Clinical trial NCT06271512: A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
Official title: A Safety and Effectiveness Registry Study of Patients with Β-Thalassemia Treated with Betibeglogene Autotemcel (the Glostar Registry)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Beta-Thalassemia
Lead sponsor: bluebird bio
Sponsor class: INDUSTRY
Collaborators: name: Center for International Blood and Marrow Transplant Research
First posted: 2024-02-21
Enrollment: 150
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with β-thalassemia treated with beti-cel in the post marketing setting at a center in the United States (US) that participates in the Registry.

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)
timeFrame: Through 15 years post-beti-cel infusion
Secondary outcomes:
measure: Number of Participants with Serious Adverse Events (SAEs)
timeFrame: Through 15 years post-beti-cel infusion
measure: Number of Participants with beti-cel related AEs
timeFrame: Through 15 years post-beti-cel infusion
measure: Event-Free Survival
timeFrame: Through 15 years post-beti-cel infusion
measure: Percentage of Participants Achieving Transfusion Independence
timeFrame: Through 15 years post-beti-cel infusion
Other outcomes:
Locations:
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospitals
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: New Hyde Park
country: United States
facility: Cohen Children's Medical Center
state: New York
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
Study officials:
affiliation: bluebird bio, Inc.
name: Himal Lal Thakar, MD
role: STUDY_DIRECTOR
References: