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

NCT06271512 | Recruiting

• 1 other identifier: REG-501
• Study Type: observational
• Target: 150
• Locations: 1 country
• Sites: 6

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.

Conditions

Beta-Thalassemia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• 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

Study Arms (1)

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

Interventions

No Intervention OTHER

This is non-interventional study.

All Participants

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

Sponsors & Collaborators

• Genetix Biotherapeutics Inc.: lead
• Center for International Blood and Marrow Transplant Research: collaborator

Study Sites (6)

UCSF Benioff Children's Hospitals

Oakland, California, 94609, United States

RECRUITING

Stanford University

Palo Alto, California, 94043, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

Cohen Children's Medical Center

New Hyde Park, New York, 11040, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Texas Children's Hospital

Houston, Texas, 77030, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Bone marrow and whole blood collected at the time of bone marrow sample collection will be stored centrally to facilitate subsequent exploratory analyses that may include gene/genomic expression studies.

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Himal Lal Thakar, MD

  bluebird bio, Inc.

  STUDY DIRECTOR

Central Study Contacts

bluebird bio

CONTACT

+1-833-999-6378; clinicaltrials@bluebirdbio.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 15 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 14, 2024
• First Posted: February 21, 2024
• Study Start: January 23, 2024
• Primary Completion (Estimated): December 1, 2043
• Study Completion (Estimated): December 1, 2043
• Last Updated: February 11, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will share

Locations

US (6)