Betibeglogene Autotemcel for Beta Thalassemia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to evaluate the long-term effects of betibeglogene autotemcel, or beti-cel, for individuals with β-thalassemia, a blood disorder that reduces hemoglobin levels. Researchers seek to understand the long-term safety and effectiveness of this treatment, particularly any new health issues, such as cancer, that might arise after using beti-cel. The trial is open to U.S. residents who have already received beti-cel and are under the care of a blood specialist. Participants will be monitored for up to 15 years to collect real-world data on their health outcomes. As a Phase 4 trial, this study focuses on understanding how the FDA-approved and proven effective treatment benefits more patients, providing valuable insights into its long-term impact.
Do I need to stop my current medications for this trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
What is the safety track record for this treatment?
Research has shown that betibeglogene autotemcel, or beti-cel, has undergone extensive study to ensure its safety for humans. Patients who received beti-cel often maintained normal hemoglobin levels, reducing the need for regular blood transfusions.
Regarding safety, studies have monitored patients for up to 10 years after treatment. These studies indicate that the treatment is generally well-tolerated. While all medical treatments can have side effects, no new major safety concerns have been specifically linked to beti-cel. This long-term data reassures those considering participation in a clinical trial for this treatment.12345Why are researchers enthusiastic about this study treatment?
Betibeglogene autotemcel, also known as beti-cel, is unique because it offers a gene therapy approach for treating β-thalassemia. Unlike traditional treatments like regular blood transfusions and iron chelation therapy, beti-cel works by modifying the patient's own stem cells to produce functional hemoglobin. This means that it targets the root cause of the disease rather than just managing symptoms. Researchers are excited because this treatment has the potential to provide a long-term solution and significantly reduce or eliminate the need for frequent transfusions, improving the quality of life for patients.
What is the effectiveness track record for beti-cel in treating β-thalassemia?
Research has shown that betibeglogene autotemcel (beti-cel) is a promising treatment for β-thalassemia. Studies have found that patients receiving beti-cel often no longer need regular blood transfusions for extended periods. This occurs because the treatment helps the body produce normal levels of hemoglobin, the protein in blood that carries oxygen. Follow-up data for up to 10 years supports the lasting effects of these benefits. Overall, beti-cel offers a potentially curative approach for those affected by β-thalassemia. Participants in this trial will be monitored for up to 15 years after infusion with beti-cel to collect real-world longitudinal data.12356
Who Is on the Research Team?
Himal Lal Thakar, MD
Principal Investigator
bluebird bio, Inc.
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive betibeglogene autotemcel (beti-cel) infusion
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Betibeglogene Autotemcel
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
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.
Find a Clinic Near You
Who Is Running the Clinical Trial?
bluebird bio
Lead Sponsor
Genetix Biotherapeutics Inc.
Lead Sponsor
Center for International Blood and Marrow Transplant Research
Collaborator
Citations
The effectiveness and value of betibeglogene autotemcel ...
Betibeglogne autotemcel (beti-cel, bluebird bio) is a potentially curative gene therapy for β-thalassemia that utilizes a lentiviral vector.
2.
investor.bluebirdbio.com
investor.bluebirdbio.com/news-releases/news-release-details/long-term-follow-data-continue-support-beti-cel-potentiallyLong-Term Follow-Up Data Continue to Support Beti-Cel ...
Updated follow-up data of up to 10 years showed that patients treated with beti-cel in clinical trials experienced durable transfusion independence and normal ...
3.
ashpublications.org
ashpublications.org/blood/article/144/Supplement%201/2194/532667/Betibeglogene-Autotemcel-beti-cel-Gene-AdditionBetibeglogene Autotemcel (beti-cel) Gene Addition Therapy ...
Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion- ...
Betibeglogene Autotemcel Gene Therapy for Non–β 0 / ...
Treatment with beti-cel resulted in a sustained HbA T87Q level and a total hemoglobin level that was high enough to enable transfusion independence.
Betibeglogene Autotemcel for Beta Thalassemia
Safety outcomes that were evaluated included hepatic veno-occlusive disease, pyrexia, stomatitis, thrombocytopenia, neutropenia, anemia and.
Betibeglogene autotemcel gene therapy in patients with ...
Beti-cel offers the potential to attain near-normal haemoglobin levels for those with severe forms of TDT, and a potentially curative option ...
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