Trial Outcomes & Findings for Nasally Delivered Pulmozyme for Sinusitis in Cystic Fibrosis (NCT NCT00416182)

Results Overview

compare sinus CT pre-op (baseline) to one year after initiation of study drug Difference in pre and post scores by Lund-McKay scoring system are reported (1 year minus baseline) The Lund-Mackay scoring system was used to evaluate the extent and severity of sinusitis. The scale ranges from 0 (best possible outcome with complete lucency of all sinuses) to 24 (worst possible outcome with complete opacification of all sinuses)

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

16 participants

Primary outcome timeframe

baseline and 1 year

Results posted on

2014-11-14

Participant Flow

Patients undergoing sinus surgery meeting inclusion criteria were approached in the outpatient or hospital setting

upon providing informed consent, subjects were randomized and blinded to treatment or placebo groups

Participant milestones

Participant milestones
Measure	Pulmozyme (Dornase Alfa) 2.5 mg/2.5mL of Pulmozyme administered intranasally once daily	Placebo 2.5mg/2.5mL placebo administered intranasally once daily
Overall Study STARTED	8	8
Overall Study COMPLETED	5	6
Overall Study NOT COMPLETED	3	2

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Nasally Delivered Pulmozyme for Sinusitis in Cystic Fibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pulmozyme (Dornase Alfa) n=8 Participants 2.5 mg/2.5 mL of intranasal Pulmozyme	Placebo n=8 Participants 2.5 mL of placebo comparator	Total n=16 Participants Total of all reporting groups
Age, Categorical <=18 years	3 Participants n=5 Participants	6 Participants n=7 Participants	9 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants	2 Participants n=7 Participants	7 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	5 Participants n=7 Participants	10 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Region of Enrollment United States	8 participants n=5 Participants	8 participants n=7 Participants	16 participants n=5 Participants

PRIMARY outcome

Timeframe: baseline and 1 year

compare sinus CT pre-op (baseline) to one year after initiation of study drug Difference in pre and post scores by Lund-McKay scoring system are reported (1 year minus baseline) The Lund-Mackay scoring system was used to evaluate the extent and severity of sinusitis. The scale ranges from 0 (best possible outcome with complete lucency of all sinuses) to 24 (worst possible outcome with complete opacification of all sinuses)

Outcome measures

Outcome measures
Measure	Pulmozyme n=5 Participants Patients receiving 2.5 mg intranasal Pulmozyme once daily	Placebo n=6 Participants Patients receiving intranasal placebo once daily
Computed Tomography Evidence of Less Sinus Disease	5.875 units on a scale Interval -0.436 to 12.19	3 units on a scale Interval 0.055 to 5.945

PRIMARY outcome

Timeframe: baseline and 1 year

periodic endoscopic photos of sinuses by ear-nose-throat (ENT) surgeon. The scale for scoring severity of disease ranges from 0 (best possible outcome) to 2 (worst possible outcome). independent blinded scoring by 2 surgeons difference in scores pre and post are reported (1 year minus baseline)

Outcome measures

Outcome measures
Measure	Pulmozyme n=5 Participants Patients receiving 2.5 mg intranasal Pulmozyme once daily	Placebo n=6 Participants Patients receiving intranasal placebo once daily
Improvement in Appearance of Nasal Passages/Sinuses	0.2 units Interval -0.84 to 1.24	-1 units Interval -2.24 to 0.24

SECONDARY outcome

Timeframe: baseline and 1 year

pre-surgery and end of trial (12 months) Reduction in scores (baseline minus 1 year) are recorded The chronic sinusitis survey consists of 6 questions, ranges from 0-24, a lower score indicates the best possible outcome.

Outcome measures

Outcome measures
Measure	Pulmozyme n=5 Participants Patients receiving 2.5 mg intranasal Pulmozyme once daily	Placebo n=6 Participants Patients receiving intranasal placebo once daily
Chronic Sinusitis Survey Score	8.5 units on a scale Interval 5.45 to 11.55	3 units on a scale Interval -1.92 to 7.92

SECONDARY outcome

Timeframe: baseline and 1 year

prior to surgery and end of study spirometry as measured by forced expiratory volume in 1 second (FEV1) percent predicted. The change over the course of the study (1 year minus baseline) is reported. A higher value indicates a better outcome.

Outcome measures

Outcome measures
Measure	Pulmozyme n=5 Participants Patients receiving 2.5 mg intranasal Pulmozyme once daily	Placebo n=6 Participants Patients receiving intranasal placebo once daily
Pulmonary Function	0.8 percentage of predicted FEV1 Interval -20.0 to 17.0	5.4 percentage of predicted FEV1 Interval -3.0 to 18.0

Adverse Events

Pulmozyme

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Placebo

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Pulmozyme n=8 participants at risk patients receiving once daily intranasal Pulmozyme	Placebo n=8 participants at risk patients receiving once daily intranasal placebo
Respiratory, thoracic and mediastinal disorders hospitalization for pulmonary exacerbation	12.5% 1/8 • Number of events 1 • 1 year (study period)	25.0% 2/8 • Number of events 2 • 1 year (study period)

Other adverse events

Other adverse events
Measure	Pulmozyme n=8 participants at risk patients receiving once daily intranasal Pulmozyme	Placebo n=8 participants at risk patients receiving once daily intranasal placebo
Respiratory, thoracic and mediastinal disorders sinus surgery	12.5% 1/8 • Number of events 1 • 1 year (study period)	0.00% 0/8 • 1 year (study period)

Additional Information

Dr. Thomas Lahiri, Principal Investigator

Vermont Children's Hospital at Fletcher Allen Health Care

Phone: 802-847-8600

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60