Beta-Thalassemia
Conditions
Brief summary
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.
Interventions
OTHERNo Intervention
This is non-interventional study.
Sponsors
Center for International Blood and Marrow Transplant Research
Genetix Biotherapeutics Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Participant must be treated with beti-cel in the post marketing setting at a center in the US that participates in the Registry. * Participant must sign an informed consent and/or assent prior to enrollment as required under applicable laws and regulations. * Participant must have signed an informed consent and/or assent permitting data to be shared with Center for International Blood and Marrow Transplant Research (CIBMTR). * Participant must be followed by a hematologist based in the US.
Exclusion criteria
\- There are no
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs) | Through 15 years post-beti-cel infusion | The Sponsor considers the following events to be AEIs (which should be reported as a medically significant Serious Adverse Event \[SAE\]): * Any newly diagnosed malignancy * Neutrophil engraftment failure: defined as healthcare provider (HCP) decision to administer back-up cells or subsequent hematopoietic stem cell transplantation (HSCT) due to neutrophil recovery failure * Newly acquired human immunodeficiency virus-1 (HIV-1), HIV-2 infection and human t-lymphotropic virus (HTLV) infection * Any newly diagnosed autoimmune disorder * Any hepatic veno-occlusive disease (VOD) * Any clinically significant bleeding events. |
Secondary
| Measure | 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 |
| Event-Free Survival | Through 15 years post-beti-cel infusion |
| Percentage of Participants Achieving Transfusion Independence | Through 15 years post-beti-cel infusion |
Countries
United States
Contacts
Primary Contactbluebird bio
Outcome results
None listed