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NCT06271512 · ClinicalTrials.gov registry record
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
A clinical trial of Beta-Thalassemia, sponsored by Genetix Biotherapeutics.
- Recruiting
- Registry status
- 150
- Enrollment target
- 6
- Study locations
NCT06271512: Recruiting study of Beta-Thalassemia, sponsored by Genetix Biotherapeutics.
NCT06271512 is a study of Beta-Thalassemia that is actively recruiting participants, run by Genetix Biotherapeutics. The registered enrollment target is 150 participants, below the 170-participant average among 14 other Beta-Thalassemia trials with a reported enrollment target (12% lower). The trial reports 6 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06271512, a study of Beta-Thalassemia, is actively recruiting participants, sponsored by Genetix Biotherapeutics.
- RECRUITING
- Registry status
- 150 participants
- Enrollment target
- 6
- Study locations
Study 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.
Primary Outcome
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 *
Conditions Studied
Interventions
- OTHER No Intervention
Study Locations (6)
California
- UCSF Benioff Children's Hospitals - Oakland
- Stanford University - Palo Alto
Minnesota
- University of Minnesota - Minneapolis
New York
- Cohen Children's Medical Center - New Hyde Park
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Texas
- Texas Children's Hospital - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 150 participants |
| Start Date | 2024-01-23 |
| Est. Completion | 2043-12 |
What NCT06271512 shows while recruiting
NCT06271512 is an observational study that tracks outcomes without assigning an intervention. The registered 150 participants enrollment target is mid-sized for trials with a published cap, below the 170-participant average among 14 other Beta-Thalassemia trials with a reported enrollment target (12% lower).
The record links to 1 condition, with Beta-Thalassemia appearing as the primary indexed condition, and to 1 intervention - of which No Intervention is the first listed.
NCT06271512 lists 6 locations in 5 states (California, Minnesota, New York).
Frequently Asked Questions
What is clinical trial NCT06271512 about?
NCT06271512 is a clinical study titled "A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel". 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 a...
What is the current status of trial NCT06271512?
This trial is currently recruiting. The enrollment target is 150 participants. The study started on 2024-01-23. Estimated completion is 2043-12.
What conditions does trial NCT06271512 study?
This clinical trial studies the following conditions: Beta-Thalassemia.
What interventions are being tested in trial NCT06271512?
The interventions under investigation include: No Intervention (OTHER).
Who is sponsoring clinical trial NCT06271512?
This trial is sponsored by Genetix Biotherapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06271512 being conducted?
This trial has 6 study locations across California, Minnesota, New York, Pennsylvania, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT06271512's enrollment target sits among peer trials
150 4th of 14 higher than 11 of 14 other Beta-Thalassemia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Beta-Thalassemia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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