A Study of Participants With β-Thalassemia Treated With Betibeglogene Autotemcel

Recruiting

• Conditions: Beta-Thalassemia
• Interventions: Other: No Intervention

• Registration Number: NCT06271512
• Lead Sponsor: bluebird bio

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2024-01-23
• Study First Submitted: 2024-02-14
• Study First Submitted QC: 2024-02-14
• Study First Posted: 2024-02-21
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-30
• Last Update Posted: 2024-07-31
• Primary Completion: 2043-12
• Study Completion: 2043-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 150

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.

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Exclusion Criteria

• There are no exclusion criteria for Registry participation.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
All Participants	No Intervention	Participants with β-thalassemia treated with beti-cel in the post-marketing setting will be followed in this registry study for up to 15 years after infusion with beti-cel to collect real-world longitudinal data.

Primary Outcome Measures

Name	Time	Method
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.

Secondary Outcome Measures

Name	Time	Method
Number of Participants with Serious Adverse Events (SAEs)	Through 15 years post-beti-cel infusion
Number of Participants with beti-cel related AEs	Through 15 years post-beti-cel infusion
Event-Free Survival	Through 15 years post-beti-cel infusion
Percentage of Participants Achieving Transfusion Independence	Through 15 years post-beti-cel infusion

Trial Locations

Locations (5)

University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States

UCSF Benioff Children's Hospitals; 🇺🇸 Oakland, California, United States

Cohen Children's Medical Center; 🇺🇸 New Hyde Park, New York, United States

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Texas Children's Hospital; 🇺🇸 Houston, Texas, United States