Core Outcome Measures in Effectiveness Trials

Evaluation of outcome measures for use in clinical trials involving subjects with Nystagmus

General Information

Nystagmus is a disorder characterised by abnormal and uncontrolled eye movements. It can occur as a congenital disorder, seemingly isolated or as part of various diseases affecting vision or neurological development or in metabolic disease. It can also occur secondary to a variety of acquired disorders. It is well known that nystagmus affects many parts of visual function beyond a simple reading chart (logMAR or Snellen chart), in a well-lit room without time restriction. Many small clinical trials for a variety of treatments, including surgery and medicines have been published but there is a striking lack of consistency regards the chosen outcome measures. These have included questionnaires, eye tracking models, visual acuity alone and head posture measurements. It is also noted that there is a consensus with regard for the need for outcome measures suitable for children as interventions before the end of visual development (approximately age 8) are most likely to be efficacious due to the phenomenon of amblyopia. This has hampered the robust assessment of a variety of potential therapeutic interventions. In our planned study, we will interrogate the intra and inter-subject variability in a variety of putative outcome measures in children over time in order to evaluate the most suitable and robust outcome measures for future trials.

Jay Self (PI): Associate Professor and Consultant Ophthalmologist, University of Southampton

Helena Lee (Collaborator): Associate Professor and Consultant Ophthalmologist, University of Southampton

Maria Theodorou (Co-PI): Consultant Ophthalmologist, Moorfields Eye Hospital London

Further Study Information

Current Stage:
June 2019 - June 2020
Funding source(s):
Funding from the Gift of Sight and Nystagmus Network Charities. Application in preparation to the NIHR.

Health Area

Disease Category
Eyes & vision

Disease Name

Target Population

Age Range
3 - 8


Nature / type of Intervention


Consensus conference
Consensus meeting
Focus group(s)
Literature review
Semi structured discussion

As above. Consensus through PPI meetings, national conferences, nystagmus care pathway development meetings and literature.

Stakeholders Involved

Clinical experts
Conference participants
Consumers (patients)
Patient/ support group representatives

Study Type

COS for clinical trials or clinical research

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