Trial Outcomes & Findings for PASS Study To Evaluate The Potential Of Zithromax To Cause Ocular Problems In Pediatric Patients (NCT NCT01919996)

Results Overview

Clinically significant worsening is an observed worsening in any of the five ophthalmic exams: 1) Clinically significant worsening in best corrected visual activity (BCVA) (distance) at the final visit, in either eye, is defined as a decrease in score of 5 or more letters from baseline in Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA. 2) An assessment of abnormal clinically significant at final visit in color vision Farnsworth Munsell 100 Hue Test (FM-100) in either eye. 3) An assessment of abnormal clinically significant at final visit in Amsler Grid in either eye. 4) Assessments of abnormal clinically significant at final visit in anterior segment biomicroscopy, in any of the 10 eye structures in either eye. 5) Assessments of abnormal clinically significant at final visit in dilated indirect ophthalmoscopy in any of the 5 eye structures in either eye.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

14 days

Results posted on

2016-08-05

Participant Flow

The sample size of 30 completed participants was specified by Food and Drug Administration (FDA) in the Post Marketing Commitment (PMC). Of the 30 pediatric participants planned for the study, 11 were screened and 8 participants received study treatment.

This was a prospective, non-comparative, open-label, single-arm study of azithromycin oral solution in 30 pediatric participants (aged 12 to 17 years) with pharyngitis/ tonsillitis who could be treated with azithromycin for their infection. The study design was intended to align with request from FDA to conduct the study.

Participant milestones

Participant milestones
Measure	Azithromycin All participants had received open-label azithromycin oral suspension immediate release (12 mg/kg/day, up to a maximum daily dose of 500 mg) on Days 1, 2, 3, 4, and 5.
Overall Study STARTED	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

PASS Study To Evaluate The Potential Of Zithromax To Cause Ocular Problems In Pediatric Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Azithromycin n=8 Participants All participants had received open-label azithromycin oral suspension immediate release (12 mg/kg/day, up to a maximum daily dose of 500 mg) on Days 1, 2, 3, 4, and 5.
Age, Continuous	14.4 Years STANDARD_DEVIATION 2.1 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: 14 days

Population: The safety population included all enrolled participants that took at least one dose of study medication. One participant was not evaluable because visual acuity was not corrected at Baseline (Day 1) and was corrected at Final Visit (Day 14).

Clinically significant worsening is an observed worsening in any of the five ophthalmic exams: 1) Clinically significant worsening in best corrected visual activity (BCVA) (distance) at the final visit, in either eye, is defined as a decrease in score of 5 or more letters from baseline in Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA. 2) An assessment of abnormal clinically significant at final visit in color vision Farnsworth Munsell 100 Hue Test (FM-100) in either eye. 3) An assessment of abnormal clinically significant at final visit in Amsler Grid in either eye. 4) Assessments of abnormal clinically significant at final visit in anterior segment biomicroscopy, in any of the 10 eye structures in either eye. 5) Assessments of abnormal clinically significant at final visit in dilated indirect ophthalmoscopy in any of the 5 eye structures in either eye.

Outcome measures

Outcome measures
Measure	Azithromycin n=8 Participants All participants had received open-label azithromycin oral suspension immediate release (12 mg/kg/day, up to a maximum daily dose of 500 mg) on Days 1, 2, 3, 4, and 5.
Occurrence of a Clinically Significant Worsening Based on Five Ophthalmic Examinations Clinically significant worsening - Yes	0.0 percentage of participants
Occurrence of a Clinically Significant Worsening Based on Five Ophthalmic Examinations Clinically significant worsening - No	87.5 percentage of participants
Occurrence of a Clinically Significant Worsening Based on Five Ophthalmic Examinations Not Evaluable	12.5 percentage of participants

SECONDARY outcome

Timeframe: 14 days

Population: The safety population included all enrolled participants that took at least one dose of study medication. One participant was not evaluable because visual acuity was not corrected at Baseline (Day 1) and was corrected at Final Visit (Day 14).

1 or more of these conditions are clinically significant improvement based on five ophthalmic exams:1) clinically significant improvement in BCVA(distance) at the final visit, in either eye, defined as an increase in score of 5 or more letters from baseline in ETDRS BCVA.2) Assessment of abnormal clinically significant at baseline and normal or abnormal, non-clinically significant at final visit in color vision(FM-100) in either eye. 3) Assessment of abnormal clinically significant at baseline and normal/abnormal, non-clinically significant at final visit in Amsler Grid in either eye. 4) Assessments of abnormal clinically significant at baseline and normal/abnormal, non-clinically significant at final visit in anterior segment biomicroscopy, in any of the 10 eye structures in either eye. 5)Assessments of abnormal clinically significant at baseline and normal/abnormal, nonclinically significant at final visit in dilated ophthalmoscopy in any of the 5 eye structures in either eye.

Outcome measures

Outcome measures
Measure	Azithromycin n=8 Participants All participants had received open-label azithromycin oral suspension immediate release (12 mg/kg/day, up to a maximum daily dose of 500 mg) on Days 1, 2, 3, 4, and 5.
Occurrence of a Clinically Significant Improvement Based on Five Ophthalmic Examinations Clinically Significant Improvement - Yes	12.5 percentage of participants
Occurrence of a Clinically Significant Improvement Based on Five Ophthalmic Examinations Clinically Significant Improvement - No	75.0 percentage of participants
Occurrence of a Clinically Significant Improvement Based on Five Ophthalmic Examinations Not Evaluable	12.5 percentage of participants

SECONDARY outcome

Timeframe: 14 days

Population: The safety population included all enrolled participants that took at least one dose of study medication. One participant was not evaluable because visual acuity was not corrected at Baseline (Day 1) and was corrected at Final Visit (Day 14).

Clinically significant change (improvement or worsening) is based on five ophthalmic exams at baseline and the final visit. Any 1 or more of these conditions are a clinically significant change: 1) A worsening in BCVA (distance), as defined in outcome measure 1 OR an improvement in BCVA (distance) as defined in outcome measure 2. 2) A worsening in color vision (FM-100), as defined in outcome measure 1 OR an improvement in color vision (FM-100) as defined in outcome measure 2. 3) A worsening in Amsler Grid, as defined in outcome measure 1, OR an improvement in Amsler Grid, as defined in outcome measure 2. 4) A worsening in anterior segment biomicroscopy, as defined in outcome measure 1 OR an improvement in anterior segment biomicroscopy as defined in outcome measure 2. 5) A worsening in dilated indirect ophthalmoscopy, as defined in outcome measure 1 OR an improvement in dilated indirect ophthalmoscopy as defined in outcome measure 2.

Outcome measures

Outcome measures
Measure	Azithromycin n=8 Participants All participants had received open-label azithromycin oral suspension immediate release (12 mg/kg/day, up to a maximum daily dose of 500 mg) on Days 1, 2, 3, 4, and 5.
Occurrence of a Clinically Significant Change (Improvement or Worsening) Based on Five Ophthalmic Examinations Significant Change (Improvement or Worsening) -Yes	12.5 percentage of participants
Occurrence of a Clinically Significant Change (Improvement or Worsening) Based on Five Ophthalmic Examinations Significant Change (Improvement or Worsening) - No	75.0 percentage of participants
Occurrence of a Clinically Significant Change (Improvement or Worsening) Based on Five Ophthalmic Examinations Not Evaluable	12.5 percentage of participants

Adverse Events

Azithromycin

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

Pfizer ClinicalTrials.gov Call Center

Pfizer, Inc.

Phone: 1-800-718-1021

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Publication restrictions are in place

Restriction type: OTHER