Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
This research is a follow-up study investigating a gene therapy named AAV5-hRKp.RPGR for a specific eye condition called X-linked retinitis pigmentosa (XLRP). XLRP is a genetic disorder that causes progressive vision loss. The study aims to understand how safe and effective this new treatment is for people who have XLRP due to changes in their RPGR gene. Participants, aged 3 and older, will receive one of two doses of the gene therapy. This is a Phase 3 trial, meaning it's one of the final stages of testing before a treatment might be considered for wider use. The therapy is designed to deliver a working version of the RPGR gene to the cells in the eye, which researchers hope could help slow down or even stop the progression of vision loss.
Auf einen Blick
Worum geht es in dieser Studie?
This study is looking into a new treatment for an eye condition called X-linked retinitis pigmentosa, or XLRP. XLRP is a genetic condition that mainly affects boys and men. It causes the light-sensing cells in the back of your eye, called the retina, to slowly break down over time. This leads to a gradual loss of vision, often starting with difficulty seeing in dim light and at the edges of your vision, eventually affecting central vision too.
The treatment being tested is a gene therapy. Our bodies are made of tiny building blocks called genes, which give instructions for how our bodies work. In XLRP, there's a problem with a specific gene called RPGR. This gene therapy aims to deliver a healthy copy of the RPGR gene to the cells in the eye. The hope is that by giving these cells the correct instructions, they can start working properly again, potentially slowing down or even halting the loss of vision. The specific gene therapy being studied is called AAV5-hRKp.RPGR, and it will be tested at two different dose levels.
This is a Phase 3 study, which means it's a large and important step in testing this new treatment. Researchers have already done earlier studies to check if the treatment is safe, and now they want to find out how well it works and compare different doses. The information gathered from this trial will help doctors understand if this gene therapy could become a new way to treat XLRP in the future.
Wichtige Erkenntnisse
- This study evaluates a gene therapy for X-linked retinitis pigmentosa (XLRP).
- It's for individuals aged 3 and over with an RPGR gene change.
- The treatment involves delivering a healthy RPGR gene to the eye.
- This is a Phase 3 trial, testing different doses of the therapy.
- It aims to understand if the treatment can help slow or stop vision loss.
Wer könnte berechtigt sein?
To be considered for this study, you need to meet certain requirements. The most important is that you must have X-linked retinitis pigmentosa (XLRP), and this diagnosis needs to be confirmed by an eye specialist who knows about these conditions. You also need to have a specific change, or 'variant', in your RPGR gene, which is known to cause the condition. This gene change will need to have been identified by a specialist lab.
The study is open to both males and females, and you must be at least 3 years old or older. There isn't an upper age limit mentioned, so people of all adult ages could potentially be included if they meet the other criteria.
At present, there are no exclusion criteria listed, meaning there aren't any specific reasons given that would stop someone from taking part if they meet the general requirements.
Könnte diese Studie für Sie geeignet sein?
Beantworten Sie diese kurzen Fragen, um zu sehen, ob Sie möglicherweise berechtigt sind. Dies ist nur ein Leitfaden – das Forschungsteam trifft die endgültige Entscheidung.
- Do you have X-linked retinitis pigmentosa (XLRP)?
- Has an eye specialist confirmed your XLRP diagnosis?
- Do you have a specific change in your RPGR gene that causes XLRP?
- Are you 3 years old or older?
Was beinhaltet die Teilnahme?
If you decide to take part in this study, you will receive the investigational gene therapy, AAV5-hRKp.RPGR. This will be given either at an intermediate dose or a low dose. The therapy is administered directly into your eye. You will have regular visits to the clinic for check-ups and various eye tests to see how you are responding to the treatment and to monitor your vision. These assessments will help the doctors understand if the therapy is working and if there are any side effects. You'll likely need to attend several appointments over a period of time to track your progress. The total duration of your participation, including all follow-up appointments, will be clearly explained to you by the study team.
Potenzielle Risiken und Vorteile
Locations (28)
- Shiley Eye Institute Jacobs Retina CenterVerifizierte PostleitzahlLa Jolla, Vereinigte Staaten
- Childrens Hospital Los AngelesVerifizierte PostleitzahlLos Angeles, Vereinigte Staaten
- Stanford Health CareVerifizierte PostleitzahlPalo Alto, Vereinigte Staaten
- VitreoRetinal Associates, PAVerifizierte PostleitzahlGainesville, Vereinigte Staaten
- Emory UniversityVerifizierte PostleitzahlAtlanta, Vereinigte Staaten
- Massachusetts Eye and Ear InfirmaryVerifizierte PostleitzahlBoston, Vereinigte Staaten
- Univ of Michigan Medical CenterVerifizierte PostleitzahlAnn Arbor, Vereinigte Staaten
- Duke Eye CenterVerifizierte PostleitzahlDurham, Vereinigte Staaten
- University of Pittsburgh Medical Center (UPMC)Verifizierte PostleitzahlPittsburgh, United States
- Retina Consultants of HoustonVerifizierte PostleitzahlBellaire, United States
- Ghent University HospitalVerifizierte PostleitzahlGent, Belgien
- Hospital For Sick ChildrenVerifizierte PostleitzahlToronto, Kanada
Häufige Fragen
What is X-linked retinitis pigmentosa (XLRP)?
XLRP is a genetic eye condition that causes cells in the back of your eye (retina) to break down, leading to gradual vision loss, often affecting men more severely.
Was ist Gentherapie?
Gene therapy is a new type of treatment that aims to correct faulty genes, in this case, by delivering a healthy copy of the RPGR gene to the eye.
Will I definitely get the treatment if I join?
Yes, if you're accepted into this study, you will receive one of the two doses of the gene therapy being tested.
Ist dies eine neue Behandlung?
Yes, AAV5-hRKp.RPGR is an experimental treatment still being studied. This is a Phase 3 trial, meaning it's in the later stages of research.
Wie lange wird die Studie dauern?
The exact duration for each participant, including follow-up, will be discussed with you by the study team if you are eligible.
Wie Sie mehr erfahren können
Sprechen Sie immer mit Ihrem Hausarzt oder Spezialisten, bevor Sie sich entscheiden, an einer Studie teilzunehmen.
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