A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: No Intervention.
- Who it may be relevant to
- Registry conditions: Beta-Thalassemia. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Safety and Effectiveness Registry Study of Patients with Β-Thalassemia Treated with Betibeglogene Autotemcel (the Glostar Registry)
Overview
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
- Other No Intervention
This is non-interventional study.
Primary outcome measures
- Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs) [Time frame: Through 15 years post-beti-cel infusion]
Secondary outcome measures (4)
- Number of Participants with Serious Adverse Events (SAEs) [Time frame: Through 15 years post-beti-cel infusion]
- Number of Participants with beti-cel related AEs [Time frame: Through 15 years post-beti-cel infusion]
- Event-Free Survival [Time frame: Through 15 years post-beti-cel infusion]
- Percentage of Participants Achieving Transfusion Independence [Time frame: Through 15 years post-beti-cel infusion]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 6 centers
- UCSF Benioff Children's Hospitals — Oakland
- Stanford University — Palo Alto
- University of Minnesota — Minneapolis
- Cohen Children's Medical Center — New Hyde Park
- Children's Hospital of Philadelphia — Philadelphia
- Texas Children's Hospital — Houston
Identifiers
NCT: NCT06271512 · REG-501