Trial Outcomes & Findings for rhDNase Eye Drops in Patients With Ocular Graft-Vs.-Host Disease (NCT NCT02702518)
NCT ID: NCT02702518
Last Updated: 2021-09-05
Results Overview
Corneal staining score as measured by dye staining using National Eye Institute (NEI) grading scale. Dye was applied to each eye and a slit lamp was used to observe corneal staining. NEI scale relies on a chart that divides the cornea into 5 sections and assigns a value from 0 (absent) to 3 (severe) to each section, based on the density of punctate staining, final staining score being the sum of individual section scores with a range of 0 (minimum) -15 (maximum) points. Complete corneal staining clearance with dye defined as a score of 0 indicating the best outcome.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
58 participants
Primary outcome timeframe
Between baseline and at 8 weeks of treatment
Results posted on
2021-09-05
Participant Flow
Participant milestones
| Measure |
rhDNase I
rhDNase I 0.1% eye drops 4 times a day for 8 weeks
rhDNase I: rhDNase I, 0.1% eye drops 4 times a day for 8 weeks
|
Vehicle
Drug vehicle eye drops 4 times a day for 8 weeks
Vehicle: Drug vehicle 4 times a day for 8 weeks
|
|---|---|---|
|
Overall Study
STARTED
|
29
|
29
|
|
Overall Study
COMPLETED
|
26
|
25
|
|
Overall Study
NOT COMPLETED
|
3
|
4
|
Reasons for withdrawal
| Measure |
rhDNase I
rhDNase I 0.1% eye drops 4 times a day for 8 weeks
rhDNase I: rhDNase I, 0.1% eye drops 4 times a day for 8 weeks
|
Vehicle
Drug vehicle eye drops 4 times a day for 8 weeks
Vehicle: Drug vehicle 4 times a day for 8 weeks
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
3
|
4
|
Baseline Characteristics
These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
Baseline characteristics by cohort
| Measure |
rhDNase I
n=29 Participants
rhDNase I 0.1% eye drops 4 times a day for 8 weeks
rhDNase I: rhDNase I, 0.1% eye drops 4 times a day for 8 weeks
|
Vehicle
n=29 Participants
Drug vehicle eye drops 4 times a day for 8 weeks
Vehicle: Drug vehicle 4 times a day for 8 weeks
|
Total
n=58 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
54 years
n=29 Participants
|
52 years
n=29 Participants
|
53 years
n=58 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=29 Participants
|
9 Participants
n=29 Participants
|
20 Participants
n=58 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=29 Participants
|
20 Participants
n=29 Participants
|
38 Participants
n=58 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=29 Participants
|
4 Participants
n=29 Participants
|
9 Participants
n=58 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
24 Participants
n=29 Participants
|
25 Participants
n=29 Participants
|
49 Participants
n=58 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=29 Participants
|
0 Participants
n=29 Participants
|
0 Participants
n=58 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=29 Participants
|
0 Participants
n=29 Participants
|
0 Participants
n=58 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=29 Participants
|
3 Participants
n=29 Participants
|
5 Participants
n=58 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=29 Participants
|
0 Participants
n=29 Participants
|
0 Participants
n=58 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=29 Participants
|
3 Participants
n=29 Participants
|
6 Participants
n=58 Participants
|
|
Race (NIH/OMB)
White
|
24 Participants
n=29 Participants
|
23 Participants
n=29 Participants
|
47 Participants
n=58 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=29 Participants
|
0 Participants
n=29 Participants
|
0 Participants
n=58 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=29 Participants
|
0 Participants
n=29 Participants
|
0 Participants
n=58 Participants
|
|
Region of Enrollment
United States
|
29 participants
n=29 Participants
|
29 participants
n=29 Participants
|
58 participants
n=58 Participants
|
|
Indication (%)
Leukemia
|
20 Participants
n=29 Participants
|
21 Participants
n=29 Participants
|
41 Participants
n=58 Participants
|
|
Indication (%)
Lymphoma
|
3 Participants
n=29 Participants
|
1 Participants
n=29 Participants
|
4 Participants
n=58 Participants
|
|
Indication (%)
Multiple myeloma
|
4 Participants
n=29 Participants
|
1 Participants
n=29 Participants
|
5 Participants
n=58 Participants
|
|
Indication (%)
Myeloproliferative disorders
|
1 Participants
n=29 Participants
|
1 Participants
n=29 Participants
|
2 Participants
n=58 Participants
|
|
Indication (%)
Other
|
1 Participants
n=29 Participants
|
5 Participants
n=29 Participants
|
6 Participants
n=58 Participants
|
|
BMT date (years)
|
5.2 years
n=29 Participants
|
2.2 years
n=29 Participants
|
2.8 years
n=58 Participants
|
|
Heart rate
|
84 Beats per minute (BPM)
n=29 Participants
|
81 Beats per minute (BPM)
n=29 Participants
|
83.5 Beats per minute (BPM)
n=58 Participants
|
|
Temperature
|
97.8 degrees Fahrenheit
n=29 Participants
|
97.6 degrees Fahrenheit
n=29 Participants
|
97.7 degrees Fahrenheit
n=58 Participants
|
|
Systemic GVHD
Skin
|
13 participants
n=20 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
14 participants
n=21 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
27 participants
n=41 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
|
Systemic GVHD
GI
|
3 participants
n=20 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
3 participants
n=21 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
6 participants
n=41 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
|
Systemic GVHD
Mouth
|
12 participants
n=20 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
12 participants
n=21 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
24 participants
n=41 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
|
Systemic GVHD
Lung
|
3 participants
n=20 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
2 participants
n=21 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
5 participants
n=41 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
|
Systemic GVHD
Joint
|
1 participants
n=20 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
0 participants
n=21 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
1 participants
n=41 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
|
Systemic GVHD
Genitalia
|
3 participants
n=20 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
1 participants
n=21 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
4 participants
n=41 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
|
Systemic GVHD
Other
|
2 participants
n=20 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
2 participants
n=21 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
4 participants
n=41 Participants • These counts only correspond to subjects with systemic GVHD. Some subjects had more than one presentation of systemic GVHD.
|
PRIMARY outcome
Timeframe: Between baseline and at 8 weeks of treatmentCorneal staining score as measured by dye staining using National Eye Institute (NEI) grading scale. Dye was applied to each eye and a slit lamp was used to observe corneal staining. NEI scale relies on a chart that divides the cornea into 5 sections and assigns a value from 0 (absent) to 3 (severe) to each section, based on the density of punctate staining, final staining score being the sum of individual section scores with a range of 0 (minimum) -15 (maximum) points. Complete corneal staining clearance with dye defined as a score of 0 indicating the best outcome.
Outcome measures
| Measure |
rhDNase I
n=29 Participants
rhDNase I 0.1% eye drops 4 times a day for 8 weeks
rhDNase I: rhDNase I, 0.1% eye drops 4 times a day for 8 weeks
|
Vehicle
n=29 Participants
Drug vehicle eye drops 4 times a day for 8 weeks
Vehicle: Drug vehicle 4 times a day for 8 weeks
|
|---|---|---|
|
The Change in the Corneal Surface Staining as Measured by Dye Staining Within Groups.
Week 8
|
3.5 score on a scale
Interval 2.75 to 5.0
|
4.5 score on a scale
Interval 4.0 to 6.0
|
|
The Change in the Corneal Surface Staining as Measured by Dye Staining Within Groups.
Baseline
|
5.00 score on a scale
Interval 3.0 to 7.0
|
4.00 score on a scale
Interval 3.0 to 6.0
|
SECONDARY outcome
Timeframe: Between baseline and at 8 weeks of treatmentOcular Surface Disease Index (OSDI), a 12-item questionnaire, assesses symptom of ocular irritation in dry eye disease (DED) and how it affects functioning related to vision in the past week. It has 3 subscales: ocular symptoms, vision-related function, and environmental triggers. Patients rate their responses on 0 to 4 scale with 0 being "none of the time" and 4 being "all of the time." OSDI score range from 0-100 with score 0-12 being normal, 13-22 being mild DED, 23-32 being moderate DED, and \>33 being severe DED. OSDI=\[(sum of scores for questions answered)×100\]/\[(total questions answered)×4\]
Outcome measures
| Measure |
rhDNase I
n=29 Participants
rhDNase I 0.1% eye drops 4 times a day for 8 weeks
rhDNase I: rhDNase I, 0.1% eye drops 4 times a day for 8 weeks
|
Vehicle
n=29 Participants
Drug vehicle eye drops 4 times a day for 8 weeks
Vehicle: Drug vehicle 4 times a day for 8 weeks
|
|---|---|---|
|
The Change in Ocular Surface Disease Index (OSDI) Score Within Groups
Week 8
|
18.4 score on a scale
Interval 9.16 to 33.1
|
25.0 score on a scale
Interval 16.5 to 44.3
|
|
The Change in Ocular Surface Disease Index (OSDI) Score Within Groups
Baseline
|
45.5 score on a scale
Interval 31.8 to 50.0
|
37.5 score on a scale
Interval 31.8 to 56.0
|
Adverse Events
rhDNase I
Serious events: 0 serious events
Other events: 17 other events
Deaths: 0 deaths
Vehicle
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
rhDNase I
n=29 participants at risk
rhDNase I 0.1% eye drops 4 times a day for 8 weeks
rhDNase I: rhDNase I, 0.1% eye drops 4 times a day for 8 weeks
|
Vehicle
n=29 participants at risk
Drug vehicle eye drops 4 times a day for 8 weeks
Vehicle: Drug vehicle 4 times a day for 8 weeks
|
|---|---|---|
|
Eye disorders
Ocular discomfort
|
20.7%
6/29 • Between baseline and 8-weeks of treatment
|
3.4%
1/29 • Between baseline and 8-weeks of treatment
|
|
Eye disorders
Burning sensation
|
20.7%
6/29 • Between baseline and 8-weeks of treatment
|
10.3%
3/29 • Between baseline and 8-weeks of treatment
|
|
Eye disorders
Pain
|
10.3%
3/29 • Between baseline and 8-weeks of treatment
|
6.9%
2/29 • Between baseline and 8-weeks of treatment
|
|
Eye disorders
Blurring of vision
|
13.8%
4/29 • Between baseline and 8-weeks of treatment
|
13.8%
4/29 • Between baseline and 8-weeks of treatment
|
|
Eye disorders
Light sensitivity
|
13.8%
4/29 • Between baseline and 8-weeks of treatment
|
3.4%
1/29 • Between baseline and 8-weeks of treatment
|
|
Eye disorders
Dryness
|
17.2%
5/29 • Between baseline and 8-weeks of treatment
|
13.8%
4/29 • Between baseline and 8-weeks of treatment
|
|
Eye disorders
Redness
|
10.3%
3/29 • Between baseline and 8-weeks of treatment
|
6.9%
2/29 • Between baseline and 8-weeks of treatment
|
|
Eye disorders
Foreign body sensation
|
13.8%
4/29 • Between baseline and 8-weeks of treatment
|
10.3%
3/29 • Between baseline and 8-weeks of treatment
|
|
Eye disorders
Itching
|
0.00%
0/29 • Between baseline and 8-weeks of treatment
|
6.9%
2/29 • Between baseline and 8-weeks of treatment
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place