Trial Outcomes & Findings for Deposition of Inhaled Prolastin in Cystic Fibrosis Patients (NCT NCT00486837)
NCT ID: NCT00486837
Last Updated: 2014-08-21
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
72 participants
Primary outcome timeframe
Baseline vs Week 4
Results posted on
2014-08-21
Participant Flow
This study was performed in 8 study centers in Germany.
Subjects inhaled saline once daily for 2 weeks during the Run-in Period.
Participant milestones
| Measure |
Peripheral Deposition
Peripheral Deposition is achieved with a breathing maneuver consisting of slow and deep breaths
|
Bronchial Deposition
Bronchial Deposition consists of inhalation of smaller volumes per breath at higher flow rates
|
|---|---|---|
|
Overall Study
STARTED
|
37
|
35
|
|
Overall Study
COMPLETED
|
28
|
28
|
|
Overall Study
NOT COMPLETED
|
9
|
7
|
Reasons for withdrawal
| Measure |
Peripheral Deposition
Peripheral Deposition is achieved with a breathing maneuver consisting of slow and deep breaths
|
Bronchial Deposition
Bronchial Deposition consists of inhalation of smaller volumes per breath at higher flow rates
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
|
Overall Study
Adverse Event
|
1
|
1
|
|
Overall Study
Protocol Violation
|
4
|
2
|
|
Overall Study
No sputum, devise issues, exacerbation
|
3
|
4
|
Baseline Characteristics
Deposition of Inhaled Prolastin in Cystic Fibrosis Patients
Baseline characteristics by cohort
| Measure |
Peripheral Deposition
n=37 Participants
Peripheral Deposition is achieved with a breathing maneuver consisting of slow and deep breaths
|
Bronchial Deposition
n=35 Participants
Bronchial Deposition consists of inhalation of smaller volumes per breath at higher flow rates
|
Total
n=72 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
10 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
27 Participants
n=5 Participants
|
25 Participants
n=7 Participants
|
52 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
|
23.78 years
STANDARD_DEVIATION 7.54 • n=5 Participants
|
27.42 years
STANDARD_DEVIATION 9.78 • n=7 Participants
|
25.46 years
STANDARD_DEVIATION 8.84 • n=5 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
40 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
32 Participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
37 participants
n=5 Participants
|
35 participants
n=7 Participants
|
72 participants
n=5 Participants
|
|
Free Elastase during the Run-In Phase
Week 0
|
36.6 µg/mL
STANDARD_DEVIATION 35.9 • n=5 Participants
|
35.9 µg/mL
STANDARD_DEVIATION 38.8 • n=7 Participants
|
36.2 µg/mL
STANDARD_DEVIATION 36.9 • n=5 Participants
|
|
Free Elastase during the Run-In Phase
Week 2
|
32.7 µg/mL
STANDARD_DEVIATION 31.7 • n=5 Participants
|
25.0 µg/mL
STANDARD_DEVIATION 20.1 • n=7 Participants
|
29.1 µg/mL
STANDARD_DEVIATION 27.0 • n=5 Participants
|
|
Free Elastase during the Run-In Phase
Absolute Change
|
-4.0 µg/mL
STANDARD_DEVIATION 21.3 • n=5 Participants
|
-10.9 µg/mL
STANDARD_DEVIATION 36.9 • n=7 Participants
|
-7.25 µg/mL
STANDARD_DEVIATION 29.6 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline vs Week 4Population: Modified Intent-to-Treat Population (mITT) was all randomized subjects who received any amount of study medication and had at least one evaluation of the primary efficacy variable (free elastase in induced sputum) post baseline and at baseline (Visit 2).
Outcome measures
| Measure |
Peripheral Deposition
n=28 Participants
Peripheral Deposition is achieved with a breathing maneuver consisting of slow and deep breaths
|
Bronchial Deposition
n=24 Participants
Bronchial Deposition consists of inhalation of smaller volumes per breath at higher flow rates
|
|---|---|---|
|
Change in Free Elastase in Induced Sputum From Baseline to Week 4
|
-7.42 ug/mL
Standard Deviation 29.96
|
6.11 ug/mL
Standard Deviation 31.30
|
SECONDARY outcome
Timeframe: Baseline vs Week 4Population: For Group 1, only 24 out of 28 subjects had applicable data to analyze. For Group 2, only 27 out of 28 subjects had applicable data to analyze.
Outcome measures
| Measure |
Peripheral Deposition
n=24 Participants
Peripheral Deposition is achieved with a breathing maneuver consisting of slow and deep breaths
|
Bronchial Deposition
n=27 Participants
Bronchial Deposition consists of inhalation of smaller volumes per breath at higher flow rates
|
|---|---|---|
|
Change in Alpha-1-anti-trypsin (A1AT) Activity in Induced Sputum From Baseline at Week 4
|
16741 ug/mL
Standard Deviation 33583
|
6469 ug/mL
Standard Deviation 18999
|
SECONDARY outcome
Timeframe: Baseline vs Week 4Population: For Group 1, only 24 out of 28 subjects had applicable data to analyze. For Group 2, only 26 out of 28 subjects had applicable data to analyze.
Outcome measures
| Measure |
Peripheral Deposition
n=24 Participants
Peripheral Deposition is achieved with a breathing maneuver consisting of slow and deep breaths
|
Bronchial Deposition
n=26 Participants
Bronchial Deposition consists of inhalation of smaller volumes per breath at higher flow rates
|
|---|---|---|
|
Change in Total Immunoglobulin G (IgG) Fragments in Induced Sputum From Baseline at Week 4
|
0.005 ug/mL
Standard Deviation 1.10
|
-0.48 ug/mL
Standard Deviation 1.53
|
SECONDARY outcome
Timeframe: Week 4Population: For Group 1, only 24 out of 28 subjects had applicable data to analyze. For Group 2, all 28 subjects had applicable data to analyze.
Outcome measures
| Measure |
Peripheral Deposition
n=24 Participants
Peripheral Deposition is achieved with a breathing maneuver consisting of slow and deep breaths
|
Bronchial Deposition
n=28 Participants
Bronchial Deposition consists of inhalation of smaller volumes per breath at higher flow rates
|
|---|---|---|
|
Change in Total Bacterial Load in Induced Sputum From Baseline to Week 4
|
22673275.8 CFU/g
Standard Deviation 253455845.23
|
5021353.2 CFU/g
Standard Deviation 130686274.77
|
SECONDARY outcome
Timeframe: Baseline vs Week 4Population: For Group 1, only 24 out of 28 subjects had applicable data to analyze. For Group 2, all 28 subjects had applicable data to analyze.
Outcome measures
| Measure |
Peripheral Deposition
n=24 Participants
Peripheral Deposition is achieved with a breathing maneuver consisting of slow and deep breaths
|
Bronchial Deposition
n=28 Participants
Bronchial Deposition consists of inhalation of smaller volumes per breath at higher flow rates
|
|---|---|---|
|
Change in Pseudomonas Load in Induced Sputum From Baseline at Week 4
|
-40.1 CFU/g
Standard Deviation 808.2
|
-22.3 CFU/g
Standard Deviation 772.4
|
SECONDARY outcome
Timeframe: Baseline vs Week 4Population: For Group 1, only 21 out of 28 subjects had applicable data to analyze. For Group 2, only 22 out of 28 subjects had applicable data to analyze.
Outcome measures
| Measure |
Peripheral Deposition
n=21 Participants
Peripheral Deposition is achieved with a breathing maneuver consisting of slow and deep breaths
|
Bronchial Deposition
n=22 Participants
Bronchial Deposition consists of inhalation of smaller volumes per breath at higher flow rates
|
|---|---|---|
|
Change in Neutrophil Number in Induced Sputum From Baseline at Week 4
|
-20.8 percentage of change
Standard Deviation 30.3
|
-8.2 percentage of change
Standard Deviation 20.9
|
Adverse Events
Peripheral Deposition
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Bronchial Deposition
Serious events: 0 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Peripheral Deposition
n=37 participants at risk
Peripheral Deposition is achieved with a breathing maneuver consisting of slow and deep breaths
|
Bronchial Deposition
n=35 participants at risk
Bronchial Deposition consists of inhalation of smaller volumes per breath at higher flow rates
|
|---|---|---|
|
Gastrointestinal disorders
Gastrointestinal Pain
|
2.7%
1/37
|
0.00%
0/35
|
|
Eye disorders
Conjunctivitis Allergic
|
0.00%
0/37
|
2.9%
1/35
|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/37
|
2.9%
1/35
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/37
|
2.9%
1/35
|
|
Gastrointestinal disorders
Gingivitis
|
0.00%
0/37
|
2.9%
1/35
|
|
Gastrointestinal disorders
Toothache
|
2.7%
1/37
|
0.00%
0/35
|
|
General disorders
Fatigue
|
0.00%
0/37
|
5.7%
2/35
|
|
General disorders
Influenza like illness
|
2.7%
1/37
|
0.00%
0/35
|
|
General disorders
Pyrexia
|
2.7%
1/37
|
0.00%
0/35
|
|
Infections and infestations
Nasopharyngitis
|
2.7%
1/37
|
14.3%
5/35
|
|
Infections and infestations
Sinusitis
|
0.00%
0/37
|
2.9%
1/35
|
|
Infections and infestations
Urinary Tract Infection
|
2.7%
1/37
|
0.00%
0/35
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.4%
2/37
|
0.00%
0/35
|
|
Psychiatric disorders
Sleep disorder
|
2.7%
1/37
|
0.00%
0/35
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/37
|
2.9%
1/35
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
2.7%
1/37
|
0.00%
0/35
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
2.7%
1/37
|
5.7%
2/35
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
2.7%
1/37
|
0.00%
0/35
|
|
Vascular disorders
Vasculitis
|
2.7%
1/37
|
0.00%
0/35
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The investigator must send a draft manuscript of the publication or abstract to the sponsor thirty days in advance of submission in order to obtain approval prior to submission of the final version for publication. This will be reviewed promptly and approval will not be withheld unreasonably. In case of a difference of opinion between the sponsor and the Investigator(s), the contents of the publication will be discussed in order to find a solution which satisfies both parties.
- Publication restrictions are in place
Restriction type: OTHER