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Completed

NCT Number: NCT02766855

Efficacy of Topical Cysteamine in Nephropathic Cyctinosis

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.

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Key information

Conditions

Nephropathic Cyctinosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Corneal Cystine Crystals Cystinosis Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

8 month–19 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Cysteamine

Drug

Patients were asked to use topical cysteamine 0.55% eye drops every 2 hours while awake to both eyes.

Primary outcomes

  1. 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.

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

The Eye Center and The Eye Foundation for Research in Ophthalmology

Other

Registry information

Important dates

Study start
2004
Primary completion
2012
Study completion
2016
First posted
May 10, 2016
Registry last updated
May 10, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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