RecruitingNCT06271512

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

A Safety and Effectiveness Registry Study of Patients with Β-Thalassemia Treated with Betibeglogene Autotemcel (the Glostar Registry)


Sponsor

Genetix Biotherapeutics Inc.

Enrollment

150 participants

Start Date

Jan 23, 2024

Study Type

OBSERVATIONAL

Conditions

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

Inclusion Criteria4

  • 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.

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Interventions

OTHERNo Intervention

This is non-interventional study.


Locations(6)

UCSF Benioff Children's Hospitals

Oakland, California, United States

Stanford University

Palo Alto, California, United States

University of Minnesota

Minneapolis, Minnesota, United States

Cohen Children's Medical Center

New Hyde Park, New York, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Texas Children's Hospital

Houston, Texas, United States

View Full Details on ClinicalTrials.gov

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NCT06271512


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