Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humansInterventional

A clinical gene therapy study with hematopoietic stem cells for the treatment of patients suffering from a malignant neoplasm of the central nervous system

This study explores a new approach called gene therapy for a severe form of brain cancer known as glioblastoma. This therapy involves using special stem cells from the blood to try and target the cancer cells. This is the very first time this treatment is being given to people, so the main goals are to check if it's safe and to understand its effects, rather than being an established treatment. Doctors conducting the research will carefully monitor participants to see how their bodies react and if the treatment shows any promise in fighting the brain tumour. It's important to remember this is an early-stage study, meaning it's still in the testing phase to gather initial information.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Genenta Science S.p.A.

Enrolment target

Start

28 Feb 2024

Glioblastoma multiforme

What is this study about?

This research is looking into a brand-new type of treatment called 'gene therapy' for a very serious brain cancer called glioblastoma. Glioblastoma is a fast-growing tumour that can be very difficult to treat with current methods. Gene therapy is a cutting-edge approach that aims to correct or change certain genes to help fight diseases.

In this particular study, the gene therapy involves using special 'hematopoietic stem cells.' These are powerful cells usually found in your bone marrow and blood that can develop into different types of blood cells. The idea is to modify these stem cells in a lab and then put them back into the patient. These modified cells are designed to help your body's immune system better recognise and fight the cancer cells.

This study is a very early stage of research, sometimes called 'Phase I and Phase II.' This means it's the first time this specific gene therapy is being given to people. The main purpose is to carefully check if the treatment is safe for patients and to see what effects it has on the body. Doctors will also start to look at whether it shows any signs of working against the glioblastoma. It's an important step in developing new ways to tackle this difficult cancer, but it's important to understand it's not a proven treatment yet.

Key takeaways

Exploring new gene therapy for glioblastoma.
First time this specific treatment is used in humans.
Mainly focused on safety and early signs of effectiveness.
For adults aged 18 and over, both men and women.
Participation involves clinic visits, treatment, and careful monitoring.
It is an experimental treatment, not a guaranteed cure.

Who may be eligible?

To be considered for this study, you would need to be an adult, at least 18 years old. There is no upper age limit, meaning older adults are also welcome to take part.

Both men and women can participate in this research study. Your gender does not affect whether you can be involved. The researchers are looking for participants across the full adult age range and for all sexes to ensure they gather broad information about the treatment.

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 a diagnosis of glioblastoma multiforme?
Do you meet other specific health criteria (which your doctor can explain)?
Are you able to attend appointments and follow the study schedule?
Are you comfortable with receiving an experimental treatment?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, it's important to know what's involved. As this is an early-stage study, there will likely be many visits to the clinic or hospital, especially at the beginning, to closely monitor your health. You would receive the gene therapy treatment, which involves a single administration. Before and after receiving the treatment, you will have various medical checks and tests. These might include blood tests, scans (like MRI scans of your brain), and physical examinations to see how you are responding and to check for any side effects.

There will also be a follow-up period to understand the long-term effects of the treatment. The total length of your participation, including the treatment and follow-up, could extend for several months, possibly over a year or more, but your study doctor will give you a clear timeline. The goal is to gather as much information as possible about this new potential treatment.

Potential risks and benefits

Taking part in any clinical trial involves both potential benefits and potential risks. The main potential benefit of this study is the chance to receive a very new and experimental treatment for glioblastoma that is not yet widely available. While we hope it might help fight the cancer, it's important to remember this is an early trial, so we don't know yet how effective it will be. Potential risks can include side effects from the gene therapy itself, which could be mild, such as fever or tiredness, or potentially more serious, as the treatment is new. Your study team will explain all known and potential risks in detail. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

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

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Unverified
Italy

Common questions

What is gene therapy?

Gene therapy is a new way to treat diseases by changing or adding certain 'genes' to cells. Genes are like instructions for your body, and this therapy tries to give your body better instructions to fight diseases like cancer.

What is glioblastoma?

Glioblastoma is a serious and fast-growing type of brain tumour. It can be very challenging to treat with current standard options.

Is this a proven treatment?

No, not yet. This is an early 'first-in-human' study, meaning it's the first time this specific treatment is being tested in people. The main goals are to check its safety and see if it shows any signs of working.

Will I get better if I join this study?

We cannot guarantee that you will get better. This is an experimental treatment, and while we hope it might help, we are still learning about its effects.

How long does the study last?

Your involvement in the study, including treatment and follow-up, could typically last for several months or even over a year, depending on the study design.