![]() If the committee determines enrollment will continue, three additional participants with RCD and three participants with ESCS will be enrolled. Safety Adverse Event Review Committee members unaffiliated with the study will review the data as a preliminary assessment of safety and efficacy and to determine whether enrollment should continue. Once all four participants have completed the 8-week visit, enrollment will be halted. The initial stage of the study will include two participants from each disease category. One eye of up to five participants with RCD with significant macular cystic changes and up to five participants with ESCS with significant macular cystic changes will receive topical IFN gamma-1b instilled as drops on the cornea. Design: This is a single-center, prospective, uncontrolled, unmasked pilot Phase I/II study of the safety, tolerability and possible efficacy of IFN gamma-1b in participants with RCD and ESCS and macular cystic changes. However, up to an additional two participants may be enrolled in order to obtain the five participants in each disease group to be included in the primary analysis if any participants withdraw from the study prior to receiving five days of treatment. Study Population: Up to five participants with RCD with significant macular cystic changes and up to five participants with ESCS with significant macular cystic changes will be enrolled to receive IFN gamma-1b administered topically in one eye. Possible disease-related pathophysiologic mechanisms will be explored using induced pluripotent stem cell (iPSC) protocols leading to iPSC-derived retinal pigment epithelium (RPE) and photoreceptor generation. The objective of this study is to evaluate the safety and potential efficacy of Interferon (IFN) gamma-1b administered topically for macular edema/retinal schisis cysts in RCD and ESCS. Acetazolamide (Diamox) and Dorzolamide (Trusopt) have been reported to have variable success in reducing these cystic changes but the effect is frequently inadequate. The reason for this association is not well understood. Macular cystic changes, often florid and usually resulting in a reduction of central acuity, are frequently associated with both diseases. Enhanced S-Cone syndrome (ESCS) is a rare autosomal recessive retinal disease with a developmental and a degenerative aspect. Objective: Rod-cone dystrophy (RCD) is a term applied to a number of genetically heterogenous diseases presenting with night vision abnormalities, visual field defects and reduced rod electroretinography responses. Study Primary Completion Date: June 1, 2018. ![]() Intervention Model: Single Group Assignment.– Participants will have a follow-up visit after 52 weeks.įull Title of Study: “Pilot Phase I/II Study of the Evaluation of Interferon Gamma-1b Administered Topically for Macular Edema/Intraretinal Schisis Cysts in Rod-Cone Dystrophy (RCD) and Enhanced S-Cone Syndrome (ESCS)” Study Type Electrodes will be attached outside of the eyes and eye function will be measured in the dark and the light. After 30 minutes, researchers will remove the eyepatches and put in numbing eyedrops and contact lenses. A small electrode will be taped to the forehead. Participants will sit in the dark wearing eyepatches. A dye injected into an arm vein will travel to the blood vessels in the eyes. – Eye exams, including eye dilation and tasks on computer screens. They may have: – Repeats of screening tests. – Participants will have 5 outpatient visits over 8 weeks, 2 of which are telephone assessments. – Participants will give themselves 4 study eyedrops 4 times daily for 2 weeks and keep a diary. – Participants will stay at NIH for 3 days and get the first eyedrops. Design: – Participants will be screened with medical history, physical exam, eye exam, and blood tests. Those with ESCS must have two mutations in the NR2E3 gene. Eligibility: – People at least 12 years old with RCD or ESCS. To see if the medicine can decrease retina fluid and help prevent vision loss. Objectives: – To see if interferon gamma-1b eyedrops are safe for people with RCD or ESCS. The medicine interferon gamma-1b may help people with these diseases. Overall Status: Terminated | Estimated Enrollment: 4 Overviewīackground: – People with rod-cone dystrophy (RCD) or enhanced S-cone syndrome (ESCS) have excess fluid under the retina of their eye. Phase: Phase 1/Phase 2 | Start Date: January 14, 2015 First Received: Janu| Last Updated: August 13, 2019
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