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

ConditionBeta-Thalassemia

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

AgeNot listed

SponsorGenetix Biotherapeutics Inc.

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

None

Trial population

Participants with β-thalassemia treated with beti-cel in the post marketing setting at a center in the United States (US) that participates in the Registry.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

No Intervention
Other intervention
This is non-interventional study.

Treatment groups

150 Participants

are divided into 1 treatment group

Group A: All Participants1 intervention

Trial 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.

Time frame