Ignite Creation Date:
2024-05-06 @ 3:37 AM
Last Modification Date:
2024-10-26 @ 11:36 AM
Study NCT ID:
NCT02338973
Status:
TERMINATED
Last Update Posted:
2019-08-13
First Post:
2015-01-14
Brief Title:
Interferon Gamma-1b Administered Topically for Macular EdemaIntraretinal Schisis Cysts in Rod-Cone Dystrophy RCD and Enhanced S-Cone Syndrome ESCS
Sponsor:
National Eye Institute NEI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Pilot Phase III Study of the Evaluation of Interferon Gamma-1b Administered Topically for Macular EdemaIntraretinal Schisis Cysts in Rod-Cone Dystrophy RCD and Enhanced S-Cone Syndrome ESCS
Status:
TERMINATED
Status Verified Date:
2018-07-26
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
- 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
Detailed Description:
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 Enhanced S-Cone syndrome ESCS is a rare autosomal recessive retinal disease with a developmental and a degenerative aspect Macular cystic changes often florid and usually resulting in a reduction of central acuity are frequently associated with both diseases The reason for this association is not well understood Acetazolamide Diamox and Dorzolamide Trusopt have been reported to have variable success in reducing these cystic changes but the effect is frequently inadequate The objective of this study is to evaluate the safety and potential efficacy of Interferon IFN gamma-1b administered topically for macular edemaretinal schisis cysts in RCD and ESCS 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
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 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
Design
This is a single-center prospective uncontrolled unmasked pilot Phase III study of the safety tolerability and possible efficacy of IFN gamma-1b in participants with RCD and ESCS and macular cystic changes 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 evidenced by optical coherence tomography OCT 275 microns central macular thickness andor disruption of foveal contour will receive topical IFN gamma-1b instilled as drops on the cornea The initial stage of the study will include two participants from each disease category Once all four participants have completed the 8-week visit enrollment will be halted 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 If the committee determines enrollment will continue three additional participants with RCD and three participants with ESCS will be enrolled The study will be completed once the final participant has received one year of follow-up
Outcome Measures
The primary outcome measure related to the safety and tolerability of IFN gamma-1b administered topically at the prescribed dosage for macular cystic changes in participants with RCD and ESCS will be assessed by the number and severity of adverse events related to the IP and the number of withdrawals at 52 weeks one year post-administration Additional safety of IFN gamma-1b administered topically in participants with RCD and ESCS will be determined from the assessment of retinal function ocular structure and occurrence of adverse events at all time points Secondary outcomes include changes in visual function including visual acuity and microperimetry and retinal imaging with OCT and fluorescein angiography
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 Enhanced S-Cone syndrome ESCS is a rare autosomal recessive retinal disease with a developmental and a degenerative aspect Macular cystic changes often florid and usually resulting in a reduction of central acuity are frequently associated with both diseases The reason for this association is not well understood Acetazolamide Diamox and Dorzolamide Trusopt have been reported to have variable success in reducing these cystic changes but the effect is frequently inadequate The objective of this study is to evaluate the safety and potential efficacy of Interferon IFN gamma-1b administered topically for macular edemaretinal schisis cysts in RCD and ESCS 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
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 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
Design
This is a single-center prospective uncontrolled unmasked pilot Phase III study of the safety tolerability and possible efficacy of IFN gamma-1b in participants with RCD and ESCS and macular cystic changes 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 evidenced by optical coherence tomography OCT 275 microns central macular thickness andor disruption of foveal contour will receive topical IFN gamma-1b instilled as drops on the cornea The initial stage of the study will include two participants from each disease category Once all four participants have completed the 8-week visit enrollment will be halted 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 If the committee determines enrollment will continue three additional participants with RCD and three participants with ESCS will be enrolled The study will be completed once the final participant has received one year of follow-up
Outcome Measures
The primary outcome measure related to the safety and tolerability of IFN gamma-1b administered topically at the prescribed dosage for macular cystic changes in participants with RCD and ESCS will be assessed by the number and severity of adverse events related to the IP and the number of withdrawals at 52 weeks one year post-administration Additional safety of IFN gamma-1b administered topically in participants with RCD and ESCS will be determined from the assessment of retinal function ocular structure and occurrence of adverse events at all time points Secondary outcomes include changes in visual function including visual acuity and microperimetry and retinal imaging with OCT and fluorescein angiography
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 15-EI-0052 | None | None | None |