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M. Gast, W. Yarnall, T. Ciulla, R. Rosen, A. DelConte, C. A. Garcia; Squalamine Lactate Therapy Results in High Levels of VA Stability in Patients With Subfoveal Choroidal Neovascularization Associated With Age-Related Macular Degeneration - A Phase 2, Randomized, Controlled Study. Invest. Ophthalmol. Vis. Sci. 2007;48(13):2874.
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To evaluate the safety profile and efficacy of squalamine lactate (SQ) at two doses compared with vehicle control at 1 year of therapy.
110 Subjects over 50 years of age with a diagnosis of subfoveal choroidal neovascularization (CNV) associated with AMD (all subtypes) were treated with intravenous SQ or vehicle weekly for 4 infusions, followed by 9 monthly infusions. Total lesion size could not exceed 16 disk areas, and a baseline visual acuity (VA) of 24 to 64 ETDRS letters was required for study entry. Subjects were randomly assigned in a 2:2:1 ratio to receive masked intravenous doses of 20 or 40 mg SQ or vehicle control. Photodynamic therapy with verteporfin (PDT) was allowed beginning at week 5 at the investigator’s discretion. Subjects could be re-treated with PDT, if indicated at 12 week intervals. Subjects were withdrawn from the trial if their VA deteriorated by 15 ETDRS letters at 2 consecutive study visits.
Mean age of subjects was 75 years. Forty-six percent of the subjects had active occult disease, 25% had minimally classic and 29% had predominantly classic CNV. Sixty-three percent of the subjects were classified as having bilateral disease. SQ treatment at both doses resulted in a higher level of stable VA than vehicle control alone, although the difference did not achieve statistical significance. In an LOCF analysis, mean VA improved in fellow affected eyes when compared to baseline for both the 20 mg and 40 mg SQ treated groups. Subjects experiencing a doubling of the visual angle were more common in both fellow and study eyes when compared to vehicle controls. This effect was more pronounced in subjects who did not receive PDT. PDT use was approximately twice as common among controls as in the SQ treated subjects. Adverse events (AEs) were generally evenly distributed between the two active treatment groups and the controls. There were no drug related serious adverse events reported. The most common drug related AEs were infusion site-related and were reported to be mild to moderate in severity. Infusion site pain in the SQ treated groups appeared to be dose related.
These results suggest that SQ therapy is safe and well tolerated. Both the 20 and 40 mg doses of SQ stabilized vision in study eyes and fellow affected eyes in the majority of subjects. Improvement in fellow eye vision was most pronounced in the subgroup of subjects who began the study with a VA of less than 34 ETDRS letters.
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