PQ-110-004, is designed to evaluate if a mobility course using multiple light levels simulating real world conditions, can detect changes in vision in subjects with a phenotype representative of LCA Type 10 and therefore serves as an assessment tool…
ID
Source
Brief title
Condition
- Eye disorders congenital
- Congenital eye disorders (excl glaucoma)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
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
Secondary outcome
Not applicable
Background summary
Leber*s congenital amaurosis (LCA) is a severe inherited retinal degenerative
disease resulting in blindness, often in early childhood. In subjects with LCA
due to the p.Cys998X mutation in Centrosomal Protein of 290 kDa (CEP290),
visual symptoms are usually detectable before 1 year of age and further
deterioration over time has also been reported (den Hollander 2008, Yzer 2012).
Patients show severe vision disturbances from an early age and slow progressive
loss of remaining vision (Cideciyan 2007, Cideciyan 2011). There are currently
no approved therapies for the treatment of LCA due to the p.Cys998X mutation in
CEP290 (subsequently referred to as the CEP290 p.Cys998X mutation) and a large
unmet medical need exists.
Study objective
PQ-110-004, is designed to evaluate if a mobility course using multiple light
levels simulating real world conditions, can detect changes in vision in
subjects with a phenotype representative of LCA Type 10 and therefore serves as
an assessment tool to evaluate efficacy endpoints in clinical studies.
Study design
Study PQ-110-004 will evaluate the feasibility and inter-visit variability of
selected visual assessments in subjects with a phenotype representative of LCA
Type 10 for use as efficacy endpoints in clinical studies. No study drug or
placebo will be administered during this study; however, participating subjects
with LCA due to the CEP290 p.Cys998X mutation may be eligible to enroll in the
phase 2/3 clinical study PQ-110-003.
Participating sites should pre identify subjects from their database known to
have a clinical diagnosis: 1) LCA Type 10, 2) any other LCA subtype or 3) any
subtype of inherited retinal disease, and who have a phenotype representative
of LCA Type 10.
Each site should plan to enroll all eligible subjects expected to be suitable
for participation in a phase 2/3 clinical study PQ-110-003.
Subjects will attend a Screening Visit to determine if they meet the
eligibility criteria. Subjects meeting all eligibility criteria will be
scheduled to undergo a series of assessments during 2 consecutive days within 1
month of screening as outlined in the Schedule of Events. All assessments will
be performed in both eyes.
Intervention
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
Study burden and risks
The study duration is about 2 months and consists of 1 screening visit (may be
completed over multiple days) and 2 consecutive days (day 1 and day 2) with
tests
The tests being done in this study:
- visual acuity test
- ophthalmic examination
- mobility course
- FST
Risks: see section E9
Zernikedreef 9
Leiden 2333 CK
NL
Zernikedreef 9
Leiden 2333 CK
NL
Listed location countries
Age
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.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL68527.000.18 |