Usher’s syndrome – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 10:36:08 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Usher’s syndrome – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on Long-Term Safety and Effects of SAR421869 for Patients with Usher Syndrome Type 1B https://clinicaltrials.eu/trial/study-on-long-term-safety-and-effects-of-sar421869-for-patients-with-usher-syndrome-type-1b/ Wed, 29 Apr 2026 14:59:34 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-and-effects-of-sar421869-for-patients-with-usher-syndrome-type-1b/ 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.

]]> Study of AAVB-081 and Prednisolone for Patients with Usher Syndrome Type 1B Retinitis Pigmentosa https://clinicaltrials.eu/trial/study-of-aavb-081-and-prednisolone-for-patients-with-usher-syndrome-type-1b-retinitis-pigmentosa/ Wed, 29 Apr 2026 14:57:37 +0000 https://clinicaltrials.eu/trial/study-of-aavb-081-and-prednisolone-for-patients-with-usher-syndrome-type-1b-retinitis-pigmentosa/ This clinical trial is focused on studying a condition called Usher Syndrome Type 1B (USH1B) Retinitis Pigmentosa. This is a genetic disorder that affects both hearing and vision, leading to progressive vision loss. The study is testing a new treatment called AAVB-081, which is a type of gene therapy. Gene therapy involves using a specially designed virus to deliver a healthy copy of a gene to cells in the body. In this case, the treatment uses an adeno-associated viral vector to deliver the MYO7A gene, which is important for normal vision, directly to the eye.

The purpose of the study is to evaluate the safety and tolerability of this new treatment when it is injected under the retina, which is the light-sensitive layer at the back of the eye. Participants will receive the treatment through a procedure called subretinal administration. The study will also aim to find the best dose that balances benefits and risks. Throughout the study, participants will undergo various health checks, including eye exams and blood tests, to monitor their response to the treatment.

Participants in the study will be monitored over a period of time to assess any side effects and to determine how well the treatment works in improving or stabilizing vision. The study will help researchers understand if this gene therapy can be a safe and effective option for people with Usher Syndrome Type 1B Retinitis Pigmentosa.

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