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

Description

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

Study Overview

Study Details

Study overview

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.

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

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

Condition
Beta-Thalassemia
Intervention / Treatment

-

Contacts and Locations

Oakland

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

Minneapolis

University of Minnesota, Minneapolis, Minnesota, United States, 55455

New Hyde Park

Cohen Children's Medical Center, New Hyde Park, New York, United States, 11040

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

Houston

Texas Children's Hospital, Houston, Texas, United States, 77030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

bluebird bio,

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

Study Record Dates

2043-12