Active not recruitingOBSERVATIONAL

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

This study is a long-term follow-up for individuals who have previously received a special type of gene therapy for a blood condition known as transfusion-dependent beta-thalassemia (TDT). Beta-thalassemia makes your body unable to produce enough healthy red blood cells, often requiring regular blood transfusions. The gene therapy aims to help the body make its own healthy red blood cells. This study carefully checks on patients who have already had this treatment, looking at their health and how well the treatment is working over many years. It is important to understand that no new gene therapy treatment is given in this study. It's purely about observing how patients are doing in the long run after their initial treatment. The goal is to gather more information about the treatment's lasting safety and benefits.

At a glance

Status

Active not recruiting

Sponsor

Genetix Biotherapeutics Inc.

Enrolment target

Start

01 Jan 2014

Estimated completion

01 Nov 2035

Transfusion-dependent Beta-Thalassemia

What is this study about?

If you or someone you know has transfusion-dependent beta-thalassemia, you might be interested to know about this study. Beta-thalassemia is a serious inherited blood disorder where the body cannot make enough healthy red blood cells. This often means people need regular blood transfusions, which can be life-saving but also come with their own challenges over time.

Some people with this condition have received a new type of treatment called ex vivo gene therapy. Ex vivo means that some of your own cells were taken out of your body, changed in a laboratory using gene therapy, and then put back into your body. The aim of this gene therapy is to help your body produce its own healthy red blood cells, potentially reducing or eliminating the need for blood transfusions.

This particular study is not about giving new treatment. Instead, it's a very important follow-up for people who have already had this gene therapy in previous studies. The researchers want to understand how these patients are doing over a long period, typically for about 15 years after they received the gene therapy. By carefully monitoring their health and how well the treatment continues to work, doctors can learn more about the long-term benefits and any potential delayed effects of this new type of treatment. This information is crucial for future patients and for understanding how best to use gene therapy for beta-thalassemia.

Key takeaways

You won't get new gene therapy in this study; it's for long-term health checks.
The study looks at people who have already had gene therapy for beta-thalassemia.
It aims to understand how the treatment works and its safety over many years.
Participation helps gather important information for future patients and treatments.
You'll be followed for up to 15 years from when you first received the gene therapy.

Who may be eligible?

This study is specifically designed for people who have already taken part in a gene therapy trial for transfusion-dependent beta-thalassemia. To join this follow-up study, you must have previously received the gene therapy treatment through a bluebird bio-sponsored clinical study.

Also, you (or your parents/guardians, if you are a child) need to be willing to give your written permission to take part in this study. This is called informed consent, and it ensures you understand what the study involves before you agree to participate.

There are no other specific features that would prevent you from joining this follow-up study if you meet the main criteria mentioned above. Both males and females, aged anywhere from 0 to 50 years, who have received the specific gene therapy, can be considered.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Have I already received gene therapy for transfusion-dependent beta-thalassemia in a bluebird bio study?
Am I willing to give my written permission to join the study?
Am I between 0 and 50 years old?
Could I attend follow-up appointments for approximately 13 more years?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will not receive any new medication or gene therapy. Your participation will involve a series of check-ups over an extended period. You will have regular visits where doctors will monitor your health to see how you are doing after your gene therapy.

These check-ups will usually involve assessments to look at your general health, how well your bone marrow is working, and to ensure the gene therapy is still effective. Researchers will also be looking out for any potential long-term side effects. These check-ups will continue for approximately 13 years after your previous study ended, meaning you will be followed for a total of about 15 years from when you first received the gene therapy. The schedule of visits will be explained in detail if you are eligible.

Potential risks and benefits

The main benefit of participating in this study is contributing to valuable knowledge about the long-term safety and effectiveness of gene therapy for beta-thalassemia. This helps future patients and supports the development of better treatments. Since no new treatment is given, the direct risks are generally low, focusing on risks associated with clinic visits and any procedures for monitoring your health, such as blood tests. You always have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (15)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Oakland, United States
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Chicago, United States
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Bethesda, United States
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New York, United States
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Philadelphia, United States
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Charleston, United States
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Sydney, Australia
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Marseille, France
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Paris, France
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Hanover, Germany
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Heidelberg, Germany
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Thessaloniki, Greece

Common questions

What is long-term follow-up?

It means doctors will keep checking on your health and how the treatment is working for many years after you first received it.

Will I get any new treatment in this study?

No, this study is only for observation. You won't receive any new gene therapy or medication as part of this follow-up.

How long will I be in this study?

You will be followed for about 13 years after your previous study finished, making a total of about 15 years since you had the gene therapy.

Why is this study important?

It helps doctors understand how safe and effective the gene therapy is over a long time, which is important for people with beta-thalassemia.

Can children join this study?

Yes, individuals from birth up to 50 years of age can join, provided they meet the other criteria.