Trial Outcomes & Findings for Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder (NCT NCT00755040)
NCT ID: NCT00755040
Last Updated: 2017-03-07
Results Overview
Data analysis will be performed on an intention-to-treat basis. Logistic regression will be used to estimate the odds ratio and 95% confidence interval of the two treatment groups with the adjustment of baseline covariates. Outcome comparisons for categorical/dichotomous variables will be assessed by 2 test or Fisher's exact test where expected cell frequencies were \<5; continuous variables will be compared by X/2 test or by Mann-Whitney U test where the data are strongly skewed.
COMPLETED
PHASE3
164 participants
Up to 2 years after transplantation
2017-03-07
Participant Flow
Participant milestones
| Measure |
Arm I
Patients receive cyclosporine ophthalmic emulsion (Restasis®) drops in each eye twice daily for up to 1 year after transplant.
cyclosporine ophthalmic emulsion: Given as eye drops
|
Arm II
Patients receive placebo ophthalmic drops in each eye twice daily for up to 1 year after transplant.
placebo: Given as eye drops
|
|---|---|---|
|
Overall Study
STARTED
|
83
|
81
|
|
Overall Study
COMPLETED
|
40
|
47
|
|
Overall Study
NOT COMPLETED
|
43
|
34
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder
Baseline characteristics by cohort
| Measure |
Ocular Cyclosporine (Restasis)
n=83 Participants
Patients receive cyclosporine ophthalmic emulsion (Restasis®) drops in each eye twice daily for up to 1 year after transplant.
cyclosporine ophthalmic emulsion: Given as eye drops
|
Placebo
n=81 Participants
Patients receive placebo ophthalmic drops in each eye twice daily for up to 1 year after transplant.
placebo: Given as eye drops
|
Total
n=164 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
81 Participants
n=99 Participants
|
75 Participants
n=107 Participants
|
156 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Age, Continuous
|
49 Years
STANDARD_DEVIATION 12.458 • n=99 Participants
|
49 Years
STANDARD_DEVIATION 11.707 • n=107 Participants
|
49 Years
STANDARD_DEVIATION 12.062 • n=206 Participants
|
|
Sex: Female, Male
Female
|
34 Participants
n=99 Participants
|
39 Participants
n=107 Participants
|
73 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
49 Participants
n=99 Participants
|
42 Participants
n=107 Participants
|
91 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
83 participants
n=99 Participants
|
81 participants
n=107 Participants
|
164 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Up to 2 years after transplantationPopulation: Number of patients that develop ocular GVHD while on study
Data analysis will be performed on an intention-to-treat basis. Logistic regression will be used to estimate the odds ratio and 95% confidence interval of the two treatment groups with the adjustment of baseline covariates. Outcome comparisons for categorical/dichotomous variables will be assessed by 2 test or Fisher's exact test where expected cell frequencies were \<5; continuous variables will be compared by X/2 test or by Mann-Whitney U test where the data are strongly skewed.
Outcome measures
| Measure |
Arm I
n=83 Participants
Patients receive cyclosporine ophthalmic emulsion (Restasis®) drops in each eye twice daily for up to 1 year after transplant.
cyclosporine ophthalmic emulsion: Given as eye drops
|
Arm II
n=81 Participants
Patients receive placebo ophthalmic drops in each eye twice daily for up to 1 year after transplant.
placebo: Given as eye drops
|
|---|---|---|
|
Number of Patients That Develop Ocular GVHD While on Study in the Two Arms (Ocular Cyclosporine (Restasis) vs. Placebo)
|
9 participants
|
8 participants
|
SECONDARY outcome
Timeframe: 1 year after transplantPopulation: Not collected/analysed due to staffing constraints
OSDI is a patient reported questionnaire consisting of 12 questions which are scored from 0 to 4. The total scored is computed and depending on the number of questions answered scores are computed which are then categorized into mild, moderate or severe dry eye symptoms. This tool should adequately reflect the morbidity of the dry eye symptoms.
Outcome measures
Outcome data not reported
Adverse Events
Arm I
Arm II
Serious adverse events
| Measure |
Arm I
n=83 participants at risk
Patients receive cyclosporine ophthalmic emulsion (Restasis®) drops in each eye twice daily for up to 1 year after transplant.
cyclosporine ophthalmic emulsion: Given as eye drops
|
Arm II
n=81 participants at risk
Patients receive placebo ophthalmic drops in each eye twice daily for up to 1 year after transplant.
placebo: Given as eye drops
|
|---|---|---|
|
Blood and lymphatic system disorders
Blood/Bone Marrow
|
3.6%
3/83 • Number of events 3
|
4.9%
4/81 • Number of events 4
|
|
General disorders
Multi-organ failure
|
0.00%
0/83
|
1.2%
1/81 • Number of events 1
|
|
Blood and lymphatic system disorders
Blood/Bone Marrow Other
|
0.00%
0/83
|
2.5%
2/81 • Number of events 2
|
Other adverse events
| Measure |
Arm I
n=83 participants at risk
Patients receive cyclosporine ophthalmic emulsion (Restasis®) drops in each eye twice daily for up to 1 year after transplant.
cyclosporine ophthalmic emulsion: Given as eye drops
|
Arm II
n=81 participants at risk
Patients receive placebo ophthalmic drops in each eye twice daily for up to 1 year after transplant.
placebo: Given as eye drops
|
|---|---|---|
|
Eye disorders
Burning
|
37.3%
31/83 • Number of events 49
|
11.1%
9/81 • Number of events 18
|
|
Eye disorders
Red eye
|
15.7%
13/83 • Number of events 13
|
22.2%
18/81 • Number of events 22
|
|
Eye disorders
Discharge
|
10.8%
9/83 • Number of events 13
|
9.9%
8/81 • Number of events 8
|
|
Eye disorders
Excessive tearing
|
16.9%
14/83 • Number of events 20
|
18.5%
15/81 • Number of events 21
|
|
Eye disorders
Eye pain
|
6.0%
5/83 • Number of events 6
|
7.4%
6/81 • Number of events 6
|
|
Eye disorders
Foreign Body Sensation
|
6.0%
5/83 • Number of events 5
|
9.9%
8/81 • Number of events 9
|
|
Eye disorders
Itching
|
19.3%
16/83 • Number of events 20
|
11.1%
9/81 • Number of events 13
|
|
Eye disorders
Stinging
|
20.5%
17/83 • Number of events 22
|
9.9%
8/81 • Number of events 9
|
|
Eye disorders
Visual disturbance
|
15.7%
13/83 • Number of events 14
|
22.2%
18/81 • Number of events 23
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place