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

Bluebird Bio

Status: Enrolling

Conditions: Beta-Thalassemia

Treatments: Other: No Intervention

Study type: Observational

Funder types: NETWORK; Industry

Identifiers

NCT06271512

REG-501

Details and patient eligibility

About

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.

Enrollment: 150 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

150 participants in 1 patient group

All Participants

Description:

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.

Treatment:

Other: No Intervention