Nephropathic CyctinosisNCT02766855
Efficacy of Topical Cysteamine in Nephropathic Cyctinosis
A study testing Cysteamine in people with Nephropathic Cyctinosis and Corneal Cystine Crystals, run by The Eye Center and The Eye Foundation for Research in Ophthalmology.
Trial 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. Read more about this study
- Who is it for?
- Ages 8 months to 19 years
- Conditions the study is for
- Nephropathic Cyctinosis, Corneal Cystine Crystals
- What is being tested?
- Cysteamine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- No sites listed yet
- Study dates
- Jan 2004to Mar 2016
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 months to 19 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- Allergy to cysteamine
About this study
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.
Read the 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.
- Conditions
- Nephropathic Cyctinosis, Corneal Cystine Crystals
- Being tested
- Cysteamine
- Keywords
- cysteamine, nephropathic cystinosis, cystine crystals
- Medical subject headings
- Cystinosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CysteamineDrug
Patients were asked to use topical cysteamine 0.55% eye drops every 2 hours while awake to both eyes.
| Study group | Role | What they receive |
|---|---|---|
| cysteamine eye drops | Experimental | Patients used cysteamine eye drops every 2 hours while awake to both eyes. Receives: Drug: Cysteamine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Photophobia, slit lamp assessment of corneal cystine crystals and visual acuity are recorded pretreatment, on each monthly visit up to 48 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 May 2016 (estimated)
- Study startJan 2004
- Primary completionDec 2012
- Study completionMar 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Nephropathic CyctinosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Eye Center and The Eye Foundation for Research in Ophthalmology
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Samir S. Shoughy, MD, FRCS (Glasg.), The Eye Center and The Eye Foundation for Research in Ophthalmology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02766855
- Sponsor's protocol ID
- TEC C123
- Status last verified
- May 2016
- First submitted
- 12 Apr 2016
- Last updated
- 10 May 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
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.
PubMed 28057644
Study at a glance
From the registry record, last updated by the sponsor on 10 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Topical Cysteamine in Nephropathic Cyctinosis
- ClinicalTrials.gov ID
- NCT02766855
- Acronym / sponsor ID
- TEC C123
- Conditions
- Nephropathic Cyctinosis, Corneal Cystine Crystals
- Keywords
- cysteamine, nephropathic cystinosis, cystine crystals
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- The Eye Center and The Eye Foundation for Research in OphthalmologyUniversity, hospital or other
- Study start
- Jan 2004
- Primary completion
- Dec 2012 (actual)
- Study completion
- Mar 2016 (actual)
- First posted
- 10 May 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| cysteamine eye drops | Experimental | Drug: CysteaminePatients used cysteamine eye drops every 2 hours while awake to both eyes. |
Who may qualify
- Ages
- 8 months to 19 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (1 listed)
- Allergy to cysteamine
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CysteaminePatients were asked to use topical cysteamine 0.55% eye drops every 2 hours while awake to both eyes. | Drug | cysteamine eye drops |
Primary outcome
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 May 2016.
- Derived from this study: PubMed 28057644Al-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.
- Individual participant dataSharing planned: Undecided
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Cysteamine in people with Nephropathic Cyctinosis and Corneal Cystine Crystals, run by The Eye Center and The Eye Foundation for Research in Ophthalmology.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 May 2016. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02766855, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 May 2016.