Pazopanib eye drops: a randomised trial in neovascular age-related macular degeneration

Br J Ophthalmol. 2014 Feb;98(2):172-8. doi: 10.1136/bjophthalmol-2013-303117. Epub 2013 Nov 13.

Abstract

Aims: To evaluate pazopanib eye drops in patients with subfoveal choroidal neovascularisation secondary to age-related macular degeneration.

Methods: 70 patients with minimally classic or occult subfoveal choroidal neovascularisation were randomly assigned to 5 mg/mL TID, 2 mg/mL TID, and 5 mg/mL QD pazopanib eye drops for 28 days in a multicentre, double-masked trial with an optional safety extension for up to 5 additional months. The primary outcomes were central retinal thickness (CRT) and best-corrected visual acuity (BCVA) at Day 29.

Results: No significant decrease from baseline in CRT was observed overall; however, an exploratory analysis showed improvement in CRT (mean decrease of 89 μm) in patients with the CFH TT genotype who received 5 mg/mL TID (p=0.01, n=5). Mean increases in BCVA were observed in the 5 mg/mL TID overall (4.32 letters (p=0.002, n=26)) and in those that with CFH Y402H TT (6.96 letters (p=0.02, n=5)) and CT (4.09 letters (p=0.05, n=9)) genotypes. No safety signals that precluded continued investigation were detected.

Conclusions: 5 mg/mL pazopanib eye drops resulted in mean improvement in BCVA at Day 29 and improvements in vision. However, improvement in macular oedema for age-related macular degeneration was found only in the subset of subjects with the CFH Y402H TT genotype, warranting further investigation.

Keywords: Clinical Trial; Diagnostic tests/Investigation; Drugs; Macula; Neovascularisation.

Publication types

  • Comparative Study
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Angiogenesis Inhibitors
  • Choroidal Neovascularization / complications
  • Choroidal Neovascularization / drug therapy*
  • Choroidal Neovascularization / metabolism
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Female
  • Follow-Up Studies
  • Humans
  • Indazoles
  • Macular Degeneration / complications
  • Macular Degeneration / drug therapy*
  • Macular Degeneration / metabolism
  • Male
  • Middle Aged
  • Ophthalmic Solutions
  • Prospective Studies
  • Pyrimidines / administration & dosage*
  • Pyrimidines / pharmacokinetics
  • Sulfonamides / administration & dosage*
  • Sulfonamides / pharmacokinetics
  • Treatment Outcome
  • Visual Acuity

Substances

  • Angiogenesis Inhibitors
  • Indazoles
  • Ophthalmic Solutions
  • Pyrimidines
  • Sulfonamides
  • pazopanib