Feasibility and Variability of Select Vision Assessments in LCA
Research type
Research Study
Full title
Study to Evaluate the Feasibility and Variability of Select Vision Assessments in Subjects with Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
IRAS ID
261967
Contact name
Michel Michaelides
Contact email
Sponsor organisation
ProQR Therapeutics
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
0 years, 2 months, 17 days
Research summary
Leber’s congenital amaurosis (LCA) is a severe inherited retinal degenerative disease resulting in blindness, often in early childhood. Patients show severe vision disturbances from an early age and slow progressive loss of remaining vision.
Currently there is no approved treatment for LCA10. Study PQ-110-004 will evaluate the feasibility and inter-visit variability of selected visual assessments in patients with LCA Type 10 for use as efficacy endpoints in clinical studies.
No study drug or placebo will be administered during this study; however, participating patients may be eligible to enrol in a Phase 2/3 clinical study of PQ-110-003.Patients will attend a screening visit to determine if they meet the eligibility criteria. Participants meeting all eligibility criteria will be scheduled to undergo a series of assessments during 2 consecutive days within 1 month of screening. All ophthalmology assessments will be performed in both eyes.
The following assessments will be performed in all participants:
• Patient Reported Outcome (PRO) of Patient Global Impressions of Severity (PGI-S), PRO of Patient Global Impressions of Change (PGI-C) performed at Screening and on both Day 1 and Day 2.
• Best Corrected Visual Activity (BCVA) performed at Screening and on both Day 1 and Day 2.
• Mobility course performed at Screening and on both Day 1 and Day 2.
• Ophthalmic Exam to include: Slit Lamp Biomicroscopy Examination, Intraocular Pressure and Diluted Fundus Examination.
• Full field stimulus testing (FST) performed at Screening and on both Day 1 and Day 2.
• Fundus Autofluorescence (FAF) performed at Screening and on Day 1.
• Spectral Domain Optical Coherence Tomography (SD-OCT) performed at Screening and on both Day 1 and Day 2.REC name
London - London Bridge Research Ethics Committee
REC reference
19/LO/1312
Date of REC Opinion
14 Aug 2019
REC opinion
Favourable Opinion