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A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

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Phase N/A

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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.

Eligibility

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.

Study details
    Beta-Thalassemia

NCT06271512

Genetix Biotherapeutics Inc.

15 October 2025

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