A Study of Participants With β-Thalassemia Treated With Betibeglogene Autotemcel

Beta-Thalassemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06271512
• Other study ID #: REG-501
• Status: Recruiting
• Start date: January 23, 2024
• Est. completion date: December 2043

Study information

• Verified date: February 2024
• Source: bluebird bio
• Contact: bluebird bio
• Phone: +1-833-999-6378
• Email: clinicaltrials[@]bluebirdbio.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: December 2043
• Est. primary completion date: December 2043
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Beta-Thalassemia
• Thalassemia

Intervention

Other:
• No Intervention
This is non-interventional study.

Locations

Country	Name	City	State
United States	UCSF Benioff Children's Hospitals	Oakland	California

Sponsors (2)

Lead Sponsor	Collaborator
bluebird bio	Center for International Blood and Marrow Transplant Research

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Number of Participants with Serious Adverse Events (SAEs)		Through 15 years post-beti-cel infusion
Secondary	Number of Participants with beti-cel related AEs		Through 15 years post-beti-cel infusion
Secondary	Event-Free Survival		Through 15 years post-beti-cel infusion
Secondary	Percentage of Participants Achieving Transfusion Independence		Through 15 years post-beti-cel infusion