Study to Evaluate the Feasibility and Variability of Select Vision Assessments in Subjects with a Leber's Congenital Amaurosis (LCA) Type Phenotype

Completed

• Conditions: Leber's Congenital Amaurosis Type Phenotype; Leber's disease; 10015920; 10010463

• Registration Number: NL-OMON48307
• Lead Sponsor: ProQR Therapeutics

Brief Summary

Trial is onging in other countries

Detailed Description

Not available

Study Timeline

• Study Completion: 2020-12-02
• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 7

Inclusion Criteria

1. Persistence of detectable outer nuclear layer (ONL) in the area of the
macula in the opinion of the Investigator, as determined by OCT
2. Clear ocular media and adequate pupillary dilation to permit good quality
retinal imaging, as assessed by the Investigator
3. An adult (* 18 years) willing and able to provide informed consent for
participation OR a minor (6 to < 18 years) with a parent or legal guardian
willing and able to provide written permission for the subject*s participation
prior to performing any study related procedures and minor subjects able to
provide age appropriate assent for study participation
4. An adult willing to comply with the protocol, follow study instructions,
attend study visits as required and willing and able to complete all study
assessments, in the opinion of the Investigator OR a minor able to complete
all study assessments and comply with the protocol and has a parent or
caregiver willing and able to follow study instructions and attend study visits
with the subject as required, in the opinion of the Investigator
5. Adequate verbal communication as to allow assessment via mobility course, in
the opinion of the Investigator
6. Clinical diagnosis of: 1) LCA Type 10, 2) any other LCA subtype or 3) any
subtype of inherited retinal disease, which has a phenotype representative of
LCA Type 10, as assessed with concurrence of the Medical Monitor.

Exclusion Criteria

1. Any ocular and/or general disease or condition that could compromise
subject*s safety or interfere with assessment of efficacy and safety, as
determined by the Investigator
2. Any parent/guardian or subject who, in the opinion of the Investigator, may
not be compliant with the study procedures or schedule.

Study & Design

• Study Type: Interventional
• Study Design: Not specified