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A Study of Betibeglogene Autotemcel for Beta-Thalassemia

This study is looking at the long-term safety and effectiveness of betibeglogene autotemcel (beti-cel) in people with beta-thalassemia. Beti-cel is a treatment you would have already received. Researchers want to understand how you are doing over time after your treatment, including any side effects or new health problems, and how well the treatment continues to work. This is an observational study, meaning you won't receive any new interventions as part of the study. The study aims to enroll 150 participants. To join, you must have already been treated with beti-cel at a participating center in the US and agree to share your health information.

Study design
This is an observational study that plans to include 150 participants. It is designed to collect real-world data on people who have already received betibeglogene autotemcel.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for up to 15 years after their beti-cel infusion to track adverse events.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06271512

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

Recruiting
Not specifiedAll AgesObservational
Genetix Biotherapeutics Inc.
~150 participants
Updated 2025-02-11 on ClinicalTrials.gov
What's tested:No Intervention

At a glance

Recruiting sites
6 of 6 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)
Measured over Through 15 years post-beti-cel infusion

Conditions

Beta-Thalassemia

Explore related trials

Beta Thalassemia

Where it's being run

6 sites across 5 states
California2
Minnesota1
New York1
Pennsylvania1
Texas1

Study leadership

  • Himal Lal Thakar, MD · STUDY_DIRECTOR · bluebird bio, Inc.

Who to contact

bluebird bio
[email protected]
+1-833-999-6378
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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.

What this trial measures

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