Trial Outcomes & Findings for Study of Brimonidine Tartrate Nanoemulsion Eye Drops in Patients With Ocular Graft-vs-Host Disease (NCT NCT03591874)

NCT ID: NCT03591874

Last Updated: 2022-07-06

Results Overview

The VBR consists of a set of ten images illustrating different degrees of ocular redness, ranging from normal to severe, and each image is assigned a value from 10 (least redness) to 100 (most redness). The bulbar conjunctival injection of the participant's eye (nasal and temporal) was examined via slit-lamp examination and compared to the reference images in the VBR and graded accordingly.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

59 participants

Primary outcome timeframe

Baseline, Day 84

Results posted on

2022-07-06

Participant Flow

Participant milestones

Participant milestones
Measure
OCU-300
One drop of Brimonidine Tartrate Nanoemulsion 0.18% administered in each eye, two times per day (BID)
Placebo
One drop of ophthalmic buffered saline administered in each eye BID
Overall Study
STARTED
40
19
Overall Study
Received at Least 1 Study Treatment
29
16
Overall Study
COMPLETED
21
12
Overall Study
NOT COMPLETED
19
7

Reasons for withdrawal

Reasons for withdrawal
Measure
OCU-300
One drop of Brimonidine Tartrate Nanoemulsion 0.18% administered in each eye, two times per day (BID)
Placebo
One drop of ophthalmic buffered saline administered in each eye BID
Overall Study
Adverse Event
4
0
Overall Study
Withdrawal by Subject
2
1
Overall Study
Investigator Withdrawal of Subject
1
0
Overall Study
On Study at Time of Study Termination
12
6

Baseline Characteristics

Study of Brimonidine Tartrate Nanoemulsion Eye Drops in Patients With Ocular Graft-vs-Host Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
OCU-300
n=29 Participants
One drop of Brimonidine Tartrate Nanoemulsion 0.18% administered in each eye BID
Placebo
n=16 Participants
One drop of ophthalmic buffered saline administered in each eye BID
Total
n=45 Participants
Total of all reporting groups
Age, Continuous
54.5 years
STANDARD_DEVIATION 13.91 • n=5 Participants
62.4 years
STANDARD_DEVIATION 7.74 • n=7 Participants
57.3 years
STANDARD_DEVIATION 12.57 • n=5 Participants
Sex: Female, Male
Female
8 Participants
n=5 Participants
6 Participants
n=7 Participants
14 Participants
n=5 Participants
Sex: Female, Male
Male
21 Participants
n=5 Participants
10 Participants
n=7 Participants
31 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
29 Participants
n=5 Participants
16 Participants
n=7 Participants
45 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
1 Participants
n=7 Participants
1 Participants
n=5 Participants
Race (NIH/OMB)
White
28 Participants
n=5 Participants
15 Participants
n=7 Participants
43 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Day 84

Population: The Intention-To-Treat (ITT) set included all randomized participants. Here, the overall number of participants analyzed are those evaluable for this Outcome Measure and the Number Analyzed per Row is the number of participants with evaluable data at each time point

The VBR consists of a set of ten images illustrating different degrees of ocular redness, ranging from normal to severe, and each image is assigned a value from 10 (least redness) to 100 (most redness). The bulbar conjunctival injection of the participant's eye (nasal and temporal) was examined via slit-lamp examination and compared to the reference images in the VBR and graded accordingly.

Outcome measures

Outcome measures
Measure
OCU-300
n=28 Participants
One drop of Brimonidine Tartrate Nanoemulsion 0.18% administered in each eye BID
Placebo
n=13 Participants
One drop of ophthalmic buffered saline administered in each eye BID
Change From Baseline to Day 84 in Validated Bulbar Redness (VBR) Score
Baseline
52.23 score on a scale
Standard Deviation 9.238
51.92 score on a scale
Standard Deviation 11.821
Change From Baseline to Day 84 in Validated Bulbar Redness (VBR) Score
Day 84
34.88 score on a scale
Standard Deviation 14.652
37.71 score on a scale
Standard Deviation 19.378
Change From Baseline to Day 84 in Validated Bulbar Redness (VBR) Score
Change from Baseline to Day 84
-17.62 score on a scale
Standard Deviation 14.086
-13.96 score on a scale
Standard Deviation 14.161

PRIMARY outcome

Timeframe: Baseline, Day 84

Population: The ITT set included all randomized participants. Here, the overall number of participants analyzed are those evaluable for this Outcome Measure and the Number Analyzed per Row is the number of participants with evaluable data at each time point

Participants were asked to rate their worst ocular pain/discomfort in the preceding 24 hours using a 10-point scale ranging from "None" (score=0) to "Unbearable/Excruciating" (score=10).

Outcome measures

Outcome measures
Measure
OCU-300
n=28 Participants
One drop of Brimonidine Tartrate Nanoemulsion 0.18% administered in each eye BID
Placebo
n=13 Participants
One drop of ophthalmic buffered saline administered in each eye BID
Change From Baseline to Day 84 in Ocular Discomfort Visual Analog Scale (VAS) Score
Baseline
6.2 score on a scale
Standard Deviation 1.77
6.5 score on a scale
Standard Deviation 1.85
Change From Baseline to Day 84 in Ocular Discomfort Visual Analog Scale (VAS) Score
Day 84
4.8 score on a scale
Standard Deviation 2.86
4.1 score on a scale
Standard Deviation 3.67
Change From Baseline to Day 84 in Ocular Discomfort Visual Analog Scale (VAS) Score
Change from Baseline to Day 84
-1.8 score on a scale
Standard Deviation 3.33
-2.6 score on a scale
Standard Deviation 2.69

SECONDARY outcome

Timeframe: Baseline, Day 84

Population: The ITT set included all randomized participants. Here, the overall number of participants analyzed are those evaluable for this Outcome Measure and the Number Analyzed per Row is the number of participants with evaluable data at each time point

The SANDE questionnaire is a short VAS assessment that quantifies both severity and frequency of current dry eye symptoms. The SANDE is comprised of two questions, and each question employs a 100-mm horizontal linear VAS. The measurement of symptom frequency ranges from "rarely" to "all of the time", and the symptom severity from "very mild" to "very severe". Data collected from the SANDE questionnaire was calculated by multiplying the frequency score by the severity score and obtaining the square root. The result is the Overall SANDE score which ranges from 0 to 100, with 100 being the maximal amount of dry eye symptoms and 0 being the minimal amount of dry eye symptoms.

Outcome measures

Outcome measures
Measure
OCU-300
n=28 Participants
One drop of Brimonidine Tartrate Nanoemulsion 0.18% administered in each eye BID
Placebo
n=13 Participants
One drop of ophthalmic buffered saline administered in each eye BID
Change From Baseline to Day 84 in Symptom Assessment iN Dry Eye (SANDE) Questionnaire Scores
Baseline
74.4 score on a scale
Standard Deviation 18.97
69.1 score on a scale
Standard Deviation 21.90
Change From Baseline to Day 84 in Symptom Assessment iN Dry Eye (SANDE) Questionnaire Scores
Day 84
50.5 score on a scale
Standard Deviation 24.05
44.1 score on a scale
Standard Deviation 36.43
Change From Baseline to Day 84 in Symptom Assessment iN Dry Eye (SANDE) Questionnaire Scores
Change from Baseline to Day 84
-26.5 score on a scale
Standard Deviation 22.24
-26.6 score on a scale
Standard Deviation 27.50

Adverse Events

OCU-300

Serious events: 2 serious events
Other events: 1 other events
Deaths: 0 deaths

Placebo

Serious events: 1 serious events
Other events: 4 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
OCU-300
n=29 participants at risk
One drop of Brimonidine Tartrate Nanoemulsion 0.18% administered in each eye BID
Placebo
n=16 participants at risk
One drop of ophthalmic buffered saline administered in each eye BID
Infections and infestations
Gastrointestinal infection
0.00%
0/29 • Day 1 (post-dose) up to 84 Days
6.2%
1/16 • Day 1 (post-dose) up to 84 Days
Respiratory, thoracic and mediastinal disorders
Dyspnoea
3.4%
1/29 • Day 1 (post-dose) up to 84 Days
0.00%
0/16 • Day 1 (post-dose) up to 84 Days
Respiratory, thoracic and mediastinal disorders
Hypoxia
3.4%
1/29 • Day 1 (post-dose) up to 84 Days
0.00%
0/16 • Day 1 (post-dose) up to 84 Days
Cardiac disorders
Coronary artery disease
3.4%
1/29 • Day 1 (post-dose) up to 84 Days
0.00%
0/16 • Day 1 (post-dose) up to 84 Days
Injury, poisoning and procedural complications
Fall
3.4%
1/29 • Day 1 (post-dose) up to 84 Days
0.00%
0/16 • Day 1 (post-dose) up to 84 Days
Infections and infestations
Pneumonia
0.00%
0/29 • Day 1 (post-dose) up to 84 Days
6.2%
1/16 • Day 1 (post-dose) up to 84 Days

Other adverse events

Other adverse events
Measure
OCU-300
n=29 participants at risk
One drop of Brimonidine Tartrate Nanoemulsion 0.18% administered in each eye BID
Placebo
n=16 participants at risk
One drop of ophthalmic buffered saline administered in each eye BID
Infections and infestations
Influenza
3.4%
1/29 • Day 1 (post-dose) up to 84 Days
6.2%
1/16 • Day 1 (post-dose) up to 84 Days
Infections and infestations
Pneumonia
0.00%
0/29 • Day 1 (post-dose) up to 84 Days
6.2%
1/16 • Day 1 (post-dose) up to 84 Days
Respiratory, thoracic and mediastinal disorders
Cough
0.00%
0/29 • Day 1 (post-dose) up to 84 Days
6.2%
1/16 • Day 1 (post-dose) up to 84 Days
Gastrointestinal disorders
Dry mouth
0.00%
0/29 • Day 1 (post-dose) up to 84 Days
6.2%
1/16 • Day 1 (post-dose) up to 84 Days

Additional Information

Vijay Tammara, VP, Strategic Regulatory Operations

Ocugen

Phone: 484-328-4751

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place