|Wet macular degeneration clinical trials look at new therapies and new combinations of existing therapies. From oral medications, to eye drops to less frequent eye injections, new therapy options are being researched to improve visual acuity, safety, efficacy and compliance.|
Clinical trials can be interventional, meaning they are looking at new treatment options or observational, which means the study is observing different functions of the eye and how a disease progresses.
You will find the most complete international listing at the National Institute of Health website at
Here are some listings of wet macular degeneration clinical trials.
The ALTISSIMO study has just finished recruiting their last patient for a total of 56 enrolled participants. Sponsored by Graybug Vision, Inc. out of Redwood City, California, this Phase 2b study was conducted at 33 sites across the U.S. The purpose of this wet macular degeneration clinical trial is to compare GB-102 (1
or 2 mg), administered every six months, to aflibercept (Eyelea) administered
every two months, in patients who have responded to anti-vascular endothelial growth
factor (anti-VEGF) eye injections.
“GB-102 has the potential to help patients control their disease by addressing the treatment burden of wet AMD with a single injection every six months ...” said Arshad M. Khanani, M.D., M.A., Managing Partner and Director of Clinical Research, Sierra Eye Associates, and Clinical Associate Professor of Ophthalmology, University of Nevada.
This Phase 2a study evaluated the therapeutic effect and safety of AKST4290 for the treatment of newly diagnosed wet age related macular degeneration. Twenty-nine patients were given an oral dose of AKST4290 twice per day for 6 weeks. Positive results were shared at the recent 2019 Retina World Congress that showed patients tolerated the medication well with no adverse events experienced during the 6 week trial. Improved Best Corrected Vision Acuity of +7 letters gained was seen in 83% of the participants and 21% of the subjects gained > 15 letters.
“Age-related macular degeneration is the leading cause of age-related blindness,” Karoly Nikolich, PhD, co-founder and chief executive officer of Alkahest, said in a company news release. “Today, the standard of care for this disease involves burdensome intravitreal injections, which can result in under-treatment of patients over time. Alkahest’s data show the potential for an orally-administered agent to safely increase visual acuity in nAMD, which would be a true medical advance for patients.”
"Gene therapy introduces new genes into cells to either replace a faulty gene or to create new gene expression and, potentially, a therapeutic protein."
Allen C. Ho, MD; and Robert L. Avery, MD
Inherited macular degeneration is the result of a faulty gene or set of genes. Using gene therapy a "bad gene" is replaced with a "good gene." Success has been seen in damaged recessive genes rather than dominant genes. Delivery of normal DNA to correct the defective gene puts a normal version of the gene back into the eye so that the protein that is produced by it is more like a healthy functioning cell. Potentially one injection directly into the eye will produce results for the lifetime of the patient.
"Gene therapy providing sustained anti-vascular endothelial growth factor levels in the retina following a single injection could drastically reduce the treatment burden and improve visual outcomes."
OPTIC is a phase 1 clinical trial of ADVM-022 intravitreal injection gene therapy for wet age-related macular degeneration. This Phase 1 study that will evaluate 2 dose levels in 12 subjects (6 per dose) with active choroidal neovascularization (CNV) in patients with macular degeneration. Because this study is designed to assess the safety and tolerability of a single intravitreal injection of ADVM-022 only a small group of patients are being recruited.
The sponsor is Adverum Biotechnologies, Inc. a gene therapy company.
This wet macular degeneration treatment clinical trial is seeking to see if by adding OPT-302 to ranibizumab (Lucentis) for a 6 month period will improve vision and/or effect the anatomical structure of the retina/macula.
Current wet macular degeneration treatments use anti-vascular endothelial growth factors like Lucentis and Eylea , known as anti-VEGF therapy, to inhibit the growth of abnormal and leaking blood vessels. However these drugs are targeting the suppression of VEGF-A, while OPT-302, developed by Australian bio tech company Opthea, will target and block the effect of VEGF-C and VEGF-D.
According to a recent press release from Opthea,"Combined inhibition of VEGF-A and VEGF-C/-D, has the potential to improve patient response by more effective inhibition of the pathways involved in disease progression."
OPT-302 is a VEGF-C/D inhibitor and the Opthea.com website describes how it works, "OPT-302, blocks two members of the vascular endothelial growth factor family, namely VEGF-C and VEGF-D, which cause blood vessels to grow and leak. Aberrant blood vessel growth and vascular leakage are hallmarks of several eye diseases including wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME)."
A phase 2b study is recruiting a total of 351 patients who have never been treated for wet (neovascular) macular degeneration at 50 U.S. sites, 50 European sites and six Israeli sites. The study is a double masked, randomized, sham controlled which means some of the participants will be given the two study drugs while some patients will receive the Lucentis and a sham drug.
The press release of August 2017 shares, "Opthea previously announced positive results of improvements in visual acuity (VA) and retinal swelling (central subfield thickness and sub-retinal fluid) in the Phase 1/2a study patients that included treatment naïve and prior-treated patients receiving OPT-302 + Lucentis® combination therapy."
You can find the study sites and contact information here:
Genentech, which is part of the Swiss company Roche, has moved from a Phase I to a Phase II clinical trial to evaluate the efficacy of an implant that offers a sustained release delivery system.
Currently Lucentis is FDA approved to treat wet macular degeneration through monthly eye injections which require regular visits to the eye doctor. It can be difficult for many patients to comply with these frequent visits to an eye specialist and some find the injections to be uncomfortable or painful which can compromise the control of the leaky blood vessels under the retina.
To determine if an implant can provide a better option for delivery of the drug, Genentech enrolled 179 patients at several study locations in the U.S. Results from the phase 2 study were reported in July, 2018 at the American Society of Retina Specialists 2018 Annual Meeting. Outcomes of the study show that the refillable eye implant allowed the majority of study participants to go 6 months or longer before needing an implant refill.
"To be able to offer our patients the outcomes achieved with monthly injections, but with a device that eliminates the need for those monthly injections, is very exciting," said Carl Awh, MD, from Tennessee Retina in Nashville.
Because of the positive Phase 2 results, Genentech plans to open a Phase 3 trial later this year.
To learn more about the study go to www.ClinicalTrials.gov
Wet macular degeneration is normally treated by frequent anti-VEGF intravitreal injections. Besides being uncomfortable and requiring regular physician office visits, anti-VEGF therapy can become ineffective over time.
SciFluor Life Sciences, Inc. a biopharmaceutical company dedicated to developing drugs that improve outcomes and delivery over existing treatments, is sponsoring a Phase 1/II study to evaluate the safety and preliminary efficacy of SF0166 in patients with wet macular degeneration and diabetetic macular edema.
What makes this study unique is that it will be given as a topical ophthalmic solution, not as an injection. Ben Askew, Ph.D., Vice President of Research at SciFluor and the lead on the SF0166 therapeutic
program, said, “The development of SF0166 provides validation of our fluorine-centered
approach to bringing new transformational therapies to patients with serious illnesses. SF0166
demonstrates how appropriately placed fluorine modifications can improve the physical
properties of a molecule, and address the challenge of getting enough drug to the back of the
eye when administered using much more convenient eye drops.”
The clinical trial is a double blind study meaning that some patients will receive the active study medication and others will receive a placebo. There will be two arms to the study with one group receiving a low dose in the study eye BID or twice a day for 28 days and the other arm will receive a high dose BID for 28 days. The recruitment of participants for this study is now closed but to find out more about this clinical trial go to:
Regeneron Pharmaceuticals and Bayer have teamed up to sponsor a Phase 2 clinical study for those with wet age-related macular degeneration. The study will evaluate the combined therapy of aflibercept (Eylea) and anti-angiopoietin 2 that will be given to patients as a combined single intravitreal injection.
Eylea belongs to the category of anti-VEGF drugs and works to prevent the formation of abnormal and leaky blood vessels in the eye. Angiopoietins work together with vascular endothelial growth factor (VEGF) to promote the development of these unusual blood vessels that leak and cause damage to the macula and to ones vision.
“Bayer is strongly committed to further expanding its ophthalmology portfolio with innovative therapies for patients suffering from vision impairment. Addressing multiple pathways offers potential additional benefit to patients with devastating retinal eye diseases,” said Dr. Joerg Moeller, member of the Executive Committee of Bayer AG’s Pharmaceuticals Division and Head of Development. “Inhibiting the Angiopoietin 2 pathway is a promising new approach for a combination therapy, and we are looking forward to working on it together with Regeneron.”
There will be 3 arms to the randomized double blind study, meaning not everyone will receive the combined therapy. Patients must be 50 years old or older with active choroidal neovascularization in the subfoveal area due to age related macular degeneration. The five study sites in California, South Dakota, Florida, Indiana and Massachusetts have completed their recruitment. To find out more about the study and how to contact a study coordinator go to:
This is a Phase 1 clinical research study which means it is examing the safety and tolerability of an experimental gene transfer agent called AAV2-sFLT01.
It is a multi-centered clinical trial at several
eye centers in the United States such as Johns Hopkins in Baltimore and
Ophthalmic Consultants of Boston. However the study sites have reached their number of needed participants and are no longer recruiting new patients.
The sponsor is Genzyme. The treatment involves a single injection into the eye.
The United Kingdom is offering this Phase IV study at 15 different locations for patients with subfoveal choroidal neovascularisation associated with wet age-related macular degeneration. Here is a description:
"This study tests a new surgical device that delivers a focal dose of radiation (epimacular brachytherapy) to the macula, to try and reduce or eliminate the need for ongoing, regular eye injections. The trial compares epimacular brachytherapy to ongoing standard treatment with ranibizumab."
The Experimental Arm will receive a single therapy with epimacular brachytherapy using the VIDION® System, with Lucentis® (0.5 mg) administered on a monthly basis as required. The Control Arm will receive only the Lucentis injections.
This study is no longer recruiting patients but still ongoing without published results yet. You can learn more about it here: