Trial Outcomes & Findings for Dornase Alfa Therapy for Ventilator Associated Lung Infections in the Neonatal Intensive Care Unit (NICU) (NCT NCT01356147)

Results Overview

Change in required supplemental oxygen from baseline or time to extubation from mechanical ventilation

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

11 participants

Primary outcome timeframe

First week of treatment or extubation

Results posted on

2018-08-07

Participant Flow

Medstar Georgetown University Hospita NICU patients recruited from May 2012 to november 2014

No enrolled participants were excluded prior to assignment to groups

Participant milestones

Participant milestones
Measure	Sham Placebo No therapy will be given to placebo arm. Respiratory therapist will shield infant from view and nebulize saline solution into incubator rather than into ventilator circuit. Placebo: No therapy will be given to placebo arm	Dornase Alfa Dornase alfa 2.5 mg nebulized endotracheally every 12 hours for 7 days or until extubation Dornase alfa: 2.5 mg nebulized endotracheally every 12 hours for 7 days or until extubation
Overall Study STARTED	5	6
Overall Study COMPLETED	5	6
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Dornase Alfa Therapy for Ventilator Associated Lung Infections in the Neonatal Intensive Care Unit (NICU)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Sham Placebo n=5 Participants No therapy will be given to placebo arm. Respiratory therapist will shield infant from view and nebulize saline solution into incubator rather than into ventilator circuit. Placebo: No therapy will be given to placebo arm	Dornase Alfa n=6 Participants Dornase alfa 2.5 mg nebulized endotracheally every 12 hours for 7 days or until extubation Dornase alfa: 2.5 mg nebulized endotracheally every 12 hours for 7 days or until extubation	Total n=11 Participants Total of all reporting groups
Age, Categorical <=18 years	5 Participants n=5 Participants	6 Participants n=7 Participants	11 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	29 days n=5 Participants	33 days n=7 Participants	31 days n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants	6 participants n=7 Participants	11 participants n=5 Participants

PRIMARY outcome

Timeframe: First week of treatment or extubation

Change in required supplemental oxygen from baseline or time to extubation from mechanical ventilation

Outcome measures

Outcome measures
Measure	Sham Placebo n=5 Participants No therapy will be given to placebo arm. Respiratory therapist will shield infant from view and nebulize saline solution into incubator rather than into ventilator circuit. Placebo: No therapy will be given to placebo arm	Dornase Alfa n=6 Participants Dornase alfa 2.5 mg nebulized endotracheally every 12 hours for 7 days or until extubation Dornase alfa: 2.5 mg nebulized endotracheally every 12 hours for 7 days or until extubation
Percent Reduction in Oxygen Requirement From Baseline	5.4 percentage FiO2 Interval -2.0 to 23.0	16.5 percentage FiO2 Interval -4.0 to 73.0

SECONDARY outcome

Timeframe: During first week of treatment or until extubation whichever is earlier

Number of infants with Tracheal aspirate WBC present on review of smear at end of therapy Bacterial load judged on presence of positive or negative culture in Tracheal aspirate

Outcome measures

Outcome measures
Measure	Sham Placebo n=5 Participants No therapy will be given to placebo arm. Respiratory therapist will shield infant from view and nebulize saline solution into incubator rather than into ventilator circuit. Placebo: No therapy will be given to placebo arm	Dornase Alfa n=6 Participants Dornase alfa 2.5 mg nebulized endotracheally every 12 hours for 7 days or until extubation Dornase alfa: 2.5 mg nebulized endotracheally every 12 hours for 7 days or until extubation
Elimination of White Blood Cells and Bacteria From Tracheal Aspirate	1 participants	4 participants

SECONDARY outcome

Timeframe: 7 days

number of infants extubated during treatment/sham

Outcome measures

Outcome measures
Measure	Sham Placebo n=5 Participants No therapy will be given to placebo arm. Respiratory therapist will shield infant from view and nebulize saline solution into incubator rather than into ventilator circuit. Placebo: No therapy will be given to placebo arm	Dornase Alfa n=6 Participants Dornase alfa 2.5 mg nebulized endotracheally every 12 hours for 7 days or until extubation Dornase alfa: 2.5 mg nebulized endotracheally every 12 hours for 7 days or until extubation
Number of Infants Requiring Ventilator Support	1 participants	0 participants

Adverse Events

Sham Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Dornase Alfa

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

Dr. Melissa Scala

Stanford University School of Medicine/Division of Neonatology

Phone: 650-724-9653

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place