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

a study on Beta Thalassemia

Summary

Location
at UCSF
Dates
study started
study ends around
Principal Investigator
by Mark Walters (ucsf)
Headshot of Mark Walters
Mark Walters

Description

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.

Official Title

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

Keywords

Beta-Thalassemia

Eligibility

You can join if…

  • 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.

You CAN'T join if...

  • There are no exclusion criteria for Registry participation.

Locations

  • UCSF Benioff Children's Hospitals accepting new patients
    Oakland California 94609 United States
  • Stanford University accepting new patients
    Palo Alto California 94043 United States

Lead Scientist at University of California Health

  • Mark Walters (ucsf)
    The over-arching goal of my research career has been to develop and expand curative therapies for hemoglobin disorders in particular and non-malignant hematopoietic disorders more broadly. I have focused on genome editing approaches most recently, which rely upon modification of autologous hematopoietic cells to elicit a curative effect.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Genetix Biotherapeutics Inc.
ID
NCT06271512
Study Type
Observational [Patient Registry]
Participants
Expecting 150 study participants
Last Updated
Contact us about this trial