Efficacy of Topical Cysteamine in Nephropathic Cyctinosis

Not Applicable

Completed

• Conditions: Corneal Cystine Crystals; Nephropathic Cyctinosis
• Interventions: Drug: Cysteamine

• Registration Number: NCT02766855
• Lead Sponsor: The Eye Center and The Eye Foundation for Research in Ophthalmology

Brief 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.

Detailed Description

A total of 26 patients with nephropathic cystinosis were included in this prospective study. Patients were evaluated for improvement of corneal cysteine deposits crystals and photophobia following treatment with topical 0.55% cysteamine eye drops.

Study Timeline

• Study Start: 2004-01
• Primary Completion: 2012-12
• Study Completion: 2016-03
• Study First Submitted: 2016-04-12
• Status Verified: 2016-05
• Study First Submitted QC: 2016-05-09
• Last Update Submitted: 2016-05-09
• Study First Posted: 2016-05-10
• Last Update Posted: 2016-05-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 26

Inclusion Criteria

• patients with confirmed diagnosis of nephropathic cystinosis
• leucocyte cystine concentration level of more than 2.0 nmol half-cystine/ mg protein
• clinical evidence of tissue deposition of cystine and tissue damage due to cystinosis

Exclusion Criteria

• Allergy to cysteamine

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
cysteamine eye drops	Cysteamine	Patients used cysteamine eye drops every 2 hours while awake to both eyes.

Primary Outcome Measures

Name	Time	Method
Number of participants with improvement of photophobia, corneal cystine crystals and visual acuity following treatment with topical cysteamine 0.55% eye drops in patients with nephropathic cystinosis.	Photophobia, slit lamp assessment of corneal cystine crystals and visual acuity are recorded pretreatment, on each monthly visit up to 48 months.	Patients had a baseline ophthalmic examination before initiation of topical cysteamine 0.55% eye drops and subsequent monthly follow up exams. The following parameters were recorded pretreatment, on each monthly visit and post treatment at last follow up visit: photophobia, slit lamp assessment of corneal cystine crystals and visual acuity. Photophobia was classified as grade 0 (none) for no photophobia, grade 1 (mild) for photophobia in bright light, grade 2 (moderate) for photophobia in room light and grade 3 (severe) for photophobia in dim light. Corneal cystine crystals were graded as Grade 0 = None, Grade 1 = 1-10 crystals/mm, Grade 2 = 11- 50 crystals/mm, Grade 3 = more than 50 crystals/mm. The level of cystine in the corneal layers was recorded and the location whether peripheral or central was also identified. Visual acuity was measured by Snellen chart at 20 feet with best distance correction.