Trial Outcomes & Findings for Safety and Efficacy Study of Besivance™ for Treatment of Congenital Nasolacrimal Duct Obstruction in Children (NCT NCT01431170)

Results Overview

The NLDO grading scale in the study eye at every visit. The scale ranges from 0 to +4: * 0: No tearing and discharge. * 1: Tearing, moderate mucous discharge around nasolacrimal punctum * 2: Moderate redness of the medial eyelid with mucous discharge * 3: Redness and swelling of the eyelid with mucopurulent discharge * 4: Redness and swelling of eyelid with purulent discharge Due to varying baseline severity (measured by NLDO grade) among subjects, change from baseline to week 8 in NLDO grade was further classified as the following: Treatment success: grade of 0 or improvement by 2 or more compared to the prior visit. Recurrence: NLDO with infection returns in the study eye, as indicated by a NLDO grade \>0 after a grade of 0 at the prior visit. Treatment Failure: grade is worse than or same as the baseline visit.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

24 participants

Primary outcome timeframe

Baseline to Week 8

Results posted on

2016-07-14

Participant Flow

Participant milestones

Participant milestones
Measure	Besivance Treatment Group Subjects receive Besivance™ ophthalmic suspension, 0.6% one drop in the study eye three times daily for 10 days.	Polytrim Treatment Group Subjects receive Polytrim ophthalmic solution one drop in the study eye three times daily for 10 days.
Overall Study STARTED	12	12
Overall Study COMPLETED	9	11
Overall Study NOT COMPLETED	3	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety and Efficacy Study of Besivance™ for Treatment of Congenital Nasolacrimal Duct Obstruction in Children

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Besivance Treatment Group n=12 Participants Subjects received Besivance™ ophthalmic suspension, 0.6% one drop in the study eye three times daily (TID) for 10 days.	Polytrim Treatment Group n=12 Participants Subjects received Polytrim ophthalmic solution one drop in the study eye three times daily (TID) for 10 days.	Total n=24 Participants Total of all reporting groups
Age, Categorical <=18 years	12 Participants n=5 Participants	12 Participants n=7 Participants	24 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	110.83 days STANDARD_DEVIATION 93.05 • n=5 Participants	158.58 days STANDARD_DEVIATION 110.62 • n=7 Participants	134.71 days STANDARD_DEVIATION 102.90 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	8 Participants n=7 Participants	13 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants	4 Participants n=7 Participants	11 Participants n=5 Participants
Region of Enrollment United States	12 participants n=5 Participants	12 participants n=7 Participants	24 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to Week 8

The NLDO grading scale in the study eye at every visit. The scale ranges from 0 to +4: * 0: No tearing and discharge. * 1: Tearing, moderate mucous discharge around nasolacrimal punctum * 2: Moderate redness of the medial eyelid with mucous discharge * 3: Redness and swelling of the eyelid with mucopurulent discharge * 4: Redness and swelling of eyelid with purulent discharge Due to varying baseline severity (measured by NLDO grade) among subjects, change from baseline to week 8 in NLDO grade was further classified as the following: Treatment success: grade of 0 or improvement by 2 or more compared to the prior visit. Recurrence: NLDO with infection returns in the study eye, as indicated by a NLDO grade \>0 after a grade of 0 at the prior visit. Treatment Failure: grade is worse than or same as the baseline visit.

Outcome measures

Outcome measures
Measure	Besivance Treatment Group n=9 Participants Subjects received Besivance ophthalmic solution in the study eye, one drop three times a day for ten days	Polytrim Treatment Group n=11 Participants Subjects received Polytrim ophthalmic solution in the study eye, one drop three times daily for ten days.
Change in Physician-rated Scale of NLDO From Baseline to Follow-Up Visit at Week 8 or From Baseline to Time of Treatment Failure, if Earlier. Number of Treatment Success	7 participants	10 participants
Change in Physician-rated Scale of NLDO From Baseline to Follow-Up Visit at Week 8 or From Baseline to Time of Treatment Failure, if Earlier. Number of Recurrence	1 participants	1 participants
Change in Physician-rated Scale of NLDO From Baseline to Follow-Up Visit at Week 8 or From Baseline to Time of Treatment Failure, if Earlier. Number of Treatment Failure	1 participants	0 participants

SECONDARY outcome

Timeframe: Baseline to Week 16 (Closeout Visit )

Population: The overall study recurrence rate was 10% study-wide (2 of 20 subjects), with one Besivance subject (11%), and one Polytrim subject (9%) experiencing recurrence.

Recurrence is defined as when the NLDO with infection in the subject's study eye returns, as indicated by a NLDO grading scale of greater than zero after achieving a grade of zero at the previous visit. Number of subjects who had a recurrence event of the subjects who completed the study by treatment Group.

Outcome measures

Outcome measures
Measure	Besivance Treatment Group n=9 Participants Subjects received Besivance ophthalmic solution in the study eye, one drop three times a day for ten days	Polytrim Treatment Group n=11 Participants Subjects received Polytrim ophthalmic solution in the study eye, one drop three times daily for ten days.
Number of Recurrences by Randomization Group	1 Recurrence Subjects	1 Recurrence Subjects

SECONDARY outcome

Timeframe: Baseline to Week 16 (Closeout Visit)

Subjects who experience recurrence were re-treated as if they were a new patient, with the same study medication, were followed up then classified as "Treatment Success" or "Treatment Failure" according to study protocol.

Outcome measures

Outcome measures
Measure	Besivance Treatment Group n=1 Participants Subjects received Besivance ophthalmic solution in the study eye, one drop three times a day for ten days	Polytrim Treatment Group n=1 Participants Subjects received Polytrim ophthalmic solution in the study eye, one drop three times daily for ten days.
Efficacy of Recurrence Treatment as Measured by Change in the Physician- Rated Scale of NLDO Number of Treatment Failure after Recurrence	0 participants	1 participants
Efficacy of Recurrence Treatment as Measured by Change in the Physician- Rated Scale of NLDO Number of Treatment Success after Recurrence	1 participants	0 participants

SECONDARY outcome

Timeframe: Baseline to the time of failure or Week 16 (Closeout Visit)

Possible treatment failure at a follow-up examination is operationally defined as follows: if the physician-grading scale of NLDO is worse than or same as the prior visit at any given follow-up visit, possible treatment failure then exists. Treatment Failure occurred if at visit #1 (2-week visit), the physician-grading scale of NLDO is worse than or same as the baseline visit. Treatment failure can also occur at recurrence visit #1 if the NLDO grading scale is worse or the same as compared to the previous visit. Subjects who meet the criteria for treatment failure were withdrawn from the study by the principal investigator and no additional data was collected. Subjects were referred for continued care.

Outcome measures

Outcome measures
Measure	Besivance Treatment Group n=9 Participants Subjects received Besivance ophthalmic solution in the study eye, one drop three times a day for ten days	Polytrim Treatment Group n=11 Participants Subjects received Polytrim ophthalmic solution in the study eye, one drop three times daily for ten days.
Treatment Failure	1 Number of Treatment Failures	1 Number of Treatment Failures

SECONDARY outcome

Timeframe: Baseline to Week 16 (Closeout Visit )

Population: No safety issues were reported

During each study visit, the Principal Investigator will evaluate any possible adverse events by assessing clinical complaints and symptoms that are experienced by subjects and observed by the parent(s)/legal guardian(s), including findings in external, lacrimal duct system and anterior segment using slit lamp and fundus exam using indirect ophthalmoscope by principal investigator, as well as clinical signs including findings in external, nasolacrimal duct system and anterior segment using slit lamp and fundus exam using indirect ophthalmoscope by principal investigator.

Outcome measures

Outcome measures
Measure	Besivance Treatment Group n=9 Participants Subjects received Besivance ophthalmic solution in the study eye, one drop three times a day for ten days	Polytrim Treatment Group n=11 Participants Subjects received Polytrim ophthalmic solution in the study eye, one drop three times daily for ten days.
Medication Safety Outcomes	0 Number of Adverse Events	0 Number of Adverse Events

POST_HOC outcome

Timeframe: Baseline to Week 16 (Close-Out Visit)

Treatment Success is defined as a grade of 0 or improvement by 2 or more compared to the prior visit.

Outcome measures

Outcome measures
Measure	Besivance Treatment Group n=9 Participants Subjects received Besivance ophthalmic solution in the study eye, one drop three times a day for ten days	Polytrim Treatment Group n=11 Participants Subjects received Polytrim ophthalmic solution in the study eye, one drop three times daily for ten days.
Number of Subjects Treated Successfully at Close-Out Visit (Week 16)	8 Number of Treatment Success	10 Number of Treatment Success

Adverse Events

Besivance Treatment Group

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Polytrim Treatment Group

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Suqin Guo, MD

NJMS, Rutgers University

Phone: 973-972-2031

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place