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.

  • STATUS
    Recruiting
  • participants needed
    40
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Updated on 19 February 2024

Summary

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.

Description

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.

FAQ


Details
Condition LCA,Leber's Congenital Amourosis
Age 18years - 90years
Clinical Study IdentifierTX6452
Last Modified on19 February 2024

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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