Actively Recruiting
A Safety and Effectiveness Registry Study of Patients with Beta-Thalassemia Treated with Betibeglogene Autotemcel (the Glostar Registry)
Led by Genetix Biotherapeutics Inc. · Updated on 2025-02-11
150
Participants Needed
6
Research Sites
N/A
Total Duration
On this page
Sponsors
G
Genetix Biotherapeutics Inc.
Lead Sponsor
C
Center for International Blood and Marrow Transplant Research
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are collecting long-term real-world data on people with beta-thalassemia who have received betibeglogene autotemcel (beti-cel) treatment after it became available on the market. The study aims to assess the safety of beti-cel over a period of up to 15 years, including monitoring for any new cancers, as well as evaluating how effective the treatment is over time. Participants who have been treated with beti-cel at participating centers in the US will be followed without any additional interventions. This observational registry study tracks patients for up to 15 years after their beti-cel infusion, gathering health information to understand long-term outcomes. During the study, researchers will collect data on any adverse events, serious health issues, and how many participants achieve independence from blood transfusions. All participants will be monitored regularly through their usual medical care with a hematologist in the US. The study focuses on safety and effectiveness outcomes over the long term, with no exclusion criteria for participation.
CONDITIONS
Brief Title
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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.
You will not qualify if you...
- There are no exclusion criteria for Registry participation.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 15 years after infusion
Participants who have been treated with betibeglogene autotemcel are followed to collect real-world longitudinal data regarding safety and effectiveness.
Trial Site Locations
Total: 6 locations
1
UCSF Benioff Children's Hospitals
Oakland, California, United States, 94609
Actively Recruiting
2
Stanford University
Palo Alto, California, United States, 94043
Actively Recruiting
3
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
4
Cohen Children's Medical Center
New Hyde Park, New York, United States, 11040
Actively Recruiting
5
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
6
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
B
bluebird bio
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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