TY - JOUR
T1 - Treatment of geographic atrophy by the topical administration of OT-551
T2 - Results of a phase ii clinical trial
AU - Wong, Wai T.
AU - Kam, Waynekid
AU - Cunningham, Denise
AU - Harrington, Molly
AU - Hammel, Keri
AU - Meyerle, Catherine B.
AU - Cukras, Catherine
AU - Chew, Emily Y.
AU - Sadda, Srinivas R.
AU - Ferris, Frederick L.
PY - 2010/12
Y1 - 2010/12
N2 - PURPOSE. To investigate the safety and preliminary efficacy of OT-551, a disubstituted hydroxylamine with antioxidant properties, for the treatment of geographic atrophy (GA), the advanced atrophic form of age-related macular degeneration (AMD). METHODS. The study was a single-center, open-label phase II trial, enrolling 10 participants with bilateral GA. Topical 0.45% OT-551 was administered in one randomly assigned eye three times daily for 2 years. Safety measures were assessed by complete ophthalmic examination, fundus photography, and review of symptoms. The primary efficacy outcome measure was the change in best corrected visual acuity at 24 months. Secondary efficacy measures included changes in area of GA, contrast sensitivity, microperimetry measurements, and total drusen area from baseline. RESULTS. Study drug was well tolerated and was associated with few adverse events. The mean change in BCVA at 2 years was+0. 2 ± 13. 3 letters in the study eyes and-11. 3 ± 7. 6 letters in fellow eyes (P= 0. 0259). However, no statistically significant differences were found between the study and fellow eyes for all other secondary outcome measures. CONCLUSIONS. OT-551 was well tolerated by study participants and was not associated with any serious adverse effects. Efficacy measurements in this small study indicate a possible effect in maintaining visual acuity. However, the absence of significant effects on other outcomes measures in this study suggests that OT-551, in the current concentration and mode of delivery, may have limited or no benefit as a treatment for GA.
AB - PURPOSE. To investigate the safety and preliminary efficacy of OT-551, a disubstituted hydroxylamine with antioxidant properties, for the treatment of geographic atrophy (GA), the advanced atrophic form of age-related macular degeneration (AMD). METHODS. The study was a single-center, open-label phase II trial, enrolling 10 participants with bilateral GA. Topical 0.45% OT-551 was administered in one randomly assigned eye three times daily for 2 years. Safety measures were assessed by complete ophthalmic examination, fundus photography, and review of symptoms. The primary efficacy outcome measure was the change in best corrected visual acuity at 24 months. Secondary efficacy measures included changes in area of GA, contrast sensitivity, microperimetry measurements, and total drusen area from baseline. RESULTS. Study drug was well tolerated and was associated with few adverse events. The mean change in BCVA at 2 years was+0. 2 ± 13. 3 letters in the study eyes and-11. 3 ± 7. 6 letters in fellow eyes (P= 0. 0259). However, no statistically significant differences were found between the study and fellow eyes for all other secondary outcome measures. CONCLUSIONS. OT-551 was well tolerated by study participants and was not associated with any serious adverse effects. Efficacy measurements in this small study indicate a possible effect in maintaining visual acuity. However, the absence of significant effects on other outcomes measures in this study suggests that OT-551, in the current concentration and mode of delivery, may have limited or no benefit as a treatment for GA.
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U2 - 10.1167/iovs.10-5637
DO - 10.1167/iovs.10-5637
M3 - Review article
C2 - 20574018
AN - SCOPUS:79955056247
SN - 0146-0404
VL - 51
SP - 6131
EP - 6139
JO - Investigative Ophthalmology and Visual Science
JF - Investigative Ophthalmology and Visual Science
IS - 12
ER -