Positive results from a Phase II advanced dry macular degeneration study has opened doors to a Phase III clinical trial  involving more than 900 patients in 20 countries.  The study will run for two years.

The drug Lampalizumab “slowed progression of dry, aged related macular degeneration in patients with advanced disease, shrinking the area of geographic atrophy (GA) by 20.4%” according to the Phase II MAHALO study results.   The principal investigator, Carl Regillo, MD, from the Wills Eye Hospital in Philadelphia, Pennsylvania states, “MAHALO is the first study to demonstrate a positive treatment effect with a complement inhibitor in geographic atrophy.”  The main purpose of the Phase III trial will be the same – to see if the drug can promote a change in the GA area.  Evaluations to monitor these changes will be by retinal imaging.

Eligibility and Intervention

The drug will be given by intravitreal injection.  However, not every participant will be given the drug.  The study is a randomized, double-blind, placebo controlled study.  This means that some patients will get the drug and others will get a placebo or sham.  Neither the participant nor the retinal doctor will know which patients are receiving the drug or the placebo until the end of the study.

Eligible candidates for the Phase III study must be 50 years or older and have evidence of Geographic Atrophy (GA) caused by macular degeneration with no choroidal neovascularization (wet AMD)  in both eyes.

Inhibiting the Complement Pathway

Lampalizumab works by inhibiting the complement pathway.  Ophthalmologist Phil Rosenfeld of Bascom Palmer Eye Institute in Miami, explains complement activation this way, “The complement pathway is responsible for preventing infection. When the pathway gets activated by bacteria, viruses, and foreign tissue, it destroys the invading organisms and transplanted organs. During the activation process, normal tissue can get damaged. In AMD, it is believed that people with AMD carry mutations in their DNA that cause the complement system to become over-activated and the pathway accidentally destroys normal tissue.

“Lampalizumab has the potential to represent a significant breakthrough for this disease and could provide real hope for GA patients,” said Sandra Horning, Roche’s chief medical officer.  “It is the only ophthalmic drug in clinical development that specifically targets complement factor D.”

Complement Activation in Macular Degeneration

Leslie Degner, RN, BSN

www.WebRN-MacularDegeneration.com