Interferon Gamma-1b Administered Topically for Macular Edema/Intraretinal Schisis Cysts in Rod-Cone Dystrophy (RCD) and Enhanced S-Cone Syndrome (ESCS)

Stopped early · Phase 1/Phase 2

Conditions studied: Inherited Ophthalmic Diseases, Inherited Retinal Degeneration

In brief

Background: \- People with rod-cone dystrophy (RCD) or enhanced S-cone syndrome (ESCS) have excess fluid under the retina of their eye. This can cause vision loss. The medicine interferon gamma-1b may help people with these diseases. Objectives: \- To see if interferon gamma-1b eyedrops are safe for people with RCD or ESCS. To see if the medicine can decrease retina fluid and help prevent vision loss. Eligibility: \- People at least 12 years old with RCD or ESCS. Those with ESCS must have two mutations in the NR2E3 gene. Design: * Participants will be screened with medical history, physical exam, eye exam, and blood tests. * Participants will stay at NIH for 3 days and get the first eyedrops. * Participants will give themselves 4 study eyedrops 4 times daily for 2 weeks and keep a diary. * Participants will have 5 outpatient visits over 8 weeks, 2 of which are telephone assessments. They may have: * Repeats of screening tests. * Questionnaires. * Small piece of skin removed. * Eye exams, including eye dilation and tasks on computer screens. * Fluorescein angiography. A dye injected into an arm vein will travel to the blood vessels in the eyes. A camera will take pictures. * Electroretinography. Participants will sit in the dark wearing eyepatches. A small electrode will be taped to the forehead. After 30 minutes, researchers will remove the eyepatches and put in numbing eyedrops and contact lenses. Participants will watch flashing lights. * Electrooculography. Electrodes will be attached outside of the eyes and eye function will be measured in the dark and the light. * Participants will have a follow-up visit after 52 weeks.

Key facts

Study ID

NCT02338973

Run by

National Eye Institute (NEI)

People needed

4

Starts

2015-01-14

Expected to finish

2018-07-26

Last updated by the study team

2019-08-13

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• A participant is not eligible if any of the following exclusion criteria are present.
• 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).
• Participant has had diagnosis or treatment of a malignancy (excluding non-melanoma skin cancer) within the previous five years.
• Participant has received investigational treatment in another clinical study related to an ocular condition in the last six months.
• Participant is pregnant, lactating, planning to become pregnant (or father a child) during the study follow-up period.
• Participant is allergic to fluorescein dye.
• 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
• The study eye must retain adequate fixation to allow for completion of protocol assessments.
• The study eye must have macular cystic changes (>275 microns and/or disruption of foveal contour on OCT).
• Study Eye Exclusion Criteria
• The study eye has lens, cornea, or other media opacities that preclude adequate visualization and testing of the retina.
• The study eye has undergone intraocular surgery within 6 months prior to enrollment.
• 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].
• 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.

Where it is running

• National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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