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 years - 90 years
All
Phase
N/A
40 participants needed
1 Location
Brief description of study
You are invited to take part in a research study for with the investigational drug, QR-110 (referred to as “the study drug”) for Leber's Congenital Amourosis . Currently there is no approved treatment for LCA10. The study drug is designed to repair the problem that causes LCA10. This may stop or possibly reverse some effects of LCA10. This study is being done to find out if the study drug works and can be tolerated in patients with LCA10.
Detailed description of study
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.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: LCA,Leber's Congenital Amourosis
-
Age: 18 years - 90 years
-
Gender: All
Updated on
19 Feb 2024.
Study ID: 833469
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