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

Last updated: February 10, 2025
Sponsor: bluebird bio
Overall Status: Active - Recruiting

Phase

N/A

Condition

Thalassemia

Treatment

No Intervention

Clinical Study ID

NCT06271512
REG-501
  • All Genders

Study Summary

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 Criteria

Inclusion

Inclusion Criteria:

  • Participant must be treated with beti-cel in the post marketing setting at a centerin the US that participates in the Registry.

  • Participant must sign an informed consent and/or assent prior to enrollment asrequired under applicable laws and regulations.

  • Participant must have signed an informed consent and/or assent permitting data to beshared with Center for International Blood and Marrow Transplant Research (CIBMTR).

  • Participant must be followed by a hematologist based in the US.

Exclusion

Exclusion Criteria:

  • There are no exclusion criteria for Registry participation.

Study Design

Total Participants: 150
Treatment Group(s): 1
Primary Treatment: No Intervention
Phase:
Study Start date:
January 23, 2024
Estimated Completion Date:
December 31, 2043

Connect with a study center

  • UCSF Benioff Children's Hospitals

    Oakland, California 94609
    United States

    Active - Recruiting

  • Stanford University

    Palo Alto, California 94043
    United States

    Active - Recruiting

  • University of Minnesota

    Minneapolis, Minnesota 55455
    United States

    Active - Recruiting

  • Cohen Children's Medical Center

    New Hyde Park, New York 11040
    United States

    Active - Recruiting

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Texas Children's Hospital

    Houston, Texas 77030
    United States

    Active - Recruiting

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