Search clinical trials for: p.Cys998X

Total 6 results

ProQR Therapeutics

Recruiting

An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. (BRIGHTEN)

Eye Diseases | Neurologic Manifestations | Retinal Degeneration | Retinal Dystrophies | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

Belgium, Brazil, Canada, Germany, Italy, Netherlands, United Kingdom
ProQR Therapeutics

Terminated

Extension Study to Study PQ-110-001 (NCT03140969) (INSIGHT)

Eye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

United States, Belgium
ProQR Therapeutics

Completed

Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Leber's Congenital Amaurosis

United States, Belgium
Editas Medicine, Inc.

Active, not recruiting

Single Ascending Dose Study in Participants With LCA10

Retinal Degeneration | Eye Diseases, Hereditary | Vision Disorders | Leber Congenital Amaurosis 10 | Inherited Retinal Dystrophies | Retinal Disease | Eye Disorders Congenital

United States
Editas Medicine, Inc.

Completed

Natural History Study of CEP290-Related Retinal Degeneration

Eye Diseases | Retinal Degeneration | Eye Diseases, Hereditary | Vision Disorders | Blindness | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

United States, Netherlands, France, Germany
ProQR Therapeutics

Active, not recruiting

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) (ILLUMINATE)

Eye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

United States, Belgium, Brazil, Canada, France, Germany, Italy, Netherlands, United Kingdom

Search clinical trials for: p.Cys998X

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