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Clinical Trial Summary

A prospective natural history study with systematic assessments and uniform follow-up to provide a high-quality dataset for assisting in the design of future clinical treatment trials involving patients with CEP290-related retinal degeneration caused by the common intron 26 mutation.


Clinical Trial Description

The purpose of the study is to describe the natural history of CEP290-related retinal degeneration caused by a compound heterozygous or homozygous intron 26 c.2991+1655A>G mutation and to better understand the best assessments for evaluation of patients with this condition in a future interventional trial. Patients meeting the entry criteria will be enrolled in the study. Visits will occur at Screening, Baseline, and Months 3, 6, and 12, for a total duration of 1 year. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03396042
Study type Observational
Source Editas Medicine, Inc.
Contact
Status Completed
Phase
Start date December 17, 2017
Completion date May 6, 2022

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