Active not recruitingPHASE2INTERVENTIONAL

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B

This study is looking into the long-term safety and tolerability of a treatment called SAR421869 for people living with Usher syndrome Type 1B. Usher syndrome affects hearing, balance, and vision. This particular study is for individuals who have already received SAR421869 as part of a previous research project. Researchers want to see if the treatment continues to be safe over a long period and if it has any lasting effects on the body. This is a follow-up study that could last for up to 15 years, involving assessments like blood tests to monitor participants' health and the treatment's impact.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Sanofi

Enrolment target

Start

12 Sep 2013

Estimated completion

13 Jun 2031

Usher's Syndrome

What is this study about?

This study is about a treatment called SAR421869 for a condition called Usher syndrome Type 1B. Usher syndrome is a rare genetic condition that usually affects a person's hearing, balance, and vision. There are different types, and Type 1B is one of them.

The main goal of this research is to see how safe this treatment is over a long time for people who have Usher syndrome Type 1B. Researchers also want to understand if the treatment has any lasting effects on the body. This study is aimed at people who have already taken part in a previous study where they received SAR421869. Think of it as a follow-up to check how they're doing years later.

By carefully watching how people respond to the treatment over a long period, doctors hope to learn more about its safety and if it has any positive impact on the condition. This information is really important for developing new and effective treatments for Usher syndrome Type 1B in the future.

Key takeaways

This study is a long-term follow-up, up to 15 years.
It's for people with Usher syndrome Type 1B who previously received SAR421869.
The main goal is to check the long-term safety and whether the treatment is still having an effect.
Participation involves health check-ups and giving blood samples.
This study will help doctors learn more about Usher syndrome Type 1B treatments.

Who may be eligible?

This study is specifically for adults, aged 18 years and older, who have Usher syndrome Type 1B. If you are interested, you must have already participated in another study called TDU13600. During that previous study, you would have received the SAR421869 treatment as an injection into your eye.

To join this study, you also need to be able to understand and sign a consent form. This form explains all the details of the study, and by signing it, you agree to take part. It's a way to make sure you have all the information and are happy to be involved.

If you did not receive the SAR421869 treatment in the previous TDU13600 study, then you would not be able to join this particular long-term follow-up 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 Usher syndrome Type 1B?
Did you take part in a previous study called TDU13600?
Did you receive the SAR421869 treatment in that previous study?
Are you able to understand and sign a consent form?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be monitored for a long time, potentially up to 15 years. You'll have regular visits to the clinic where doctors and nurses will check your health. One of the main things you can expect is to have blood samples taken. These blood tests help the researchers understand how the SAR421869 treatment is affecting your body over time and to make sure you remain safe. While the specific number of visits isn't detailed, they will be spread out over many years to track the long-term effects. You won't receive the SAR421869 treatment again in this study; it's purely to observe those who received it previously.

Potential risks and benefits

Taking part in this study may not directly benefit you, but the information collected could help doctors learn more about Usher syndrome Type 1B and potentially lead to better treatments for others in the future. As with any study, there could be some small risks, such as discomfort from blood draws. The researchers will monitor your health closely to ensure your safety. Remember, participation is completely voluntary, and you have the right to withdraw from the study at any time without giving a reason, and it will not affect your future medical care.

Locations (2)

Oregon Health and Science University Site Number : 840001
Verified postcode
Portland, United States
Investigational Site Number : 250001
Verified postcode
Paris, France

Common questions

What is Usher syndrome Type 1B?

Usher syndrome Type 1B is a genetic condition that affects hearing, balance, and vision. It's rare and can vary in how it affects people.

What is SAR421869?

SAR421869 is a treatment that was given to participants in a previous study for Usher syndrome Type 1B. This study is now checking its long-term effects.

How long will I be in this study?

This study could last for a very long time, up to 15 years. It's designed for long-term monitoring.

What will I have to do if I join?

You'll have regular check-ups and provide blood samples. These are to help researchers understand the long-term safety and effects of the treatment you received previously.

Will I get money for participating?

The information provided here doesn't mention payment for participation, but you should always ask the study team about any expenses or payments.