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2017-22: A Multicentre, Prospective, Longitudinal, Observational Natural History Study to Evaluate Disease Progression in Subjects with Usher Syndrome type 1B (USH1B)

Children and inhertable diseases
Outcome research
Investigator Initiated Research
Inclusion open since 12-12-2018

Drug development in non-syndromic and syndromic retinitis pigmentosa (RP) has proved to be challenging, also due to the difficulty in applying endpoints commonly used in ophthalmology clinical trials. For instance, best corrected visual acuity (BCVA), which is used as a registration endpoint in many ophthalmologic clinical trials,

may be not a viable endpoint for use in this slowly progressing disease.

In addition, due to the slow progression of RP and the large amount of variability inherent to visual function assessments, some assessments have insufficient sensitivity for quantifying clinically meaningful changes in photoreceptor loss in a time frame desirable for clinical trials.

This natural history study is being conducted to understand the progression of disease in USH1B patients as measured by a number of vision-related assessments. Disease progression will be evaluated as change over time in these measures, and associations between the endpoints will be examined.

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