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

Genetix Biotherapeutics Inc.150 enrolled

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.

Study Type

OBSERVATIONAL

Enrollment

150

Conditions

Beta-Thalassemia

Interventions

No InterventionOTHER

This is non-interventional study.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (6)

UCSF Benioff Children's Hospitals

Oakland, California, United States

RECRUITING

Stanford University

Palo Alto, California, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, United States

Outcomes

Primary Outcomes

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

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.