Trial Outcomes & Findings for Effect of Lucinactant on Mucus Clearance in Cystic Fibrosis Lung Disease (NCT NCT00934362)

NCT ID: NCT00934362

Last Updated: 2017-03-13

Results Overview

Clearance of radiolabeled particles, following inhalation, are followed over time. Average clearance rate through 60 minutes post inhaled isotope deposition is calculated. Absolute difference between baseline and post-treatment (e.g. \<60 minutes after the last dose of lucinactant or placebo) reported.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

16 participants

Primary outcome timeframe

1 hour after final treatment (5th dose) minus baseline

Results posted on

2017-03-13

Participant Flow

two week washout period between treatment phases of this cross-over study

Participant milestones

Participant milestones
Measure
Lucinactant First, Then Placebo
20 mg/ml x 6 ml (5 doses) lucinactant then 0.9% NaCl x 6 ml (5 doses)
Placebo First, Then Lucinactant
0.9% NaCl x 6 ml (5 doses) then 20 mg/ml x 6 ml lucinactant (5 doses)
Overall Study
STARTED
8
8
Overall Study
COMPLETED
7
8
Overall Study
NOT COMPLETED
1
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Lucinactant First, Then Placebo
20 mg/ml x 6 ml (5 doses) lucinactant then 0.9% NaCl x 6 ml (5 doses)
Placebo First, Then Lucinactant
0.9% NaCl x 6 ml (5 doses) then 20 mg/ml x 6 ml lucinactant (5 doses)
Overall Study
Adverse Event
1
0

Baseline Characteristics

Effect of Lucinactant on Mucus Clearance in Cystic Fibrosis Lung Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Lucinactant First, Then Placebo
n=8 Participants
20 mg/ml x 6 ml (5 doses) lucinactant then 0.9% NaCl x 6 ml (5 doses)
Placebo First, Then Lucinactant
n=8 Participants
0.9% NaCl x 6 ml (5 doses) then 20 mg/ml x 6 ml lucinactant (5 doses)
Total
n=16 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=5 Participants
1 Participants
n=7 Participants
1 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
8 Participants
n=5 Participants
7 Participants
n=7 Participants
15 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
28.3 years
STANDARD_DEVIATION 8.8 • n=5 Participants
25.9 years
STANDARD_DEVIATION 6.4 • n=7 Participants
27.1 years
STANDARD_DEVIATION 7.5 • n=5 Participants
Sex: Female, Male
Female
4 Participants
n=5 Participants
4 Participants
n=7 Participants
8 Participants
n=5 Participants
Sex: Female, Male
Male
4 Participants
n=5 Participants
4 Participants
n=7 Participants
8 Participants
n=5 Participants
Region of Enrollment
United States
8 participants
n=5 Participants
8 participants
n=7 Participants
16 participants
n=5 Participants
Baseline Mucociliary Clearance Rate
9.2 Percentage Clearance
STANDARD_DEVIATION 4.8 • n=5 Participants
12.0 Percentage Clearance
STANDARD_DEVIATION 10.5 • n=7 Participants
10.5 Percentage Clearance
STANDARD_DEVIATION 8.1 • n=5 Participants

PRIMARY outcome

Timeframe: 1 hour after final treatment (5th dose) minus baseline

Population: Per protocol

Clearance of radiolabeled particles, following inhalation, are followed over time. Average clearance rate through 60 minutes post inhaled isotope deposition is calculated. Absolute difference between baseline and post-treatment (e.g. \<60 minutes after the last dose of lucinactant or placebo) reported.

Outcome measures

Outcome measures
Measure
Lucinactant
n=15 Participants
20 mg/ml x 6 ml lucinactant (5 doses)
Placebo
n=15 Participants
0.9% NaCl x 6 ml (5 doses)
Change in Mucociliary Clearance
7.4 percent clearance
Standard Deviation 9.3
9.5 percent clearance
Standard Deviation 10.0

SECONDARY outcome

Timeframe: after 5 doses

Population: per protocol

Percent change (relative) in FEV1 between pre-treatment baseline and following 5 doses of study treatment. Post treatment values obtained 3 and approximately 22 hours after 5th dose were averaged to determine the treatment effect.

Outcome measures

Outcome measures
Measure
Lucinactant
n=15 Participants
20 mg/ml x 6 ml lucinactant (5 doses)
Placebo
n=15 Participants
0.9% NaCl x 6 ml (5 doses)
Spirometry
.81 percent change
Standard Deviation .9
.23 percent change
Standard Deviation 1.1

Adverse Events

Lucinactant

Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths

Placebo

Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Lucinactant
n=16 participants at risk
20 mg/ml x 6 ml (5 doses)
Placebo
n=15 participants at risk
0.9% NaCl x 6 ml (5 doses)
Respiratory, thoracic and mediastinal disorders
cough
18.8%
3/16 • Number of events 3
13.3%
2/15 • Number of events 2
Respiratory, thoracic and mediastinal disorders
sinonasal congestion
12.5%
2/16 • Number of events 3
0.00%
0/15
Nervous system disorders
headache
6.2%
1/16 • Number of events 1
13.3%
2/15 • Number of events 2
Respiratory, thoracic and mediastinal disorders
hemoptysis
0.00%
0/16
13.3%
2/15 • Number of events 3
Respiratory, thoracic and mediastinal disorders
sore throat
6.2%
1/16 • Number of events 1
6.7%
1/15 • Number of events 1
General disorders
fatigue
0.00%
0/16
6.7%
1/15 • Number of events 1
Investigations
Decreased lung function
6.2%
1/16 • Number of events 1
0.00%
0/15
Gastrointestinal disorders
Abdominal pain
0.00%
0/16
6.7%
1/15 • Number of events 1
Respiratory, thoracic and mediastinal disorders
Cystic Fibrosis exacerbation
0.00%
0/16
6.7%
1/15 • Number of events 1
Respiratory, thoracic and mediastinal disorders
Nasal dryness
6.2%
1/16 • Number of events 1
0.00%
0/15

Additional Information

Dr. Scott Donaldson

University of North Carolina at Chapel Hill

Phone: 919-966-9198

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: LTE60