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Age-related macular degeneration (AMD) is a chronic, progressive eye disease that develops with age. AMD impacts the macula, or central part of the retina. This disease mainly contributes to the loss of central vision which makes everyday tasks like driving, reading, and recognizing faces challenging. The advanced stage of AMD is called Geographic Atrophy (GA). GA affects over 5 million people globally, including approximately 1.5 million people in the United States alone. Participation in clinical research studies helps contribute to ongoing research which can potentially help people with GA in the future.
Geographic atrophy (GA) is an advanced form of age-related macular degeneration (AMD), an eye condition that affects central vision. GA develops when the light sensing tissue at the back of your eye (the macula or central part of the retina) becomes damaged. GA often begins in small areas and gradually spreads over time. As the damaged areas of the retina grow larger, central vision loss can increase, making everyday activities more challenging.
The complement system is part of your body’s natural immune defenses. It helps protect you by responding to injury, infection, and damaged cells. In a healthy eye, this system is carefully balanced and helps maintain normal tissue health.
In GA, this balance can be disrupted. Research suggests that overactivation of the complement system may lead to ongoing inflammation in the retina. Over time, this chronic inflammation can damage the macula, the part of the retina responsible for sharp, central vision, and contribute to the gradual loss of retinal cells seen in GA.
Understanding how the complement system contributes to GA has been a major focus of research. This growing knowledge is helping scientists develop new ways to measure disease progression and explore potential future therapies aimed at slowing damage to the retina.
Gene therapy is a medical technique that treats or prevents disease by modifying, adding, or removing genetic material (DNA or RNA) within a patient's cells.
CTx001 is an investigational gene therapy designed to deliver a therapeutic gene to cells at the back of the eye (macula). The therapeutic gene produces a protein that has the potential to rebalance the complement system and reduce inflammation. The reduction of inflammation may stop further damage in the macula and prevent further vision loss in patients with geographic atrophy.
i-GAIN: A long-term observational study designed to track the natural progression of GA in participants over time, focusing on genetics, imaging, and biomarkers. i-GAIN is now closed for new participants.
Pre-GAIN: Pre-GAIN is a short-term observational study designed to closely monitor your eye condition over a brief period of time. This study does not include treatment. Instead, it uses novel endpoints to better understand how geographic atrophy progresses over a short timeframe. Data from Pre-GAIN helps researchers evaluate disease stage and patient eligibility, which may inform consideration for future Complement Therapeutics sponsored clinical studies, including Opti-GAIN.
Opti-GAIN: A clinical study testing a potential new one-time gene therapy called CTx001 for patients with GA secondary to AMD.
The i-GAIN study (NCT05797896) was a natural history study investigating the progression of geographic atrophy (GA) and its relationship with key biomarkers. Now closed, the study enrolled over 230 participants and followed them for up to 24 months.
Insights from i-GAIN have been instrumental in shaping the design and eligibility criteria for the Opti-GAIN interventional trial. Participants who met the criteria were invited to continue in follow-on studies, including Pre-GAIN and Opti-GAIN.
Pre-GAIN (NCT07144137) is a short-term observational study designed to provide insights into the short-term progression of GA secondary to AMD in participants aged ≥55 years. An observational study means no treatment is available. The study aims to investigate novel endpoints by closely tracking structural and functional changes in GA patients every 3 months, up to Month 6, in participants aged 55 years and older.
Although no treatment is provided in the Pre-GAIN study, participation helps researchers better understand disease stage and progression and may be important for consideration in future Complement Therapeutics sponsored studies.
Study participants may be able to join the study if they meet the following requirements*:
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55 years of age or older
Confirmed GA secondary to AMD in both eyes, with at least 1 eye meeting further criteria to participate in the study
Meet other vision criteria
*Other study requirements will apply.
Participation in a clinical study is voluntary. You can ask any questions you have and may leave the study at any time, for any reason.
The Opti-GAIN study (NCT07392255) is researching a potential new one-time investigational gene therapy called CTx001 for people with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). There is currently no approved treatment for GA in the UK or Europe, with limited treatment options elsewhere. CTx001 is only available to participants of the Opti-GAIN study. Whether you benefit from participation in clinical studies like Opti-GAIN or not, participation in clinical studies such as this can potentially help others with GA in the future.
Study participants may be able to join the study if they meet the following requirements*:
65 years of age or older
Have confirmed GA secondary to AMD in both eyes, with at least 1 eye meeting further criteria
Meet other vision criteria
*Other study requirements will apply.
The study therapy, CTx001, is an innovative gene therapy designed to modulate the complement system to correct imbalances that are thought to be key drivers of disease in Geographic Atrophy (GA) secondary to Age-Related Macular Degeneration (AMD). The Opti-GAIN study is evaluating the safety and potential beneficial effect of CTx001 in relation to reducing the detrimental impact of GA.
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In particular, CTx001 has the potential to:
Reach the Right Areas of the Eye
• In geographic atrophy, the back of the eye is damaged. There’s a thin barrier there (called Bruch’s membrane) that blocks many treatments from reaching where they need to go.
• CTx001 is designed to cross that barrier and work on both sides — right where the disease is happening.
Target the Root Cause
• Your body has a natural immune system called the “complement system”. In GA, this system becomes overactive and causes damage.
• CTx001 calms down two major parts of this system to reduce inflammation and slow the disease.
Reduce Inflammation in the Retina
• Inflammation is a key driver of damage in GA. CTx001 switches off the proteins (C3 and C4) that trigger this inflammation, which may help protect the cells in the back of the eye that are responsible for vision.
Provide One-Time Treatment
• CTx001 is being developed as a one-time gene therapy that is given under the retina.
• This means patients could avoid the need for monthly injections into the eye, which are required by currently approved products in the US and can be uncomfortable and burdensome.
Here are some common questions and answers about study participation.
Clinical research studies, or trials, help scientists and doctors explore whether a medical strategy, device, or medication is safe and effective for people. Before any medication can be approved and made available to the public, it must go through several phases of clinical research.
Before enrolling in a clinical trial, you must sign an Informed Consent Form (ICF). The ICF contains information about the study, including study goals, how long the study will last, benefits and risks, and the tests and procedures you will receive.
Study participation usually involves visiting a clinic regularly, taking or receiving an investigational medicine, and having assessments to monitor your health. You can still see your regular doctor, but you should let them know that you are participating in a study.
Participation in clinical research studies is your choice, and you may stop at any time.
If you have additional questions about participating in a clinical research study, contact a study clinic near you.
Use the filters, map, and listing to find the study clinic closest to you. Contact the study clinic to learn more.
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All participants who qualify for the Opti-GAIN study will receive a single injection of the study therapy (CTx001). Pre-GAIN is a short-term observational study and therefore participants will not receive any placebo or treatment.
View the study clinic locations map to contact a site near you. The study team can provide more information about potential study participation.
Participation in the Pre-GAIN study lasts at least 6 months with the potential for extended participation to be approved every 6 months. Participation will depend on the progression of your GA.
Participation in the Opti-GAIN study lasts up to 5 years and 5 months, with long-term follow-up visits occurring from year 2 to year 5. All participants will receive a single subretinal injection of the study therapy and will be followed a total of approximately 5 years post dosing.
For more information about the Complement studies, contact our team at clinicaltrials@complementtx.com.