Efficacy of Topical Cysteamine in Nephropathic Cyctinosis

May 9, 2016 updated by: Samir S. Shoughy, MD, FRCS (Glasg.), The Eye Center and The Eye Foundation for Research in Ophthalmology
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

26

Phase

  • Not Applicable

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 months to 19 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: cysteamine eye drops
Patients used cysteamine eye drops every 2 hours while awake to both eyes.
Drug: Cysteamine
Patients were asked to use topical cysteamine 0.55% eye drops every 2 hours while awake to both eyes.

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The Eye Center and The Eye Foundation for Research in Ophthalmology

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2004

Primary Completion (ACTUAL)

December 1, 2012

Study Completion (ACTUAL)

March 1, 2016

Study Registration Dates

First Submitted

April 12, 2016

First Submitted That Met QC Criteria

May 9, 2016

First Posted (ESTIMATE)

May 10, 2016

Study Record Updates

Last Update Posted (ESTIMATE)

May 10, 2016

Last Update Submitted That Met QC Criteria

May 9, 2016

Last Verified

May 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TEC C123

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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