Observational study on patients with Beta-Thalassemia treated with betibeglogene autotemcel
A Safety and Effectiveness Registry Study of Patients with Β-Thalassemia Treated with Betibeglogene Autotemcel (the Glostar Registry)
This study is looking at how well betibeglogene autotemcel works and its long-term safety for people with beta-thalassemia who have already received this treatment.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 150 (estimated) |
| Sex | All |
| Sponsor | Genetix Biotherapeutics Inc. Industry-sponsored |
| Locations | 6 sites (Oakland, California and 5 other locations) |
| Trial ID | NCT06271512 on ClinicalTrials.gov |
What this trial studies
This study aims to collect real-world longitudinal data on individuals with β-thalassemia who have been treated with betibeglogene autotemcel (beti-cel) in a post-marketing setting. It focuses on assessing the long-term safety of the treatment, including the risk of newly diagnosed malignancies, as well as evaluating its long-term effectiveness. Participants will be followed by hematologists at participating centers in the US, and their data will be shared with the Center for International Blood and Marrow Transplant Research (CIBMTR).
Who should consider this trial
Good fit: Ideal candidates for this study are individuals with β-thalassemia who have been treated with beti-cel and are being followed by a hematologist in the US.
Not a fit: Patients who have not received beti-cel treatment or are not being followed by a hematologist in the US may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term safety and effectiveness of beti-cel treatment for patients with β-thalassemia.
How similar studies have performed: Other studies have shown success in collecting real-world data on gene therapies, making this approach promising but not entirely novel.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Oakland, California and 5 other locations
- UCSF Benioff Children's Hospitals — Oakland, California, United States (Recruiting)
- Stanford University — Palo Alto, California, United States (Recruiting)
- University of Minnesota — Minneapolis, Minnesota, United States (Recruiting)
- Cohen Children's Medical Center — New Hyde Park, New York, United States (Recruiting)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
- Texas Children's Hospital — Houston, Texas, United States (Recruiting)
Study contacts
- Study coordinator: bluebird bio
- Email: clinicaltrials@bluebirdbio.com
- Phone: +1-833-999-6378
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.