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

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

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 Overview

• Status: Recruiting
• Conditions: Beta-Thalassemia
• Intervention / Treatment: Other: No Intervention

• Study Type: Observational
• Enrollment (Estimated): 150

Contacts and Locations

• Study Contact: Name: bluebird bio; Phone Number: +1-833-999-6378; Email: clinicaltrials@bluebirdbio.com

Study Locations

• United States
  • California
    • Oakland, California, United States, 94609
      • Recruiting
      • UCSF Benioff Children's Hospitals

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study 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.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
All Participants; 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.	Other: No Intervention; This is non-interventional study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Through 15 years post-beti-cel infusion

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: bluebird bio
• Collaborators: Center for International Blood and Marrow Transplant Research
• Investigators: Study Director: Himal Lal Thakar, MD, bluebird bio, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 23, 2024
• Primary Completion (Estimated): December 1, 2043
• Study Completion (Estimated): December 1, 2043

Study Registration Dates

• First Submitted: February 14, 2024
• First Submitted That Met QC Criteria: February 14, 2024
• First Posted (Estimated): February 21, 2024

Study Record Updates

• Last Update Posted (Estimated): February 21, 2024
• Last Update Submitted That Met QC Criteria: February 14, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; beta-Thalassemia
• Other Study ID Numbers: REG-501

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: bluebird bio is committed to transparency. Appropriately de-identified participant-level datasets and supporting documents may be shared (if bluebird bio is contractually permitted to do so) following completion of this study, completion of all applicable regulatory submissions and consistent with criteria established by bluebird bio, our collaborators and/or industry best practices to protect confidential information and maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No