We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Efficacy of Topical Cysteamine in Nephropathic Cyctinosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02766855
Recruitment Status : Completed
First Posted : May 10, 2016
Last Update Posted : May 10, 2016
Sponsor:
Information provided by (Responsible Party):
Samir S. Shoughy, MD, FRCS (Glasg.), The Eye Center and The Eye Foundation for Research in Ophthalmology

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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.

Condition or disease Intervention/treatment Phase
Nephropathic Cyctinosis Corneal Cystine Crystals Drug: Cysteamine Not Applicable

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 Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 26 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Study Start Date : January 2004
Actual Primary Completion Date : December 2012
Actual Study Completion Date : March 2016

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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.


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  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.


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   8 Months to 19 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

No Contacts or Locations Provided
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Layout table for additonal information
Responsible Party: Samir S. Shoughy, MD, FRCS (Glasg.), Ophthamolgist, uveitis, Cornea & External Eye Disease, The Eye Center and The Eye Foundation for Research in Ophthalmology
ClinicalTrials.gov Identifier: NCT02766855    
Other Study ID Numbers: TEC C123
First Posted: May 10, 2016    Key Record Dates
Last Update Posted: May 10, 2016
Last Verified: May 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by Samir S. Shoughy, MD, FRCS (Glasg.), The Eye Center and The Eye Foundation for Research in Ophthalmology:
cysteamine
nephropathic cystinosis
cystine crystals
Additional relevant MeSH terms:
Layout table for MeSH terms
Cysteamine
Cystine Depleting Agents
Molecular Mechanisms of Pharmacological Action