Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
This research study is looking into a new treatment called AAVB-081 for a rare inherited condition called Usher Syndrome type 1B (USH1B). This condition can cause both hearing and vision loss, specifically a type of progressive sight loss known as retinitis pigmentosa. The study focuses on people aged 18 to 50 whose Usher Syndrome type 1B is caused by a change in a gene known as MYO7A. The main goals are to find out if a single injection of AAVB-081, given into the back of the eye (subretinal), is safe and well-tolerated by participants. Researchers will also be looking closely to see if the treatment shows any early signs of helping with vision. This is a crucial early-stage study (Phase 1/2) to understand the potential of this new genetic therapy.
Em resumo
Sobre o que é este estudo?
This clinical trial is designed to investigate a new treatment, known as AAVB-081, for individuals living with Usher Syndrome type 1B (USH1B). Usher Syndrome is a genetic condition that typically affects both hearing and vision. Specifically, this study focuses on the vision problems, which are often a progressive loss of sight called retinitis pigmentosa. This type of Usher Syndrome is linked to a specific change in the MYO7A gene.
The main purpose of this study is to carefully assess the safety and how well people tolerate a single dose of AAVB-081. This treatment is delivered directly into the back of the eye using a special injection technique. Additionally, the researchers will be watching for any early signs to see if the treatment is effective in improving or stabilising vision.
This is an early-stage study, meaning it's one of the first times this treatment is being tested in people. The information gathered from this trial will be vital for understanding if AAVB-081 could be a promising future treatment option for people with Usher Syndrome type 1B and retinitis pigmentosa.
Pontos principais
- This is an early-stage study for Usher Syndrome type 1B (USH1B) related vision loss.
- The treatment, AAVB-081, is a gene therapy delivered by eye injection.
- The study aims to check safety, tolerability, and early signs of vision improvement.
- Participants must have USH1B due to a MYO7A gene mutation and be aged 18-50.
- Close monitoring will be in place after the treatment.
Quem pode ser elegível?
To be considered for this study, you must be between 18 and 50 years old. You need to have a confirmed diagnosis of Usher Syndrome type 1B that is specifically caused by a mutation in the MYO7A gene. It's also important that you are willing and able to follow all the study's instructions and attend all required appointments.
There are certain reasons why you might not be able to join. For example, if you've taken part in another clinical trial involving an investigational medicine in the last six months, or if you've ever had a gene therapy trial before, you wouldn't be eligible. Also, if you have any eye conditions or other health issues that would make eye surgery difficult or risky, or if you simply don't wish to meet the study's requirements, you wouldn't be able to participate.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- Are you between 18 and 50 years old?
- Do you have a confirmed diagnosis of Usher Syndrome type 1B due to a MYO7A gene change?
- Are you willing and able to follow all study instructions and attend appointments?
- Have you *not* participated in another gene therapy trial before?
- Do you have no other eye or health conditions that would make eye surgery difficult or risky?
O que envolve a participação?
If you decide to take part in this study, you would receive the AAVB-081 treatment through a single injection into the back of one of your eyes. This is a specialised surgical procedure. Following the injection, you would have a series of regular visits to the clinic so the research team can closely monitor your health, check how your eye is healing, and assess your vision.
These visits will involve various eye tests and general health checks. The exact number and frequency of visits, as well as the total duration of your participation, would be explained in detail by the study team. This ongoing monitoring is crucial to understand the safety and potential effects of the treatment over time.
Riscos e benefícios potenciais
Localizações (3)
- University of Campania Luigi VanvitelliCódigo postal verificadoNápoles, Itália· Recrutamento
- Hospital Oftalmológico MoorfieldsCódigo postal verificadoLondres, Reino Unido· Recrutamento
- Retina Clinic LondonCódigo postal verificadoLondres, Reino Unido· Recrutamento
Perguntas frequentes
What is Usher Syndrome type 1B?
It's a rare genetic condition that affects both hearing and vision. The vision loss is a progressive condition called retinitis pigmentosa, specific to a MYO7A gene change in this type.
What is AAVB-081?
AAVB-081 is an experimental gene therapy designed to address the underlying genetic cause of vision loss in Usher Syndrome type 1B.
How is the treatment given?
The treatment is given as a single injection directly into the back of one of your eyes, which is a specialised surgical procedure.
What are the main goals of the study?
The study aims to understand if AAVB-081 is safe and well-tolerated, and to look for early signs that it might help improve or stabilise vision.
Posso sair do estudo se mudar de ideia?
Yes, you are free to withdraw from the study at any time, for any reason, and your decision will not affect your medical care.
Como saber mais
Clinical Operations Manager
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
Interessado em participar?
Discussão
Discussão da comunidade
Desenvolvido pelo nosso fórum em community.patient.info. Por favor, seja respeitoso — isto não é um conselho médico.