Wilson DiseaseWILLYNCT04408300
Study of Retinal Vascular Parameters in Patients With Wilson's Disease
A study testing Ophthalmological exam in people with Wilson Disease, run by Fondation Ophtalmologique Adolphe de Rothschild at 1 site in France.
Trial summary
The rare disease reference center " Wilson disease and other rare copper-related diseases" of the Rothschild Foundation follows a large number of patients with Wilson's with varying degrees of impairment and located at different times of their care. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Wilson Disease
- What is being tested?
- Ophthalmological exam
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 124 participants
- Where
- 1 siteFrance
- Study dates
- 4 Jun 2020to 15 Feb 2022
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Wilson's disease with Ferenci score ≥4
Who cannot join
- Neurological or behavioral handicap that does not allow for ophthalmological examinations
- Hepatic decompensation that does not allow for ophthalmological examinations
- Pregnant or breastfeeding woman
Trial locations and contacts
1 site in 1 country.
About this study
The rare disease reference center " Wilson disease and other rare copper-related diseases" of the Rothschild Foundation follows a large number of patients with Wilson's with varying degrees of impairment and located at different times of their care.
Many people with Wilson's disease have a characteristic greenish-brown ring, known as Kayser-Fleischer, appearing at the periphery of the cornea due to a deposit of copper at the Descemet membrane. As a general rule, if the patient is compliant with his treatment, the ring usually disappears within a few years, although it may persist in some patients. However, apart from the stage of diagnosis, and the evolution of the ring, ophthalmological examinations are little used for the follow-up of these patients.
The objective of this study is to describe the retinal parameters, in particular vascular with two new retinal imaging technologies (OCT-A :Optical Coherence Tomography-Angiography , Adaptive optics) in patients with Wilson's disease and to correlate them with the parameters of the usual follow-up of these patients (hepatic assessment, exchangeable plasma copper, neurological scores, compliance, etc.).
- Conditions
- Wilson Disease
- Being tested
- Ophthalmological exam
- Keywords
- Optical Coherence Tomography, Adaptive optics
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ophthalmological examProcedure or surgery
Retinophotography, OCT-A (Optical Coherence Tomography- Angiography), OCT-B scan (Optical Coherence Tomography B Scan), Measurement of corrected visual acuity in ETDRS (Early Treatment Diabetic Retinopathy Study), Measurement of intraocular pressure, adaptive optics
| Study group | Role | What they receive |
|---|---|---|
| Ophthalmological exam | Experimental | Not described Receives: Procedure: Ophthalmological exam |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measuring the width of the Kayser-Fleischer ring
OCT-B scan
Measured: Enrollment
Corrected visual acuity measurement
ETDRS
Measured: Enrollment
Intraocular pressure measurement (mmHg)
Measured: Enrollment
Measurement of the thickness of the retinal nerve fibers layer (RNFL)
OCT-B
Measured: Enrollment
Measurement of the central avascular zone (in µm2)
OCT-A
Measured: Inclusion
Measurement of the capillary density of the superficial plexus (in%)
OCT-A
Measured: Enrollment
Measurement of the capillary density of the deep plexus (in%)
OCT-A
Measured: Enrollment
Measurement of photoreceptor density (per mm2)
Adaptive optics
Measured: Enrollment
Measurement of wall to lumen ratio (in %)
Adaptive optics
Measured: Enrollment
Measurement of the arterial wall section area (µm²)
Adaptive optics
Measured: Enrollment
Measurement of the diameter of the arterial lumen (µm)
Adaptive optics
Measured: Enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 May 2020
- Study start4 Jun 2020
- Primary completion6 Jan 2022
- Study completion15 Feb 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wilson DiseaseStudies of the same condition, open and under way.
- More trials run by Fondation Ophtalmologique Adolphe de RothschildThe sponsor's other registered studies.
- 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
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 124 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Aurélia POUJOIS, MD, Principal investigator, Fondation A. de Rothschild
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04408300
- Sponsor's protocol ID
- APS_2020_5
- Acronym
- WILLY
- Status last verified
- Mar 2022
- First submitted
- 26 May 2020
- Last updated
- 3 Mar 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Retinal Vascular Parameters in Patients With Wilson's Disease
- ClinicalTrials.gov ID
- NCT04408300
- Acronym / sponsor ID
- WILLY / APS_2020_5
- Conditions
- Wilson Disease
- Keywords
- Optical Coherence Tomography, Adaptive optics
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 124 participants (actual)
- Lead sponsor
- Fondation Ophtalmologique Adolphe de RothschildResearch network
- Study start
- 4 Jun 2020 (actual)
- Primary completion
- 6 Jan 2022 (actual)
- Study completion
- 15 Feb 2022 (actual)
- First posted
- 29 May 2020 (actual)
- 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 rare disease reference center " Wilson disease and other rare copper-related diseases" of the Rothschild Foundation follows a large number of patients with Wilson's with varying degrees of impairment and located at different times of their care.
Many people with Wilson's disease have a characteristic greenish-brown ring, known as Kayser-Fleischer, appearing at the periphery of the cornea due to a deposit of copper at the Descemet membrane. As a general rule, if the patient is compliant with his treatment, the ring usually disappears within a few years, although it may persist in some patients. However, apart from the stage of diagnosis, and the evolution of the ring, ophthalmological examinations are little used for the follow-up of these patients.
The objective of this study is to describe the retinal parameters, in particular vascular with two new retinal imaging technologies (OCT-A :Optical Coherence Tomography-Angiography , Adaptive optics) in patients with Wilson's disease and to correlate them with the parameters of the usual follow-up of these patients (hepatic assessment, exchangeable plasma copper, neurological scores, compliance, etc.).
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ophthalmological exam | Experimental | Procedure: Ophthalmological exam |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Wilson's disease with Ferenci score ≥4
Key exclusion criteria (3 listed)
- Neurological or behavioral handicap that does not allow for ophthalmological examinations
- Hepatic decompensation that does not allow for ophthalmological examinations
- Pregnant or breastfeeding woman
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 |
|---|---|---|
| Ophthalmological examRetinophotography, OCT-A (Optical Coherence Tomography- Angiography), OCT-B scan (Optical Coherence Tomography B Scan), Measurement of corrected visual acuity in ETDRS (Early Treatment Diabetic Retinopathy Study), Measurement of intraocular pressure, adaptive optics | Procedure or surgery | Ophthalmological exam |
Primary outcome
Measuring the width of the Kayser-Fleischer ring
Time frame: Enrollment
OCT-B scan
Corrected visual acuity measurement
Time frame: Enrollment
ETDRS
Intraocular pressure measurement (mmHg)
Time frame: Enrollment
Measurement of the thickness of the retinal nerve fibers layer (RNFL)
Time frame: Enrollment
OCT-B
Measurement of the central avascular zone (in µm2)
Time frame: Inclusion
OCT-A
Measurement of the capillary density of the superficial plexus (in%)
Time frame: Enrollment
OCT-A
Measurement of the capillary density of the deep plexus (in%)
Time frame: Enrollment
OCT-A
Measurement of photoreceptor density (per mm2)
Time frame: Enrollment
Adaptive optics
Measurement of wall to lumen ratio (in %)
Time frame: Enrollment
Adaptive optics
Measurement of the arterial wall section area (µm²)
Time frame: Enrollment
Adaptive optics
Measurement of the diameter of the arterial lumen (µm)
Time frame: Enrollment
Adaptive optics
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 3 Mar 2022.
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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.
Aurélia POUJOIS, MD principal investigator
Fondation A. de Rothschild
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Ophthalmological exam in people with Wilson Disease, run by Fondation Ophtalmologique Adolphe de Rothschild at 1 site in France.
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 1 inclusion and 3 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 3 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
Measuring the width of the Kayser-Fleischer ring (time frame: Enrollment). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Mar 2022.