A Safety and Effectiveness Registry Study of Patients with Β-Thalassemia Treated with Betibeglogene Autotemcel (the Glostar Registry)
Genetix Biotherapeutics Inc.
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.
Eligibility
- Age range
- Not specified
- Sex
- 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 exclusion criteria for Registry participatio…
Interventions
- OtherNo Intervention
This is non-interventional study.
Locations (6)
- UCSF Benioff Children's HospitalsOakland, California
- Stanford UniversityPalo Alto, California
- University of MinnesotaMinneapolis, Minnesota
- Cohen Children's Medical CenterNew Hyde Park, New York
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania
- Texas Children's HospitalHouston, Texas