Observational study on patients with Beta-Thalassemia treated with betibeglogene autotemcel

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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).

Why it matters

Who can join: the full rules

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 it's running

• 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

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.