RecruitingOBSERVATIONAL

Study of Viral Transduction of Human Auditory Sensory Cells for the Development of Gene Therapy

This research is exploring a new way to treat hearing loss and balance issues using something called gene therapy. The idea is to use tiny, harmless viruses (called AAVs) to deliver new genetic material into the cells of the inner ear. Scientists already know these viruses work well in animal studies. This particular study will look at human inner ear cells that are removed during surgery for a specific type of brain tumour called a vestibular schwannoma. By studying these cells in the lab, researchers hope to understand if this gene therapy approach could be a safe and effective treatment for people with hearing problems in the future. It's a key first step in developing new treatments.

At a glance

Status

Recruiting

Sponsor

Institut Pasteur

Enrolment target

100

Start

19 Feb 2019

Estimated completion

18 Feb 2027

Hearing Loss, Sensorineural

What is this study about?

Hearing loss and problems with balance can significantly affect people's lives. For many, these issues are caused by damage to the sensitive cells within the inner ear. Currently, treatment options can be limited, so scientists are always looking for new and effective ways to help.

One exciting area of research is gene therapy. This involves introducing new genetic material into cells to help them work better or to correct problems. In this study, researchers are focusing on a particular type of delivery system for gene therapy: using a modified, harmless virus. These viruses, called "Associated adenoviruses" or AAVs, are like tiny natural delivery trucks that can carry genetic instructions into cells without making you sick. Animal studies have shown that AAVs are a promising way to get genetic material into the inner ear cells.

This specific study aims to take a closer look at how these AAV viruses can deliver genetic material to *human* inner ear cells. To do this, they will use small samples of inner ear tissue collected during a type of surgery. This surgery is performed when someone needs to have a non-cancerous growth, called a vestibular schwannoma, removed from near their inner ear. By studying these human cells in a lab, researchers can learn more about how to develop safe and effective gene therapies for hearing loss and balance disorders in the future. It's an important step in translating promising lab results into potential treatments for people.

Key takeaways

This study helps develop new gene therapies for hearing and balance.
It uses inner ear cells collected during pre-existing surgery.
You won't receive new treatments or medication as part of the study.
Participation involves no extra risks beyond your planned surgery.
It's a foundational step for future hearing loss treatments.

Who may be eligible?

To be considered for this study, you must be at least 18 years old. You also need to be having an operation to remove a non-cancerous growth called a vestibular schwannoma, and your surgeon must have decided that the best approach for your specific situation is a 'non-conservative' removal. This means the surgeon is planning to remove the growth in a way that might involve taking some inner ear tissue. You must also give your full permission to take part by signing an informed consent form, and you need to have eligibility for French social health protection.

There are also some reasons why you wouldn't be able to join. For example, if your tumour has grown into the very inside of your ear (either into the balance part or the hearing part), you wouldn't be eligible. Pregnant women also cannot take part. Additionally, if you have certain health conditions that would make taking part unsafe, or if there are specific administrative checks that prevent participation, you wouldn't be able to join.

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?
Are you having surgery for a vestibular schwannoma, with a 'non-conservative' removal planned?
Are you able to give your full permission to take part?
Are you not pregnant?
Does your tumour NOT extend into the deep inner ear (balance or hearing part)?

Answer every question to see your result.

What does participation involve?

This study does not involve you directly receiving any treatment or medication. If you are eligible and choose to take part, the researchers would simply collect a small sample of your inner ear cells during your planned surgery to remove a vestibular schwannoma. This collection would happen during the operation you are already having. There are no extra doctor's visits, assessments, or follow-up appointments required because of this study. Your participation simply involves allowing the research team to use a small part of the tissue that is already being removed during your surgery for this important research. The total duration of your 'participation' would effectively be limited to the time of your surgery when the sample is collected.

Potential risks and benefits

The main benefit of taking part in this study is contributing to important medical research that could one day lead to new treatments for hearing loss and balance problems. Your participation would help scientists understand how gene therapy might work in human inner ear cells. For you personally, there are no direct benefits or changes to your health from participating. Since the cells are collected during a surgery you are already undergoing, and no extra procedures are performed specifically for the study, there are no additional risks beyond those of your planned surgery. You have the right to withdraw your consent to participate at any time, without it affecting your medical care.

Locations (2)

Hôpital de Bicêtre
Verified postcode
Le Kremlin-Bicêtre, France· Recruiting
Hôpital Pitié-Salpétrière
Verified postcode
Paris, France· Recruiting

Common questions

What is gene therapy?

Gene therapy is a way to treat diseases by introducing new genetic material into cells to help them work better or correct problems.

What are AAVs?

AAVs are tiny, modified viruses that are used as a delivery system to carry new genetic instructions into cells without making you sick.

Will this study treat my hearing loss?

No, this is a lab-based study using cells collected during surgery. It will not directly treat your hearing loss or balance problems.

Does my participation involve extra surgery?

No, the cells are collected during the surgery you are already having for your vestibular schwannoma. No additional procedures are performed for the study.

Who is funding this research?

Information on specific funding is not provided in the brief summary. You can ask the research team for more details if interested.

How to find out more

Ghizlene Lahlou

ghizlene.lahlou@pasteur.fr 33 1 45688891 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.