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Trial registered on ANZCTR


Registration number
ACTRN12618000738224
Ethics application status
Approved
Date submitted
25/04/2018
Date registered
3/05/2018
Date last updated
3/05/2018
Type of registration
Retrospectively registered

Titles & IDs
Public title
The Western Australia Retinal Degeneration Study
Scientific title
The Western Australia Retinal Degeneration Study: An natural history observational cohort study of retinal degenerations and in vitro retinal disease modelling using patient derived stem cells
Secondary ID [1] 294715 0
Nil
Universal Trial Number (UTN)
U1111-1212-8843
Trial acronym
WARDS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
retinal disease 307588 0
genetic eye disease 307589 0
macular degeneration 307590 0
Condition category
Condition code
Eye 306651 306651 0 0
Diseases / disorders of the eye
Human Genetics and Inherited Disorders 306652 306652 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Observational
Patient registry
True
Target follow-up duration
5
Target follow-up type
Years
Description of intervention(s) / exposure
Patients with retinal disease due to genetic mutation, age-related degeneration, post-inflammatory degeneration or vascular disease-related degeneration will be observed for 5 years. Blood sampling and skin biopsy will be obtained at baseline for biomarker study. Each patient will undergo retinal imaging once every 6 months during the course of the study.
Intervention code [1] 301009 0
Early Detection / Screening
Comparator / control treatment
Patients with no retinal disease and / or genetic mutation that can cause retinal disease
Control group
Active

Outcomes
Primary outcome [1] 305651 0
Visual acuity as measured on the Early Treatment of Diabetic Retinopathy Study (ETDRS) letter chart.
Timepoint [1] 305651 0
Post enrolment: 6 month, 12 month, 18 month, 24 months (primary timepoint), 30 months, 36 months, 42 months, 48 months and 60 months
Primary outcome [2] 305701 0
Rate of change in total macular volume (mm3) and retinal thickness in ETDRS zones using spectral domain optical coherence tomography.
Timepoint [2] 305701 0
Post enrolment: 6 month, 12 month, 18 month, 24 months (primary timepoint), 30 months, 36 months, 42 months, 48 months and 60 months
Secondary outcome [1] 346045 0
Rate of change in area of atrophy (mm2) and number of new hyperautofluorescent lesions using Spectralis fundus autofluorescence imaging (30 and 55 degrees) and Optos fundus autofluorescence imaging (200 degrees).
Timepoint [1] 346045 0
Post enrolment: 6 month, 12 month, 18 month, 24 months, 30 months, 36 months, 42 months, 48 months and 60 months
Secondary outcome [2] 346046 0
Cone density (cones per degree2), separation (degrees) and packing arrangement (% hexagonal voronoi) issing rtx1 AO camera (adaptive optics).
Timepoint [2] 346046 0
Post enrolment: 6 month, 12 month, 18 month, 24 months, 30 months, 36 months, 42 months, 48 months and 60 months
Secondary outcome [3] 346047 0
Rate of change in mean and median macular mesopic retinal sensitivity (dB) and number of dense scotoma loci using fundus controlled MAIA microperimeter device
Timepoint [3] 346047 0
Post enrolment: 6 month, 12 month, 18 month, 24 months, 30 months, 36 months, 42 months, 48 months and 60 months
Secondary outcome [4] 346357 0
Antiretinal auto-antibodies will be examined using Western blot and immunohistochemistry against retinal tissue.
Timepoint [4] 346357 0
Serum will be collected at baseline study visit. The presence and profile of autoantibodies against human retina will be correlated with disease progression rates.

Eligibility
Key inclusion criteria
Two groups of patient are included:
(1) Patients with retinal disease due to age-related retinal degeneration, inherited retinal disease, post-inflammatory retinal degeneration, vascular disease associated retinal degeneration
(2) Subjects with no retinal disease as controls
Minimum age
10 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
Patients or their guardian unable to to give informed consent

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Convenience sample
Timing
Prospective
Statistical methods / analysis
Median, mean, standard deviation and range will be calculated for all variables. Normality of data distribution will be assessed to guide the choice of parametric vs non-parametric methods. Changes in visual acuity and retinal imaging parameters will be analysed by multivariate regression controlling for patient and eye independent variables.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
1/12/2015
Date of last participant enrolment
Anticipated
20/11/2020
Actual
Date of last data collection
Anticipated
31/12/2025
Actual
Sample size
Target
1000
Accrual to date
510
Final
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 10786 0
Lions Eye Institute Day Surgery Centre - Nedlands
Recruitment postcode(s) [1] 22525 0
6009 - Nedlands

Funding & Sponsors
Funding source category [1] 299321 0
Charities/Societies/Foundations
Name [1] 299321 0
Lions Eye Institute
Country [1] 299321 0
Australia
Funding source category [2] 299349 0
Self funded/Unfunded
Name [2] 299349 0
Dr Fred Chen
Country [2] 299349 0
Australia
Primary sponsor type
Charities/Societies/Foundations
Name
Lions Eye Institute
Address
2 Verdun Street, Nedlands, WA 6009
Country
Australia
Secondary sponsor category [1] 298624 0
Individual
Name [1] 298624 0
Dr Fred Chen
Address [1] 298624 0
Lions Eye Institute
2 Verdun Street, Nedlands, WA 6009
Country [1] 298624 0
Australia
Other collaborator category [1] 280084 0
Individual
Name [1] 280084 0
Professor Steven Wilton
Address [1] 280084 0
The Perron Institute for Neurological and Translational Science
RR Block, QE II Medical Centre, 8 Verdun St, Nedlands WA 6009
Country [1] 280084 0
Australia
Other collaborator category [2] 280085 0
Individual
Name [2] 280085 0
Dr David Alonso-Caneiro
Address [2] 280085 0
Queensland University of Technology
O Block (B wing), Room B556, Victoria Park Road , Kelvin Grove, Brisbane, QLD 4059
Country [2] 280085 0
Australia
Other collaborator category [3] 280086 0
Individual
Name [3] 280086 0
Professor Sue Fletcher
Address [3] 280086 0
Murdoch University
90 South St, Murdoch WA 6150
Country [3] 280086 0
Australia
Other collaborator category [4] 280087 0
Individual
Name [4] 280087 0
Dr Shaun Frost
Address [4] 280087 0
CSIRO CCI WA | Preventative Health Flagship
Australian e-Health Research Centre
Private bag 5, Wembley WA 6913 Australia
Country [4] 280087 0
Australia
Other collaborator category [5] 280088 0
Individual
Name [5] 280088 0
Professor Rodney Dilley
Address [5] 280088 0
Ear Science Institute
1/1 Salvado Rd, Subiaco WA 6008
Country [5] 280088 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 300229 0
The University of Western Australia Human Ethics Office of Research Enterprise
Ethics committee address [1] 300229 0
M459, 35 Stirling Highway
Crawley 6009 WA
Ethics committee country [1] 300229 0
Australia
Date submitted for ethics approval [1] 300229 0
27/09/2015
Approval date [1] 300229 0
20/11/2015
Ethics approval number [1] 300229 0
RA/4/1/7916

Summary
Brief summary
The purpose of this research is to understand the molecular mechanisms of eye disease and to correlate this with variations in the speed of deterioration and disease manifestation between individuals.

Part I of this project is to monitor the retina once every 6 months through a series of detailed tests which allows us to determine if the disease has progressed over a 5 year period.

Part II of this project is to collect blood, urine saliva, skin and or eye tissues to study RNA, DNA and biomarkers in the blood and to create induced pluripotent stem cells from somatic cells.

Combining Part I and Part II of the study will facilitate discovery of new methods or biomarkers to better predict retinal disease progression and future treatment response.

Part I:
High resolution retinal imaging can provide information on microscopic changes within the living retina. Fundus autofluorescence, optical coherence tomography and adaptive optics imaging are new techniques that may enhance our ability to detect very subtle changes within the retina in patients with slowly progressive retinal degeneration. The aim of part I of the research is to find out if new methods of analysis can detect changes in these serial retinal images over a period of years which an experienced image grader is unable to identify or measure.

Part II:
Biomarkers in the blood will be analysed. This includes anti-retinal antibodies, RNA and DNA. Induced pluripotent stem (iPS) cells are stem cells generated in the laboratory, generally from skin cells that have been reprogrammed and induced to become pluripotent that is, they have the ability to form any cell type of the body. This characteristic makes iPS cells similar to embryonic stem cells with the potential to form any organ in the body. The aim of part II of the research is to generate iPSC from skin cells of people with retinal disease. This will allow the development of stem cell lines that will then be studied to further understand the mechanisms of disease. This can be correlated with specific genetic variants to determine how genetic mutations cause disease and vision impairment. Importantly, new knowledge into the molecular mechanisms of disease can help to identify potential targets for therapies for people with eye disease.

This research is based at the Lions Eye Institute incorporating the Centre for Ophthalmology & Visual Science, The University of Western Australia.
Trial website
https://www.lei.org.au/research/clinical-trials/
Trial related presentations / publications
N/A
Public notes

Contacts
Principal investigator
Name 82986 0
Dr Fred Chen
Address 82986 0
Lions Eye Institute
2 Verdun Street, Nedlands, WA 6009
Country 82986 0
Australia
Phone 82986 0
+61893810817
Fax 82986 0
Email 82986 0
[email protected]
Contact person for public queries
Name 82987 0
Fred Chen
Address 82987 0
Lions Eye Institute
2 Verdun Street, Nedlands, WA 6009
Country 82987 0
Australia
Phone 82987 0
+61893810817
Fax 82987 0
Email 82987 0
[email protected]
Contact person for scientific queries
Name 82988 0
Fred Chen
Address 82988 0
Lions Eye Institute
2 Verdun Street, Nedlands, WA 6009
Country 82988 0
Australia
Phone 82988 0
+61893810817
Fax 82988 0
Email 82988 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

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No documents have been uploaded by study researchers.

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