NCT06271512Recruiting
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
Genetix Biotherapeutics Inc.
Start Date
1/23/2024
Completion Date
12/1/2043
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 Criteria
Age Range: No minimum to No maximum
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 participation.
Interventions
OTHER
No Intervention
Conditions
Beta-Thalassemia
Locations
UCSF Benioff Children's Hospitals
Oakland, California 94609
United States
Stanford University
Palo Alto, California 94043
United States
University of Minnesota
Minneapolis, Minnesota 55455
United States
Cohen Children's Medical Center
New Hyde Park, New York 11040
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
Texas Children's Hospital
Houston, Texas 77030
United States