April 2010
Volume 51, Issue 13
Free
ARVO Annual Meeting Abstract  |   April 2010
Targeting Complement Factor 5 in Combination with Vascular Endothelial Growth Factor (VEGF) Inhibition for Neovascular Age Related Macular Degeneration (AMD): Results of a Phase 1 Study
Author Affiliations & Notes
  • S. W. Cousins
    Ophthalmology, Duke University, Durham, North Carolina
  • Ophthotech Study Group
    Ophthalmology, Duke University, Durham, North Carolina
  • Footnotes
    Commercial Relationships  S.W. Cousins, Alcon, Allergan, Eyetech, Genentech, Heidelberg Engineering, Novartis, Ophthotech, C; Alcon, Allergan, Eyetech, Genentech, Heidelberg Engineering, Novartis, Ophthotech, Pfizer, R.
  • Footnotes
    Support  Ophthotech
Investigative Ophthalmology & Visual Science April 2010, Vol.51, 1251. doi:
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    • Get Citation

      S. W. Cousins, Ophthotech Study Group; Targeting Complement Factor 5 in Combination with Vascular Endothelial Growth Factor (VEGF) Inhibition for Neovascular Age Related Macular Degeneration (AMD): Results of a Phase 1 Study. Invest. Ophthalmol. Vis. Sci. 2010;51(13):1251.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose: : Genetic linkage and pathobiological studies have implicated activation of the complement system in AMD. Preclinical studies confirm a role for complement effector molecules in the induction and severity of choroidal neovascularization. This study assessed the safety of intravitreal injections of the aptamer ARC1905, which inhibits activation of complement factor 5, in combination with ranibizumab for the treatment of neovascular AMD.

Methods: : A phase 1, prospective, noncontrolled multicenter, dose escalating study was initiated in 58 patients with all subtypes of subfoveal neovascular AMD. Best-corrected visual acuity (VA) in the study eye was 20/63 to 20/200. Subjects received 3-6 monthly administrations of ARC1905 (0.03, 0.3, 1, or 2 mg) in combination with ranibizumab (0.5mg). Secondary endpoints included visual acuity and OCT. Complement-associated SNP analysis was conducted in a cohort of patients.

Results: : Dose escalation was completed without evidence of dose-limiting toxicity. Forty-eight (48) subjects received at least 2 doses of the experimental regimen. The mean change in VA was +10.2 letters at Week 8. Thirty-five percent of patients gained ≥3 lines of VA. The mean change in OCT center point thickness was -124µm.

Conclusions: : The combination of C5 and VEGF inhibition in neovascular AMD is well tolerated without evidence of acute toxicity.

Clinical Trial: : www.clinicaltrials.gov NCT00709527

Keywords: age-related macular degeneration • inflammation 
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