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

Part of paid clinical trials in Oakland, California.

Sponsor
Genetix Biotherapeutics Inc.
Study ID
NCT06271512
Status
Recruiting
Apply to this trial

Conditions

  • Beta-Thalassemia

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • No Intervention — OTHER
    This is non-interventional study.

Study Details

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.

Key Dates

Start date
Jan 23, 2024
Status verified
Feb 2025
Primary completion
Dec 31, 2043
Completion
Dec 31, 2043

Study Design

Enrollment
150 participants (estimated)

Arms

  • Arm: 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.

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

Locations (6)

FacilityCityStateZIPSite coordinators
UCSF Benioff Children's HospitalsOaklandCalifornia94609-
Stanford UniversityPalo AltoCalifornia94043-
University of MinnesotaMinneapolisMinnesota55455-
Cohen Children's Medical CenterNew Hyde ParkNew York11040-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
Texas Children's HospitalHoustonTexas77030-

Find similar trials in Oakland, CA

By research site
UCSF Benioff Children's Hospitals· Oakland, CAStanford University· Palo Alto, CAUniversity of Minnesota· Minneapolis, MNCohen Children's Medical Center· New Hyde Park, NYChildren's Hospital of Philadelphia· Philadelphia, PATexas Children's Hospital· Houston, TX

Related Studies