12 and older, any sex, with Inherited Ophthalmic Diseases or Inherited Retinal Degeneration. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number and Severity of IP-related AEsPrimary· Study duration, up to 52 weeks
The number and severity of adverse events related to the investigation product (IP).
Severity: Mild
Group
Value
95% CI
Interferon Gamma-1b
3
Severity: Severe
Group
Value
95% CI
Interferon Gamma-1b
1
Number of Participants Who WithdrewPrimary· Study duration, up to 52 weeks
The number of participants who withdrew early.
Group
Value
95% CI
Interferon Gamma-1b
0
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 1Secondary· Day 1
Change in BCVA from baseline as compared to Day 1 by participant in both study and fellow eyes.
Participant 001
Group
Value
95% CI
BCVA at Baseline in Study Eye
77
BCVA at Baseline in Fellow Eye
80
BCVA at Day 1 in Study Eye
76
BCVA at Day 1 in Fellow Eye
79
Difference in BCVA at Day 1 From Baseline in Study Eye
-1
Difference in BCVA at Day 1 From Baseline in Fellow Eye
-1
Participant 002
Group
Value
95% CI
BCVA at Baseline in Study Eye
39
BCVA at Baseline in Fellow Eye
61
BCVA at Day 1 in Study Eye
32
BCVA at Day 1 in Fellow Eye
62
Difference in BCVA at Day 1 From Baseline in Study Eye
-7
Difference in BCVA at Day 1 From Baseline in Fellow Eye
1
Participant 003
Group
Value
95% CI
BCVA at Baseline in Study Eye
69
BCVA at Baseline in Fellow Eye
76
BCVA at Day 1 in Study Eye
74
BCVA at Day 1 in Fellow Eye
70
Difference in BCVA at Day 1 From Baseline in Study Eye
5
Difference in BCVA at Day 1 From Baseline in Fellow Eye
-6
Participant 004
Group
Value
95% CI
BCVA at Baseline in Study Eye
14
BCVA at Baseline in Fellow Eye
42
BCVA at Day 1 in Study Eye
18
BCVA at Day 1 in Fellow Eye
44
Difference in BCVA at Day 1 From Baseline in Study Eye
4
Difference in BCVA at Day 1 From Baseline in Fellow Eye
2
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 2Secondary· Day 2
Change in BCVA from baseline as compared to Day 2 by participant in both study and fellow eyes.
Participant 001
Group
Value
95% CI
BCVA at Baseline in Study Eye
77
BCVA at Baseline in Fellow Eye
80
BCVA at Day 2 in Study Eye
75
BCVA at Day 2 in Fellow Eye
81
Difference in BCVA at Day 2 From Baseline in Study Eye
-2
Difference in BCVA at Day 2 From Baseline in Fellow Eye
1
Participant 002
Group
Value
95% CI
BCVA at Baseline in Study Eye
39
BCVA at Baseline in Fellow Eye
61
BCVA at Day 2 in Study Eye
36
BCVA at Day 2 in Fellow Eye
58
Difference in BCVA at Day 2 From Baseline in Study Eye
-3
Difference in BCVA at Day 2 From Baseline in Fellow Eye
-3
Participant 003
Group
Value
95% CI
BCVA at Baseline in Study Eye
69
BCVA at Baseline in Fellow Eye
76
BCVA at Day 2 in Study Eye
75
BCVA at Day 2 in Fellow Eye
69
Difference in BCVA at Day 2 From Baseline in Study Eye
6
Difference in BCVA at Day 2 From Baseline in Fellow Eye
-7
Participant 004
Group
Value
95% CI
BCVA at Baseline in Study Eye
14
BCVA at Baseline in Fellow Eye
42
BCVA at Day 2 in Study Eye
21
BCVA at Day 2 in Fellow Eye
45
Difference in BCVA at Day 2 From Baseline in Study Eye
7
Difference in BCVA at Day 2 From Baseline in Fellow Eye
3
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 3Secondary· Day 3
Change in BCVA from baseline as compared to Day 3 by participant in both study and fellow eyes.
Participant 001
Group
Value
95% CI
BCVA at Baseline in Study Eye
77
BCVA at Baseline in Fellow Eye
80
BCVA at Day 3 in Study Eye
81
BCVA at Day 3 in Fellow Eye
78
Difference in BCVA at Day 3 From Baseline in Study Eye
4
Difference in BCVA at Day 3 From Baseline in Fellow Eye
-2
Participant 002
Group
Value
95% CI
BCVA at Baseline in Study Eye
39
BCVA at Baseline in Fellow Eye
61
BCVA at Day 3 in Study Eye
41
BCVA at Day 3 in Fellow Eye
61
Difference in BCVA at Day 3 From Baseline in Study Eye
2
Difference in BCVA at Day 3 From Baseline in Fellow Eye
0
Participant 003
Group
Value
95% CI
BCVA at Baseline in Study Eye
69
BCVA at Baseline in Fellow Eye
76
BCVA at Day 3 in Study Eye
74
BCVA at Day 3 in Fellow Eye
73
Difference in BCVA at Day 3 From Baseline in Study Eye
5
Difference in BCVA at Day 3 From Baseline in Fellow Eye
-3
Participant 004
Group
Value
95% CI
BCVA at Baseline in Study Eye
14
BCVA at Baseline in Fellow Eye
42
BCVA at Day 3 in Study Eye
23
BCVA at Day 3 in Fellow Eye
47
Difference in BCVA at Day 3 From Baseline in Study Eye
9
Difference in BCVA at Day 3 From Baseline in Fellow Eye
5
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 2Secondary· Week 2
Change in BCVA from baseline as compared to Week 2 by participant in both study and fellow eyes.
Participant 001
Group
Value
95% CI
BCVA at Baseline in Study Eye
77
BCVA at Baseline in Fellow Eye
80
BCVA at Week 2 in Study Eye
75
BCVA at Week 2 in Fellow Eye
78
Difference in BCVA at Week 2 From Baseline in Study Eye
-2
Difference in BCVA at Week 2 From Baseline in Fellow Eye
-2
Participant 002
Group
Value
95% CI
BCVA at Baseline in Study Eye
39
BCVA at Baseline in Fellow Eye
61
BCVA at Week 2 in Study Eye
0
BCVA at Week 2 in Fellow Eye
66
Difference in BCVA at Week 2 From Baseline in Study Eye
-39
Difference in BCVA at Week 2 From Baseline in Fellow Eye
5
Participant 003
Group
Value
95% CI
BCVA at Baseline in Study Eye
69
BCVA at Baseline in Fellow Eye
76
BCVA at Week 2 in Study Eye
66
BCVA at Week 2 in Fellow Eye
67
Difference in BCVA at Week 2 From Baseline in Study Eye
-3
Difference in BCVA at Week 2 From Baseline in Fellow Eye
-9
Participant 004
Group
Value
95% CI
BCVA at Baseline in Study Eye
14
BCVA at Baseline in Fellow Eye
42
BCVA at Week 2 in Study Eye
23
BCVA at Week 2 in Fellow Eye
42
Difference in BCVA at Week 2 From Baseline in Study Eye
9
Difference in BCVA at Week 2 From Baseline in Fellow Eye
0
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 5Secondary· Week 5
Change in BCVA from baseline as compared to Week 5 by participant in both study and fellow eyes.
Participant 001
Group
Value
95% CI
BCVA at Baseline in Study Eye
77
BCVA at Baseline in Fellow Eye
80
BCVA at Week 5 in Study Eye
71
BCVA at Week 5 in Fellow Eye
82
Difference in BCVA at Week 5 From Baseline in Study Eye
-6
Difference in BCVA at Week 5 From Baseline in Fellow Eye
2
Participant 002
Group
Value
95% CI
BCVA at Baseline in Study Eye
39
BCVA at Baseline in Fellow Eye
61
BCVA at Week 5 in Study Eye
0
BCVA at Week 5 in Fellow Eye
65
Difference in BCVA at Week 5 From Baseline in Study Eye
-39
Difference in BCVA at Week 5 From Baseline in Fellow Eye
4
Participant 003
Group
Value
95% CI
BCVA at Baseline in Study Eye
69
BCVA at Baseline in Fellow Eye
76
BCVA at Week 5 in Study Eye
72
BCVA at Week 5 in Fellow Eye
75
Difference in BCVA at Week 5 From Baseline in Study Eye
3
Difference in BCVA at Week 5 From Baseline in Fellow Eye
-1
Participant 004
Group
Value
95% CI
BCVA at Baseline in Study Eye
14
BCVA at Baseline in Fellow Eye
42
BCVA at Week 5 in Study Eye
26
BCVA at Week 5 in Fellow Eye
45
Difference in BCVA at Week 5 From Baseline in Study Eye
12
Difference in BCVA at Week 5 From Baseline in Fellow Eye
3
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 8Secondary· Week 8
Change in BCVA from baseline as compared to Week 8 by participant in both study and fellow eyes.
Participant 001
Group
Value
95% CI
BCVA at Baseline in Study Eye
77
BCVA at Baseline in Fellow Eye
80
BCVA at Week 8 in Study Eye
74
BCVA at Week 8 in Fellow Eye
80
Difference in BCVA at Week 8 From Baseline in Study Eye
-3
Difference in BCVA at Week 8 From Baseline in Fellow Eye
0
Participant 002
Group
Value
95% CI
BCVA at Baseline in Study Eye
39
BCVA at Baseline in Fellow Eye
61
BCVA at Week 8 in Study Eye
43
BCVA at Week 8 in Fellow Eye
65
Difference in BCVA at Week 8 From Baseline in Study Eye
4
Difference in BCVA at Week 8 From Baseline in Fellow Eye
4
Participant 003
Group
Value
95% CI
BCVA at Baseline in Study Eye
69
BCVA at Baseline in Fellow Eye
76
BCVA at Week 8 in Study Eye
72
BCVA at Week 8 in Fellow Eye
70
Difference in BCVA at Week 8 From Baseline in Study Eye
3
Difference in BCVA at Week 8 From Baseline in Fellow Eye
-6
Participant 004
Group
Value
95% CI
BCVA at Baseline in Study Eye
14
BCVA at Baseline in Fellow Eye
42
BCVA at Week 8 in Study Eye
24
BCVA at Week 8 in Fellow Eye
47
Difference in BCVA at Week 8 From Baseline in Study Eye
10
Difference in BCVA at Week 8 From Baseline in Fellow Eye
5
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 52Secondary· Week 52
Change in BCVA from baseline as compared to Week 52 by participant in both study and fellow eyes.
Participant 001
Group
Value
95% CI
BCVA at Baseline in Study Eye
77
BCVA at Baseline in Fellow Eye
80
BCVA at Week 52 in Study Eye
79
BCVA at Week 52 in Fellow Eye
78
Difference in BCVA at Week 52 From Baseline in Study Eye
2
Difference in BCVA at Week 52 From Baseline in Fellow Eye
-2
Participant 002
Group
Value
95% CI
BCVA at Baseline in Study Eye
39
BCVA at Baseline in Fellow Eye
61
BCVA at Week 52 in Study Eye
47
BCVA at Week 52 in Fellow Eye
65
Difference in BCVA at Week 52 From Baseline in Study Eye
8
Difference in BCVA at Week 52 From Baseline in Fellow Eye
4
Participant 003
Group
Value
95% CI
BCVA at Baseline in Study Eye
69
BCVA at Baseline in Fellow Eye
76
BCVA at Week 52 in Study Eye
65
BCVA at Week 52 in Fellow Eye
65
Difference in BCVA at Week 52 From Baseline in Study Eye
-4
Difference in BCVA at Week 52 From Baseline in Fellow Eye
-11
Participant 004
Group
Value
95% CI
BCVA at Baseline in Study Eye
14
BCVA at Baseline in Fellow Eye
42
BCVA at Week 52 in Study Eye
20
BCVA at Week 52 in Fellow Eye
44
Difference in BCVA at Week 52 From Baseline in Study Eye
6
Difference in BCVA at Week 52 From Baseline in Fellow Eye
2
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 1Secondary· Day 1
Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Day 1 by participant in both study and fellow eyes.
Participant 001
Group
Value
95% CI
Subretinal Fluid Volume at Baseline in Study Eye
7.25
Subretinal Fluid Volume at Baseline in Fellow Eye
7.1
Subretinal Fluid Volume at Day 1 in Study Eye
7.35
Subretinal Fluid Volume at Day1 in Fellow Eye
7.35
Difference at Day 1 From Baseline in Study Eye
0.1
Difference at Day 1 From Baseline in Fellow Eye
0.25
Participant 002
Group
Value
95% CI
Subretinal Fluid Volume at Baseline in Study Eye
14.15
Subretinal Fluid Volume at Baseline in Fellow Eye
11.2
Subretinal Fluid Volume at Day 1 in Study Eye
13.95
Subretinal Fluid Volume at Day1 in Fellow Eye
11.25
Difference at Day 1 From Baseline in Study Eye
-0.2
Difference at Day 1 From Baseline in Fellow Eye
0.05
Participant 003
Group
Value
95% CI
Subretinal Fluid Volume at Baseline in Study Eye
16.1
Subretinal Fluid Volume at Baseline in Fellow Eye
18.55
Subretinal Fluid Volume at Day 1 in Study Eye
15.15
Subretinal Fluid Volume at Day1 in Fellow Eye
17.2
Difference at Day 1 From Baseline in Study Eye
-0.95
Difference at Day 1 From Baseline in Fellow Eye
-1.35
Participant 004
Group
Value
95% CI
Subretinal Fluid Volume at Baseline in Study Eye
36.3
Subretinal Fluid Volume at Baseline in Fellow Eye
9.25
Subretinal Fluid Volume at Day 1 in Study Eye
37.7
Subretinal Fluid Volume at Day1 in Fellow Eye
9.2
Difference at Day 1 From Baseline in Study Eye
1.4
Difference at Day 1 From Baseline in Fellow Eye
-0.05
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 2Secondary· Day 2
Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Day 2 by participant in both study and fellow eyes.
Participant 001
Group
Value
95% CI
Subretinal Fluid Volume at Baseline in Study Eye
7.25
Subretinal Fluid Volume at Baseline in Fellow Eye
7.1
Subretinal Fluid Volume at Day 2 in Study Eye
7.1
Subretinal Fluid Volume at Day 2 in Fellow Eye
7
Difference at Day 2 From Baseline in Study Eye
-0.15
Difference at Day 2 From Baseline in Fellow Eye
-0.1
Participant 002
Group
Value
95% CI
Subretinal Fluid Volume at Baseline in Study Eye
14.15
Subretinal Fluid Volume at Baseline in Fellow Eye
11.2
Subretinal Fluid Volume at Day 2 in Study Eye
14.1
Subretinal Fluid Volume at Day 2 in Fellow Eye
10.9
Difference at Day 2 From Baseline in Study Eye
-0.05
Difference at Day 2 From Baseline in Fellow Eye
-0.3
Participant 003
Group
Value
95% CI
Subretinal Fluid Volume at Baseline in Study Eye
16.1
Subretinal Fluid Volume at Baseline in Fellow Eye
18.55
Subretinal Fluid Volume at Day 2 in Study Eye
15.15
Subretinal Fluid Volume at Day 2 in Fellow Eye
15.55
Difference at Day 2 From Baseline in Study Eye
-0.95
Difference at Day 2 From Baseline in Fellow Eye
-3
Participant 004
Group
Value
95% CI
Subretinal Fluid Volume at Baseline in Study Eye
36.3
Subretinal Fluid Volume at Baseline in Fellow Eye
9.25
Subretinal Fluid Volume at Day 2 in Study Eye
37.3
Subretinal Fluid Volume at Day 2 in Fellow Eye
9.05
Difference at Day 2 From Baseline in Study Eye
1
Difference at Day 2 From Baseline in Fellow Eye
-0.2
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 3Secondary· Day 3
Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Day 3 by participant in both study and fellow eyes.
Participant 001
Group
Value
95% CI
Subretinal Fluid Volume at Baseline in Study Eye
7.25
Subretinal Fluid Volume at Baseline in Fellow Eye
7.1
Subretinal Fluid Volume at Day 3 in Study Eye
7.5
Subretinal Fluid Volume at Day 3 in Fellow Eye
7.45
Difference at Day 3 From Baseline in Study Eye
0.25
Difference at Day 3 From Baseline in Fellow Eye
0.35
Participant 002
Group
Value
95% CI
Subretinal Fluid Volume at Baseline in Study Eye
14.15
Subretinal Fluid Volume at Baseline in Fellow Eye
11.2
Subretinal Fluid Volume at Day 3 in Study Eye
13.75
Subretinal Fluid Volume at Day 3 in Fellow Eye
11.25
Difference at Day 3 From Baseline in Study Eye
-0.4
Difference at Day 3 From Baseline in Fellow Eye
0.05
Participant 003
Group
Value
95% CI
Subretinal Fluid Volume at Baseline in Study Eye
16.1
Subretinal Fluid Volume at Baseline in Fellow Eye
18.55
Subretinal Fluid Volume at Day 3 in Study Eye
15.3
Subretinal Fluid Volume at Day 3 in Fellow Eye
17
Difference at Day 3 From Baseline in Study Eye
-0.8
Difference at Day 3 From Baseline in Fellow Eye
-1.55
Participant 004
Group
Value
95% CI
Subretinal Fluid Volume at Baseline in Study Eye
36.3
Subretinal Fluid Volume at Baseline in Fellow Eye
9.25
Subretinal Fluid Volume at Day 3 in Study Eye
35
Subretinal Fluid Volume at Day 3 in Fellow Eye
9.1
Difference at Day 3 From Baseline in Study Eye
-1.3
Difference at Day 3 From Baseline in Fellow Eye
-0.15
Adverse events — posted to ClinicalTrials.gov
Time frame: Up to 52 Weeks.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
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.
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
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Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Eye Institute (NEI)
Last refreshed: 13 August 2019
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02338973.