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

Study to Evaluate the Feasibility and Variability of Select Vision Assessments in Subjects with a Leber's Congenital Amaurosis (LCA) Type Phenotype - PQ-110-004

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON48307

Enrollment

Registered

2019-04-18

Start date

2019-07-23

Completion date

Unknown

Last updated

2024-04-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Leber's Congenital Amaurosis Type Phenotype Leber's disease

Interventions

Study subjects have to complete a mobility course with multiple light levels. In addition, they need to undergo the following tests: - visual acuity test including LLVA - ophthalmic examination - FST

Eligibility

Age

2 Years to 99 Years

Inclusion criteria

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

Exclusion criteria

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.

Design outcomes

Primary

Measure	Time frame
The primary objectives of this study are to evaluate: * The feasibility of the mobility course as an assessment tool to evaluate efficacy endpoints in clinical studies in subjects with a phenotype representative of LCA Type 10 * The feasibility of performing the following assessments in subjects with a phenotype representative of LCA Type 10: o Best corrected visual acuity (BCVA)including low luminance visual acuity [LLVA]) o Mobility course o Full field stimulus testing (FST) o Spectral Domain Optical Coherence Tomography (SD-OCT) o Fundus Autofluorescence (FAF) o Patient Reported Outcome (PRO) of Patient Global Impressions of Severity (PGI-S) o PRO of Patient Global Impressions of Change (PGI-C) * The inter-visit variability of the following assessments in subjects with a phenotype representative of LCA Type 10: o BCVA (including LLVA) o FST o Mobility course o PGI-S o PGI-C	—

Measure

Time frame

The primary objectives of this study are to evaluate: * The feasibility of the mobility course as an assessment tool to evaluate efficacy endpoints in clinical studies in subjects with a phenotype representative of LCA Type 10 * The feasibility of performing the following assessments in subjects with a phenotype representative of LCA Type 10: o Best corrected visual acuity (BCVA)including low luminance visual acuity [LLVA]) o Mobility course o Full field stimulus testing (FST) o Spectral Domain Optical Coherence Tomography (SD-OCT) o Fundus Autofluorescence (FAF) o Patient Reported Outcome (PRO) of Patient Global Impressions of Severity (PGI-S) o PRO of Patient Global Impressions of Change (PGI-C) * The inter-visit variability of the following assessments in subjects with a phenotype representative of LCA Type 10: o BCVA (including LLVA) o FST o Mobility course o PGI-S o PGI-C

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Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)