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NCT Number: NCT06271512

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

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.

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Key information

Conditions

Beta-Thalassemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia

Sex eligibility

All sexes

Study type

Observational

Primary location

UCSF Benioff Children's Hospitals, Oakland, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

No intervention

Other

This is non-interventional study.

Primary outcomes

  1. Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)

    Time frame: Through 15 years post-beti-cel infusion

    The Sponsor considers the following events to be AEIs (which should be reported as a medically significant Serious Adverse Event [SAE]):

    • Any newly diagnosed malignancy
    • Neutrophil engraftment failure: defined as healthcare provider (HCP) decision to administer back-up cells or subsequent hematopoietic stem cell transplantation (HSCT) due to neutrophil recovery failure
    • Newly acquired human immunodeficiency virus-1 (HIV-1), HIV-2 infection and human t-lymphotropic virus (HTLV) infection
    • Any newly diagnosed autoimmune disorder
    • Any hepatic veno-occlusive disease (VOD)
    • Any clinically significant bleeding events.

Secondary outcomes

  1. Number of Participants with Serious Adverse Events (SAEs)

    Time frame: Through 15 years post-beti-cel infusion

  2. Number of Participants with beti-cel related AEs

    Time frame: Through 15 years post-beti-cel infusion

  3. Event-Free Survival

    Time frame: Through 15 years post-beti-cel infusion

  4. Percentage of Participants Achieving Transfusion Independence

    Time frame: Through 15 years post-beti-cel infusion

Study contacts

Contact information is provided by the study sponsor or research team.

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CONTACT

[email protected]

+1-833-999-6378

Sponsors and collaborators

Lead sponsor

Genetix Biotherapeutics Inc.

Industry

Collaborators

  • Center for International Blood and Marrow Transplant Research

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2043
Study completion
2043
First posted
Feb 21, 2024
Registry last updated
Feb 11, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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