A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
ConditionBeta-Thalassemia
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorGenetix Biotherapeutics Inc.
About this trial
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
Qualifiers
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.
Disqualifiers
None
Trial design
Treatments tested in this trial
- No Intervention
Treatment groups
150 Participants
are divided into 1 treatment groupSponsors and collaborators
Genetix Biotherapeutics Inc.
Lead sponsor
Center for International Blood and Marrow Transplant Research
Collaborator
Source ClinicalTrials.gov