Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD
This study is looking into a new gene therapy, ABBV-RGX-314, for wet age-related macular degeneration (wet AMD). Wet AMD can cause vision loss due to new, leaky blood vessels behind the eye. Currently, people with wet AMD need regular eye injections, often every 4 to 12 weeks, to save their eyesight. This can be a burden and sometimes people miss treatments, which can harm their vision. This new gene therapy is designed to be a one-time treatment that could hopefully prevent vision loss without the need for frequent injections. The study will compare two different doses of ABBV-RGX-314 to the usual treatment, aflibercept (EYLEA®), to see how well it works and if it's safe. Around 714 people will take part.
At a glance
What is this study about?
This study is called the "Pivotal 2 Study" and it's looking at a new treatment for a common eye condition called wet age-related macular degeneration, often shortened to wet AMD. Wet AMD is a major cause of vision loss, mostly affecting older people. It happens when abnormal, leaky blood vessels grow at the back of your eye, damaging your sharp central vision.
At the moment, the main treatment for wet AMD involves regular injections directly into the eye. These injections, like aflibercept (also known as EYLEA®), are good at stopping vision loss, but they need to be given frequently, often every few weeks. This can be difficult and tiring for patients, and if injections are missed, eyesight can get worse.
The new treatment being tested is called ABBV-RGX-314, and it's a type of gene therapy. The idea behind gene therapy is to deliver a special gene to the eye that could help to stop these leaky blood vessels from growing, hopefully with just one treatment. This study is in 'Phase 3', which means it's a large and important step to find out if this new gene therapy is effective and safe before it might become widely available. The main goal is to see if it can improve or maintain vision as well as, or better than, the current regular injections.
Key takeaways
- New gene therapy (ABBV-RGX-314) for wet AMD being tested.
- Aims to replace frequent eye injections with a single treatment.
- Compares two gene therapy doses to standard treatment (EYLEA®).
- Focuses on improving or maintaining vision and safety.
- Phase 3 study, meaning it's a large and important trial.
Who may be eligible?
To join the main study, you generally need to be between 50 and 89 years old and have wet AMD in one eye that has been treated before with anti-VEGF injections. Your vision in that eye needs to be within a certain range, and you must have had cataract surgery on that eye at least 12 weeks ago, meaning you have an artificial lens.
There's also a smaller part of the study for people with wet AMD in both eyes. If you want to take part in this, your vision in both eyes needs to be within a specific range, and you should have had cataract surgery on both eyes at least 12 weeks ago. If you receive the new gene therapy in the main study, you might then be able to receive it in your other eye as well.
You cannot join if your wet AMD is caused by something other than age-related issues, or if you have severe scarring or thinning at the back of your eye. Other reasons you couldn't join include certain heart problems in the last six months, recent eye surgery or other eye treatments, glaucoma that is severe, or if you've already had gene therapy before.
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 between 50 and 89 years old?
- Do you have wet AMD in at least one eye?
- Have you had anti-VEGF injections for your wet AMD before?
- Have you had cataract surgery on your affected eye (at least 12 weeks ago)?
- Have you *not* had any other gene therapy before?
What does participation involve?
If you decide to take part, you'll be assigned to one of three groups. Two groups will receive one of two different doses of the new gene therapy, ABBV-RGX-314. The third group will receive the standard treatment, aflibercept. It's partially 'masked', meaning some people involved won't know which treatment you're getting. The gene therapy is given as a single treatment. You will have regular check-ups, including eye tests, to see how your vision changes and to check for any side effects. These appointments will involve visits to the clinic over a period of time to monitor your progress. The total duration of follow-up can be quite long, but the details of the schedule will be fully explained to you by the study team. There is also an optional smaller study for people with wet AMD in both eyes, which involves similar assessments.
Potential risks and benefits
Locations (181)
- Barnet Dulaney Eye Center-Phoenix /ID# 256340Verified postcodePhoenix, United States· Recruiting
- Retinal Research Institute /ID# 256238Verified postcodePhoenix, United States· Recruiting
- Retina Macula Institute of Arizona /ID# 271026Verified postcodeScottsdale, United States· Recruiting
- California Retina Consultants - Bakersfield /ID# 256240Verified postcodeBakersfield, United States· Recruiting
- Retina Vitreous Assoc Med Grp /ID# 256246Verified postcodeBeverly Hills, United States· Recruiting
- Retinal Diagnostic Center /ID# 256262Verified postcodeCampbell, United States· Recruiting
- The Retina Partners - Encino /ID# 259660Verified postcodeEncino, United States· Recruiting
- Retina Consultants of Orange County /ID# 256267Verified postcodeFullerton, United States· Active not recruiting
- Jacobs Retina Center at UCSD/ID# 256320Verified postcodeLa Jolla, United States· Active not recruiting
- California Retina Consultants - Oxnard - North Ventura Road /ID# 262883Verified postcodeOxnard, United States· Recruiting
- Byers Eye Institute Stanford /ID# 262853Verified postcodePalo Alto, United States· Recruiting
- Retina Consultants of San Diego /ID# 256258Verified postcodePoway, United States· Active not recruiting
Common questions
What is gene therapy?
Gene therapy is a new type of treatment that aims to correct a problem at its source by delivering new genetic material to the body, in this case, to your eye.
What is wet AMD?
Wet AMD (Age-related Macular Degeneration) is an eye condition where new, leaky blood vessels grow at the back of your eye, leading to blurred vision or blind spots in your central eyesight.
How is this new treatment given?
The new gene therapy is designed to be a one-time treatment. The specific way it is given to your eye will be fully explained by the study team.
Will I know if I'm getting the new treatment or the standard one?
Some people involved in the study, including some patients, may not know which treatment you are receiving. This helps ensure the study results are fair and accurate. You will be told if this applies to you.
Why is this study important?
This study is important because it's looking for a new, potentially one-time treatment for wet AMD that could reduce the burden of frequent eye injections and improve the quality of life for patients.
How to find out more
Patient Advocacy
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.