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NCT06271512Recruiting

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

Genetix Biotherapeutics Inc.

Start Date

1/23/2024

Completion Date

12/1/2043

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

Age Range: No minimum to No maximum

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.

Interventions

OTHER

No Intervention

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Conditions

Beta-Thalassemia

Locations

UCSF Benioff Children's Hospitals

Oakland, California 94609

United States

Stanford University

Palo Alto, California 94043

United States

University of Minnesota

Minneapolis, Minnesota 55455

United States

Cohen Children's Medical Center

New Hyde Park, New York 11040

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

Texas Children's Hospital

Houston, Texas 77030

United States