Efficacy of Topical Cysteamine in Nephropathic Cyctinosis

Completed 26 enrolled The Eye Center and The Eye Foundation for Research in Ophthalmology

NaSingle-groupOpen-labelTreatment

Summary

The aim of this study is to evaluate the efficacy of topical cysteamine eye drops in the treatment of corneal cystine crystals deposits in patients with nephropathic cystinosis.

Timeline

Start: 2004-01
Primary completion: 2012-12
Completion: 2016-03

Outcome

Missed primary endpoint

paper Improvement of photophobia was not clinically significant in symptomatic patients. PMID 28057644 ↗

paper Patients displayed statistically significant worsening of corneal cystine deposits during the follow-up period. PMID 28057644 ↗

Publications

Al-Hemidan A, Shoughy SS, Kozak I, Tabbara KF. Efficacy of topical cysteamine in nephropathic cystinosis. Br J Ophthalmol. 2017 Sep;101(9):1234-1237. doi: 10.1136/bjophthalmol-2016-309278. Epub 2017 Jan 5.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Cysteamine	Unknown	0.55 %	Topical