Study on Long-Term Safety and Effects of SAR421869 for Patients with Usher Syndrome Type 1B

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What is this study about?

This clinical trial is focused on studying , a genetic condition that affects hearing, balance, and vision. The treatment being investigated is called , which is a special type of injection known as a . This vector is derived from a virus called the and contains a human gene known as . The purpose of the study is to evaluate the long-term safety and tolerability of this treatment in patients with Usher syndrome Type 1B.

Participants in the study will receive an injection of and will be monitored over a period of time to assess how well they tolerate the treatment and to observe any potential side effects. The study will also look at how the treatment affects the progression of the disease, particularly in terms of eye health and vision. The study is open-label, meaning that both the researchers and participants know what treatment is being administered.

The main focus of the study is to ensure that the treatment is safe for long-term use and to understand its effects on delaying the degeneration of the retina, which is a part of the eye that is crucial for vision. Participants will undergo regular assessments to monitor their eye health and any changes in their condition. The study aims to provide valuable information on the potential benefits and risks of using for treating Usher syndrome Type 1B.

1 joining the study

Participation begins after providing signed and dated written informed consent. This includes any necessary local authorizations, such as the Health Insurance Portability and Accountability Act (HIPAA).

Eligibility requires previous enrollment in protocol TDU13600 and having received a subretinal injection of SAR421869.

2 treatment administration

SAR421869 is administered as a suspension for injection. The specific route of administration is not detailed in the available information.

3 monitoring and assessments

The study aims to evaluate the long-term safety and tolerability of SAR421869 in patients with Usher syndrome Type 1B.

Primary assessments focus on the incidence of adverse events.

Secondary assessments include monitoring for clinically important changes in eye safety and any delay in retinal degeneration.

4 study duration

The study is expected to continue until June 18, 2031.

Who Can Join the Study?

Provide signed and dated written informed consent. This means you agree to participate in the study and understand what it involves. If needed, provide assent, which is a way for younger participants to agree.
Have any required local authorization, such as the Health Insurance Portability and Accountability Act (HIPAA), which is a law that protects your health information.
Must have been enrolled in a previous study with the code TDU13600.
Must have received a subretinal injection of SAR421869. A subretinal injection is a procedure where a small amount of medicine is placed under the retina, which is a part of the eye.
Both males and females can participate.
Participants can be from vulnerable populations, which means groups that might need special protection or care.

Who Cannot Join the Study?

Patients who do not have Usher’s syndrome Type 1B cannot participate. Usher’s syndrome is a condition that affects hearing and vision.
Patients who are not within the specified age range for the study cannot participate. The age range is not specified here, but it is important for eligibility.
Patients who are not part of the specified clinical trial group cannot participate. This refers to a specific group of patients that the study is focusing on.
Patients who are not male or female cannot participate. The study includes both male and female participants.
Patients who are not considered part of a vulnerable population cannot participate. A vulnerable population includes groups that may need special protection or care.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Quinze-Vingts National Ophthalmology Hospital	Paris	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	30.05.2013

Trial locations

SAR421869 is a medication being studied for its long-term safety and tolerability in patients with Usher syndrome Type 1B. This trial aims to understand how well patients can handle the medication over an extended period and to observe any biological effects it may have. Usher syndrome Type 1B is a genetic condition that affects hearing and vision, and this study is focused on finding a treatment that could potentially help manage these symptoms.

Investigated diseases:

Usher's syndrome

Usher Syndrome Type 1B – Usher Syndrome Type 1B is a genetic disorder characterized by hearing loss and progressive vision loss. Individuals with this condition are typically born with severe to profound hearing impairment and experience balance issues due to inner ear problems. Vision loss usually begins in childhood or adolescence and is caused by a condition called retinitis pigmentosa, which leads to the degeneration of the retina. As the disease progresses, individuals may experience night blindness and a narrowing of the field of vision. Over time, the vision impairment can become more severe, affecting daily activities. The progression of symptoms can vary among individuals, but the combination of hearing and vision loss significantly impacts communication and mobility.

Trial ID:

2024-513500-34-00

Protocol code:

LTS13619

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on Long-Term Safety and Effects of SAR421869 for Patients with Usher Syndrome Type 1B

What is this study about?

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