A single arm, open label, multicenter, single-dose, phase 2b clinical study evaluating efficacy and safety of gene therapy using autologous CD34+ hematopoietic stem cells transduced with the GLOBE lentiviral vector using an improved transduction protocol in subjects with transfusion-dependent beta-thalassemia.
This research is looking at a new type of treatment called gene therapy for adults who have beta-thalassemia and rely on regular blood transfusions. The therapy involves using a patient's own blood stem cells, which are specially modified in the lab. These modified cells are then put back into the patient. Doctors want to find out how safe and effective this new treatment is. The main aim is to see if participants can stop needing blood transfusions for at least 12 months after receiving the therapy. This study will also check for any side effects and how patients are doing overall for up to two years after treatment.
At a glance
What is this study about?
This study is about a new and exciting gene therapy for adults with beta-thalassemia. Beta-thalassemia is a condition where the body doesn't make enough healthy red blood cells, which carry oxygen around the body. People with severe beta-thalassemia often need regular blood transfusions to stay healthy.
The gene therapy being tested works by taking some of your own special blood cells, called stem cells, from your body. These cells are then changed in a lab using a harmless ‘delivery system’ (called a lentiviral vector) to carry a working copy of the gene that makes healthy red blood cells. Once these improved cells are ready, they are given back to you through a drip. The hope is that these new cells will start producing healthy red blood cells, reducing or even ending your need for transfusions.
The main goal of the study is to see if this treatment can help people stop needing blood transfusions for at least a year. The doctors will carefully watch your blood levels to see if your body is making enough healthy red blood cells on its own. They will also be looking very closely at how safe the treatment is and if there are any side effects. This research could be a big step forward for people living with beta-thalassemia.
Key takeaways
- This study evaluates a new gene therapy for adults with transfusion-dependent beta-thalassemia.
- The therapy uses your own modified stem cells to help your body make healthy red blood cells.
- The main aim is to see if participants can stop needing blood transfusions for at least 12 months.
- Safety is a key focus, and participants will be closely monitored for side effects.
- Participation will involve initial treatments, the gene therapy, and long-term follow-up for at least two years.
Who may be eligible?
This study is designed for adults aged 18 and over who have beta-thalassemia and regularly need blood transfusions. Both men and women are welcome to take part.
To be considered for this study, you would undergo some health checks to make sure the treatment is suitable for you. Doctors will need to confirm you meet specific health requirements related to your beta-thalassemia and overall well-being.
Important note: This study is for adults only. If you are under 18, unfortunately, you cannot participate in this particular research.
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.
- Are you 18 years old or older?
- Do you have beta-thalassemia?
- Do you regularly receive blood transfusions for your condition?
- Are you willing to undergo specific medical checks to confirm your suitability?
- Are you able to commit to several years of follow-up appointments?
What does participation involve?
If you decide to take part, you'll first have some preparatory treatments. This includes medication to help your body produce more stem cells, which will then be collected. You'll also receive a medicine called Busulfan, which helps prepare your body for the modified cells. After receiving your specially prepared stem cells through a drip, you'll be closely monitored.
During the study, you will have regular visits to the clinic for blood tests, check-ups, and to see how well the treatment is working. Doctors will especially be looking at your blood count to see if you can reduce or stop your blood transfusions. They will also check for any side effects.
Your participation will involve detailed monitoring after the cell treatment, with follow-up appointments over at least two years to ensure your long-term health and the lasting effects of the therapy are carefully tracked. The total duration of active participation will vary, but regular check-ups will continue for quite some time after the main treatment.
Potential risks and benefits
Locations (1)
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Common questions
What is gene therapy?
Gene therapy is a new type of treatment where doctors add a healthy gene to your body to replace a faulty one. In this study, it aims to help your body make healthy red blood cells.
What is beta-thalassemia?
Beta-thalassemia is a blood condition where your body doesn't produce enough healthy red blood cells, which carry oxygen. This often leads to tiredness and needing blood transfusions.
Will I still need blood transfusions?
The main goal of this study is to see if the gene therapy can help you stop needing blood transfusions for at least 12 months. However, there's no guarantee, and you might still need them.
How long will the study last?
The study involves a period of treatment and then careful monitoring for at least two years to check your health and how the treatment is working long-term.
Is this treatment available to everyone?
No, this is a research study for specific people. You need to be an adult with beta-thalassemia who regularly needs transfusions, and you'll need to meet other health criteria to be eligible.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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