Trial Outcomes & Findings for A Study of Pulmozyme® (Dornase Alpha) in 3- to 5-Year-Old Patients With Cystic Fibrosis (NCT NCT00680316)
NCT ID: NCT00680316
Last Updated: 2017-01-12
Results Overview
The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung.
TERMINATED
PHASE4
3 participants
from Visit 2 to Visit 3 (16 +/- 2 days)
2017-01-12
Participant Flow
Participant milestones
| Measure |
Dornase Alfa
2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days
|
Placebo
2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days
|
|---|---|---|
|
Overall Study
STARTED
|
1
|
2
|
|
Overall Study
COMPLETED
|
1
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of Pulmozyme® (Dornase Alpha) in 3- to 5-Year-Old Patients With Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Dornase Alfa
n=1 Participants
2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days
|
Placebo
n=2 Participants
2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days
|
Total
n=3 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
5.6 years
n=99 Participants
|
4.6 years
STANDARD_DEVIATION 1.5 • n=107 Participants
|
4.9 years
STANDARD_DEVIATION 1.2 • n=206 Participants
|
|
Gender
Female
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Gender
Male
|
0 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: from Visit 2 to Visit 3 (16 +/- 2 days)Population: Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT.
The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: from Visit 2 to Visit 3 (16 +/- 2 days)Population: Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT.
The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: from Visit 2 to Visit 3 (16 +/- 2 days)Population: Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT.
The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Resistance is complex measure that incorporates the lack of changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (10Hz was used for the secondary endpoint).
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: from Visit 2 to Visit 3 (16 +/- 2 days)Population: Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT.
The CFQ-R for Preschoolers and the CFQ-R for Parents of Preschoolers was designed specifically to measure the impact of CF for patients with a diagnosis of CF. Each question is answered using a 4-point Likert scale. In order to calculate the domain/symptom scale scores, the following algorithm is followed * Re-number items which have been reverse coded * Calculate the mean of the items to be included. If more than half of the items are missing, then the score is considered missing * Re-scale to result in a scaled score which ranges from 0 to 100, with higher scores indicating better health
Outcome measures
Outcome data not reported
Adverse Events
Dornase Alfa
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Dornase Alfa
n=1 participants at risk
2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days
|
Placebo
n=2 participants at risk
2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Increase in cough
|
100.0%
1/1
|
0.00%
0/2
|
|
Psychiatric disorders
Bad dreams
|
0.00%
0/1
|
50.0%
1/2
|
|
General disorders
Flu symptoms
|
0.00%
0/1
|
50.0%
1/2
|
Additional Information
Medical Communications
Genentech, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
- Publication restrictions are in place
Restriction type: OTHER