Efficacy of Topical Cysteamine in Nephropathic Cyctinosis

NCT02766855 | Completed

• 1 other identifier: TEC C123
• Study Type: interventional
• Target: 26
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Nephropathic Cyctinosis; Corneal Cystine Crystals

Keywords

cysteamine; nephropathic cystinosis; cystine crystals

Outcome Measures

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.

  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.

  Photophobia, slit lamp assessment of corneal cystine crystals and visual acuity are recorded pretreatment, on each monthly visit up to 48 months.

Study Arms (1)

cysteamine eye drops

EXPERIMENTAL

Patients used cysteamine eye drops every 2 hours while awake to both eyes.

Drug: Cysteamine

Interventions

Cysteamine DRUG

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

cysteamine eye drops

Eligibility Criteria

• Age: 8 Months - 19 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• Allergy to cysteamine

Sponsors & Collaborators

• The Eye Center and The Eye Foundation for Research in Ophthalmology: lead

Related Publications (1)

• 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.

  PMID: 28057644 DERIVED

MeSH Terms

Conditions

Cystinosis

Interventions

Cysteamine

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Mercaptoethylamines; Ethylamines; Amines; Organic Chemicals; Sulfhydryl Compounds; Sulfur Compounds

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Ophthamolgist, uveitis, Cornea & External Eye Disease

Study Record Dates

• First Submitted: April 12, 2016
• First Posted: May 10, 2016
• Study Start: January 1, 2004
• Primary Completion: December 1, 2012
• Study Completion: March 1, 2016
• Last Updated: May 10, 2016

Record last verified: 2016-05