|---|
| Himal Lal Thakar, MD | bluebird bio, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Benioff Children's Hospitals | Recruiting | Oakland | California | 94609 | United States | |
bluebird bio is committed to transparency. Appropriately de-identified participant-level datasets and supporting documents may be shared (if bluebird bio is contractually permitted to do so) following completion of this study, completion of all applicable regulatory submissions and consistent with criteria established by bluebird bio, our collaborators and/or industry best practices to protect confidential information and maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D017086 | beta-Thalassemia |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
Not provided
Not provided
Not provided
Not provided
Not provided
Bone marrow and whole blood collected at the time of bone marrow sample collection will be stored centrally to facilitate subsequent exploratory analyses that may include gene/genomic expression studies.
| Event-Free Survival |
| Through 15 years post-beti-cel infusion |
| Percentage of Participants Achieving Transfusion Independence | Through 15 years post-beti-cel infusion |
| Stanford University |
| Recruiting |
| Palo Alto |
| California |
| 94043 |
| United States |
| University of Minnesota | Recruiting | Minneapolis | Minnesota | 55455 | United States |
| Cohen Children's Medical Center | Recruiting | New Hyde Park | New York | 11040 | United States |
| Children's Hospital of Philadelphia | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
| Texas Children's Hospital | Recruiting | Houston | Texas | 77030 | United States |
| D006402 |
| Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |