Recruiting

Betibeglogene Autotemcel

Sponsor:

bluebird bio

Code:

NCT06271512

Conditions

Beta-Thalassemia

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

No Intervention

Study Details

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

Study ID

NCT06271512

Start date

Jan 23, 2024

Status verified date

Feb, 2025

Completion date

Dec, 2043

Anticipated

Primary completion date

Dec, 2043

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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 Design

Enrollment

150 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

Interventions

No Intervention

This is non-interventional study.

Primary outcome measure

  • Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs) [ Time Frame: Through 15 years post-beti-cel infusion ]

Central Contacts and Locations

Central contacts

bluebird bio

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

Locations

UCSF Benioff Children's Hospitals

Recruiting

Oakland, California, United States, 94609

Stanford University

Recruiting

Palo Alto, California, United States, 94043

University of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55455

Cohen Children's Medical Center

Recruiting

New Hyde Park, New York, United States, 11040

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Texas Children's Hospital

Recruiting

Houston, Texas, United States, 77030

More Information

Sponsor

bluebird bio

Last update posted

Feb 11, 2025

Last verified

Feb, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by bluebird bio on 2025-02-11.