Trials / Completed
CompletedNCT03396042
Natural History Study of CEP290-Related Retinal Degeneration
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 26 (actual)
- Sponsor
- Editas Medicine, Inc. · Industry
- Sex
- All
- Age
- 3 Years – 99 Years
- Healthy volunteers
- Not accepted
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.
Detailed 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.
Conditions
- Blindness
- Leber Congenital Amaurosis 10
- Vision Disorders
- Eye Diseases
- Eye Diseases, Hereditary
- Eye Disorders Congenital
- Retinal Disease
- Retinal Degeneration
Timeline
- Start date
- 2017-12-17
- Primary completion
- 2022-05-06
- Completion
- 2022-05-06
- First posted
- 2018-01-10
- Last updated
- 2022-05-19
Locations
7 sites across 4 countries: United States, France, Germany, Netherlands
Source: ClinicalTrials.gov record NCT03396042. Inclusion in this directory is not an endorsement.