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Key inclusion & exclusion criteria
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- INCLUSION CRITERIA:
To be eligible, the following inclusion criteria must be met, where applicable.
1. Participant must be 12 years of age or older.
2. Participant (or legal guardian or legal representative) must understand and sign the
protocol informed consent.
3. Participant is willing to comply with the study procedures and is expected to be able
to return for all study visits.
4. Participant must carry a clinical diagnosis of RCD or ESCS.
5. ESCS participant must have molecular confirmation with two alleles for NR2E3 gene
mutations
6. Female participant of childbearing potential must not be pregnant or breast-feeding,
must have a negative pregnancy test at screening and must be willing to undergo
pregnancy tests at scheduled study visits.
7. Female participant of childbearing potential, and any male participant able to father
children must have (or have a partner who has) had a hysterectomy or vasectomy, be
completely abstinent from intercourse or must agree to practice two reliable methods
of contraception while taking the IP and six weeks after completion. Acceptable
methods of contraception include:
- Hormonal contraception (i.e., birth control pills, injected hormones, dermal
patch or vaginal ring);
- Intrauterine device;
- Barrier methods (diaphragm, condom) with spermicide; or
- Surgical sterilization (tubal ligation).
EXCLUSION CRITERIA:
A participant is not eligible if any of the following exclusion criteria are present.
1. Participant has a history of other ocular disease likely to contribute significantly
to visual disruption (e.g., optic neuropathy, glaucoma, uveitis, or other retinal
disease).
2. Participant has had diagnosis or treatment of a malignancy (excluding non-melanoma
skin cancer) within the previous five years.
3. Participant has received investigational treatment in another clinical study related
to an ocular condition in the last six months.
4. Participant is pregnant, lactating, planning to become pregnant (or father a child)
during the study follow-up period.
5. Participant is allergic to fluorescein dye.
6. Participant has a systemic condition that, in the opinion of the investigator, would
preclude participation in the study (e.g., multiple sclerosis (MS), as IFN gamma may
cause MS exacerbations).
Study Eye Eligibility Criteria
A participant must have at least one eye meeting all inclusion criteria and none of the
exclusion criteria listed below.
Study Eye Inclusion Criteria
1. The study eye must retain adequate fixation to allow for completion of protocol
assessments.
2. The study eye must have macular cystic changes (>275 microns and/or disruption of
foveal contour on OCT).
Study Eye Exclusion Criteria
1. The study eye has lens, cornea, or other media opacities that preclude adequate
visualization and testing of the retina.
2. The study eye has undergone intraocular surgery within 6 months prior to enrollment.
3. The study eye has a disease that may confound the outcome of the study [e.g.,
choroidal neovascularization (CNV) in the fovea or parafoveal area].
4. Participant is unwilling to discontinue wearing a contact lens in the study eye during
IP administration.
Study Eye Selection Criteria in Cases of Bilateral Disease
RCD and ESCS usually affect both eyes to a similar degree. In case both eyes of a
participant meet the study eye eligibility criteria, the following criteria will be used to
select the study eye:
- The eye with more intraretinal fluid will be selected as the study eye;
- If both eyes have similar levels of intraretinal fluid, the eye with worse visual
acuity will be selected as the study eye;
- If both eyes have the similar levels of intraretinal fluid and visual acuities, the
right eye will be selected as the study eye.
Age minimum:
12 Years
Age maximum:
N/A
Gender:
All
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Secondary Outcome(s)
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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 52
[Time Frame: Week 52]
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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 2
[Time Frame: Week 2]
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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 2
[Time Frame: Day 2]
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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 1
[Time Frame: Day 1]
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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 5
[Time Frame: Week 5]
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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 1
[Time Frame: Day 1]
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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 5
[Time Frame: Week 5]
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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 8
[Time Frame: Week 8]
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Change in Central Visual Field Sensitivity at Day 2 and Week 5 Compared to Baseline.
[Time Frame: Day 2 and Week 5]
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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 5
[Time Frame: Week 5]
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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 2
[Time Frame: Day 2]
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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 3
[Time Frame: Day 3]
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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 1
[Time Frame: Day 1]
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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 2
[Time Frame: Week 2]
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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 2
[Time Frame: Week 2]
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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 2
[Time Frame: Day 2]
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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 52
[Time Frame: Week 52]
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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 2
[Time Frame: Day 2]
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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 5
[Time Frame: Week 5]
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Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 52
[Time Frame: Week 52]
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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 8
[Time Frame: Week 8]
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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 2
[Time Frame: Week 2]
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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 3
[Time Frame: Day 3]
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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 52
[Time Frame: Week 52]
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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 3
[Time Frame: Day 3]
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Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 8
[Time Frame: Week 8]
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Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 3
[Time Frame: Day 3]
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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 8
[Time Frame: Week 8]
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Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 1
[Time Frame: Day 1]
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