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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

150 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-01-23

Study Completion Date

2043-12-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Beta-Thalassemia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

No Intervention

Intervention Type OTHER

This is non-interventional study.

Interventions

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No Intervention

This is non-interventional study.

Intervention Type OTHER

Eligibility Criteria

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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.

Eligible Sex

ALL

Accepts Healthy Volunteers

No