More About the Study
Working to Unlock the Mystery of AMD
Phase 1 clinical trial for age-related macular degeneration (AMD) has proven to be successful and safe. The results showed a decrease in the progression rate of the disease.
AMD starts as small yellow accumulations of extracellular proteins and lipids to form a toxic deposit called drusen. The word drusen, from the German word for node or geode, forms in the macula, which is part of the retina at the back of the eye and responsible for our central vision. In some of the subjects, the disease advances to seriously damage the macula which we use for our central, or reading vision.
Figure A is the retinal image of a 70-year-old woman with AMD prior to treatment. She had limited vision, the big E, or 20/200. Note the presence of the drusen, marked with arrows. Her life was severely restricted, unable to drive a car, contemplating assisted living and without hope. She had already lost vision in left eye which was limited to finger counting vision. She volunteered to enter the phase 1 study.
Figure B is after one month of treatment. Note the disappearance of the drusen. Her vision had improved to 20/60 and she could read again. Subsequently, she developed a hemorrhage in the macula (the wet form of AMD, but she was successfully treated by ocular injection of anti-vascular endothelial factor by a retinal specialist at the Northwestern University Department of Ophthalmology, Feinberg School of Medicine).
Figure C is after one year, her vision has improved to 20/40 and is stable (“M” macula in the images). Note: the wet form of macular degeneration is an exclusionary diagnosis for phase 2 of the study.
More About the Study
Under this open label, (both researchers and participants know which study drug is being given to participants) double arm (study participants are randomized to either the control or active intervention arms) phase 2 study, researchers will find out more about investigational products (a product that is being tested and is not approved for sale as a drug in the U.S. by the U.S. Food and Drug Administration (FDA).
The main purpose of this study is to see whether the study medications can help to reduce drusen volume and slow or prevent progression to advanced age-related macular degeneration. Researchers also want to find out if these products are safe and tolerable.
Safety will be evaluated using a series of tests. Adverse events will be recorded if applicable at every study visit for all participants.
Efficacy will be evaluated using a series of OCT-based retinal photography and image processing techniques to evaluate drusen size, pigmentary changes, and geographic atrophy (GA), which is an advanced form of dry AMD. Best-corrected visual acuity will also serve as a primary outcome measure of efficacy with consideration for the clinical importance of functional vision loss.
- First demonstration of a safe oral study treatment (RQC is the active formulation) and effective way to slow the progression of drusen size in subjects with pre-AMD (early, intermediate, and large drusen).
- First evidence of a safe and effective oral study treatment (RQC is the active formulation) to slow the progression of geographic atrophy.
The Phase 2 study for age-related macular degeneration (AMD) is ongoing and a larger study. It may also provide research into a potentially new diagnostic test for Alzheimer’s disease. Our research efforts will hopefully discover treatments that will lead to breakthroughs in the treatments of these diseases.
What to Expect?
As a part of the Insight to Macular Degeneration Study (IMDS), you will receive a series of exams and study-related treatments. This is a randomized study where you will be given one of two study medication regimens. Both regimens are in pill form.
The study lasts for two years. In that time, you will be required to come into our Chicago office for seven study visits where you will be examined to determine how well you are progressing and a series of questions regarding your state of health. Each study visit includes (at no cost to you): an optical coherence tomography (OCT) retinal scan, visual acuity testing and an interview with the study staff.
You can also expect a twice-daily regimen of the study medication(s).
We will call you for an initial screening. During the study you can reach out any time with questions or concerns.
You will have an initial (in-person) visit which will last approximately 1 hour where we will:
- Obtain a complete medical history which involves several questions
- Dilate your pupils
- Conduct retinal imaging
You will have subsequent visits which will last approximately 30 minutes where we will:
- Test visual acuity
- Conduct retinal imaging, dilation may be required
- Ask questions about how you’re feeling (any changes or improvements)
- All study-related treatments will be provided
Frequently Asked Questions
Who is paying for this study?
A company called Zaparackas and Knepper, Ltd., and RQC Prevention, LLC are funding the study.
Will it cost anything to be in this study?
You and/or your health-care payer/insurer will not be billed for the costs of the study. Validated parking may be available and a 24-month supply of study medications will be provided at no cost.
You can ask the study doctor or study staff to find out more about costs.
How long will I be in the study?
If you decide to be in this study and the study doctor says you can be in the study, your participation will last about 24 months (two years).
You will visit the study center to have the procedures and tests. Ask the study doctor or study staff about your study visit schedule.
Will I receive any new information during the study?
If the study doctor or study staff learns any new information that might change your mind about continuing in the study, the study doctor or study staff will tell you about it.
What if I get hurt or sick while I am in this study?
If you experience a change in your health status while participating in the study, please contact the study team to inform them as soon as possible.
Will I receive payment?
You will not receive any payment for your participation in this study.
Do I have to be in this study?
Your participation in this study is voluntary. You can decide not to be in the study, and you can change your mind about being in the study at any time. If you want to stop being in the study, tell the study doctor or study staff.
The study doctor or study staff or sponsor can remove you from the study at any time, even if you want to stay in the study. This could happen if, for example:
- The study doctor or study staff believes it is best for you to stop being in the study.
- You do not follow directions about the study.
- The sponsor stops the study for any reason.
If you stop being in the study early, the study doctor or study staff may ask you some questions about being in the study. The study doctor or study staff may ask you to participate in some procedures or tests to help you leave the study safely and/or to collect more information for the study.
If you leave the study, the study doctor and study staff will still be able to use your information that they have already collected.
How will my information be kept confidential?
Your identity will be protected as required by law and according to any policies the study center or sponsor may have. Be aware that your study records (which includes your information from your medical records, the videos and photos, your signed and dated consent form, and other information) will be shared as needed for the study. For example, the FDA, the sponsor, and Advarra Review may look at your study and medical records.
All video files and OCT image files will be de-identified (coded). The list that matches the code with your name will be stored separately from the files.