Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 4 of 33 for:    CYSTEAMINE

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

Tracking Information
First Submitted Date  ICMJE April 12, 2016
First Posted Date  ICMJE May 10, 2016
Last Update Posted Date May 10, 2016
Study Start Date  ICMJE January 2004
Actual Primary Completion Date December 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 9, 2016)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy of Topical Cysteamine in Nephropathic Cyctinosis
Official Title  ICMJE Not Provided
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 Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Nephropathic Cyctinosis
  • Corneal Cystine Crystals
Intervention  ICMJE Drug: Cysteamine
Patients were asked to use topical cysteamine 0.55% eye drops every 2 hours while awake to both eyes.
Study Arms  ICMJE Experimental: cysteamine eye drops
Patients used cysteamine eye drops every 2 hours while awake to both eyes.
Intervention: Drug: Cysteamine
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 9, 2016)
26
Original Actual Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE March 2016
Actual Primary Completion Date December 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 8 Months to 19 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02766855
Other Study ID Numbers  ICMJE TEC C123
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Samir S. Shoughy, MD, FRCS (Glasg.), The Eye Center and The Eye Foundation for Research in Ophthalmology
Study Sponsor  ICMJE The Eye Center and The Eye Foundation for Research in Ophthalmology
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account The Eye Center and The Eye Foundation for Research in Ophthalmology
Verification Date May 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP