Study to evaluate the efficacy, safety, tolerability and systemic exposure of QR-110 subjects with Leber's Congenital Amourosis (LCA) Due to to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene.

Study to evaluate the efficacy, safety, tolerability and systemic exposure of QR-110 subjects with Leber's Congenital Amourosis (LCA) Due to to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene.

Study to evaluate the efficacy, safety, tolerability and systemic exposure of QR-110 subjects with Leber's Congenital Amourosis (LCA) Due to to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene.

Recruiting
18-90 years
All
Phase N/A
  • Simplified description (AI rewritten)
  • Technical description (Original)
The study drug is being developed by ProQR Therapeutics (the “Sponsor”). ProQR Therapeutics is the company that pays for and has responsibility for the study. This research study is for patients who have Leber’s Congenital Amaurosis type 10 with the p.Cys998X mutation in the CEP290 gene (called LCA10). What does my participation in the study involve? If you decide to take part in the study: You will be in the study for approximately 25 months. You will visit the study site approximately 18 times. Some of these visits will be planned on 2 days (or more if needed). You may need to stay overnight near to the study site. Your will receive a telephone call 1 week after each dosing visit to find out how you're doing.
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Study details
    LCA
    Leber's Congenital Amourosis

TX6452

19 February 2024

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