Trial Outcomes & Findings for Safety and Tolerability Trial of Inhaled Alpha1-Proteinase Inhibitor (Human), Hydrophobic Chromatography Process (Alpha-1 HC) in Subjects With Cystic Fibrosis (NCT NCT01684410)
NCT ID: NCT01684410
Last Updated: 2016-02-08
Results Overview
adverse event frequency
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
3 weeks
Results posted on
2016-02-08
Participant Flow
A total of 41 subjects provided informed consent and were screened for the study. Eleven (11) subjects were screen failures, and a total of 30 subjects were randomized to one of three treatment groups: 200 mg or 100 mg of Alpha-1 HC or placebo daily.
Participant milestones
| Measure |
Alpha-1 HC 100 mg
100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Alpha-1 HC 200 mg
200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Placebo
Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate).
Placebo
|
|---|---|---|---|
|
Overall Study
STARTED
|
10
|
10
|
10
|
|
Overall Study
COMPLETED
|
10
|
10
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Tolerability Trial of Inhaled Alpha1-Proteinase Inhibitor (Human), Hydrophobic Chromatography Process (Alpha-1 HC) in Subjects With Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Alpha-1 HC 100 mg
n=10 Participants
100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Alpha-1 HC 200 mg
n=10 Participants
200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Placebo
n=10 Participants
Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate).
Placebo
|
Total
n=30 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
28.2 years
STANDARD_DEVIATION 10.62 • n=5 Participants
|
28.1 years
STANDARD_DEVIATION 11.43 • n=7 Participants
|
29.3 years
STANDARD_DEVIATION 9.96 • n=5 Participants
|
28.5 years
STANDARD_DEVIATION 10.32 • n=4 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
20 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
10 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
10 participants
n=5 Participants
|
10 participants
n=7 Participants
|
10 participants
n=5 Participants
|
30 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 3 weeksPopulation: Safety Population: included all subjects who received any dose of IP (included those withdrawn from treatment for any reason)
adverse event frequency
Outcome measures
| Measure |
Alpha-1 HC 100 mg
n=10 Participants
100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Alpha-1 HC 200 mg
n=10 Participants
200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Placebo
n=10 Participants
Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate).
Placebo
|
|---|---|---|---|
|
Adverse Events
|
100 percentage of participants
|
80 percentage of participants
|
60 percentage of participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 3 weeksPopulation: Safety Population: included all subjects who received any dose of Investigational Product (included those withdrawn from treatment for any reason)
FEV1 conducted before and after inhalation of the investigational product at study visits.
Outcome measures
| Measure |
Alpha-1 HC 100 mg
n=10 Participants
100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Alpha-1 HC 200 mg
n=10 Participants
200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Placebo
n=10 Participants
Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate).
Placebo
|
|---|---|---|---|
|
Percent Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Week 3
|
1.5 percent
Standard Deviation 5.20
|
-2.1 percent
Standard Deviation 12.28
|
0.5 percent
Standard Deviation 5.91
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 3 weeksPopulation: Safety Population: included all subjects who received any dose of Investigational Product (included those withdrawn from treatment for any reason)
FVC conducted before and after inhalation of the investigational product
Outcome measures
| Measure |
Alpha-1 HC 100 mg
n=10 Participants
100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Alpha-1 HC 200 mg
n=10 Participants
200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Placebo
n=10 Participants
Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate).
Placebo
|
|---|---|---|---|
|
Percent Change From Baseline in Forced Vital Capacity (FVC) at Week 3
|
1.2 percent
Standard Deviation 5.23
|
-2.3 percent
Standard Deviation 11.57
|
-0.9 percent
Standard Deviation 3.73
|
Adverse Events
Alpha-1 HC 100 mg
Serious events: 1 serious events
Other events: 10 other events
Deaths: 0 deaths
Alpha-1 HC 200 mg
Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths
Placebo
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Alpha-1 HC 100 mg
n=10 participants at risk
100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Alpha-1 HC 200 mg
n=10 participants at risk
200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Placebo
n=10 participants at risk
Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate).
Placebo
|
|---|---|---|---|
|
Gastrointestinal disorders
ABDOMINAL PAIN
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
PULMONARY EXACERBATION/
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
Other adverse events
| Measure |
Alpha-1 HC 100 mg
n=10 participants at risk
100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Alpha-1 HC 200 mg
n=10 participants at risk
200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.
Alpha-1 HC: Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma.
|
Placebo
n=10 participants at risk
Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate).
Placebo
|
|---|---|---|---|
|
Gastrointestinal disorders
MOUTH ULCERATION
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Gastrointestinal disorders
VOMITING
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
General disorders
CHEST DISCOMFORT
|
0.00%
0/10 • 3 weeks
|
20.0%
2/10 • Number of events 2 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
General disorders
FATIGUE
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Infections and infestations
ORAL HERPES
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Infections and infestations
SINUSITIS
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Investigations
BLOOD CREATININE INCREASED
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Investigations
BLOOD PRESSURE INCREASED
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Investigations
BREATH SOUNDS ABNORMAL
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Investigations
C-REACTIVE PROTEIN INCREASED
|
0.00%
0/10 • 3 weeks
|
20.0%
2/10 • Number of events 2 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Investigations
FORCED EXPIRATORY VOLUME DECREASED
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Investigations
PHYSICAL EXAMINATION ABNORMAL
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Investigations
PULMONARY FUNCTION TEST DECREASED
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Investigations
SPUTUM ABNORMAL
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Metabolism and nutrition disorders
DECREASED APPETITE
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Musculoskeletal and connective tissue disorders
FLANK PAIN
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Nervous system disorders
DIZZINESS
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Nervous system disorders
SINUS HEADACHE
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Psychiatric disorders
DEPRESSION
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Reproductive system and breast disorders
ENDOMETRIOSIS
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
BRONCHIAL SECRETION RETENTION
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
BRONCHOSPASM
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
30.0%
3/10 • Number of events 3 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
DRY THROAT
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
HAEMOPTYSIS
|
40.0%
4/10 • Number of events 4 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
OROPHARYNGEAL PAIN
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
20.0%
2/10 • Number of events 2 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
PHARYNGEAL ERYTHEMA
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
PHARYNGEAL OEDEMA
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
PULMONARY CONGESTION
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
PULMONARY EXACERBATION
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
30.0%
3/10 • Number of events 3 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
RALES
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
RESPIRATORY TRACT IRRITATION
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
RHINITIS ALLERGIC
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
RHONCHI
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
SPUTUM INCREASED
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
THROAT IRRITATION
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Respiratory, thoracic and mediastinal disorders
WHEEZING
|
30.0%
3/10 • Number of events 3 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Skin and subcutaneous tissue disorders
ALOPECIA
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Skin and subcutaneous tissue disorders
PHOTOSENSITIVITY REACTION
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Skin and subcutaneous tissue disorders
RASH MACULAR
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Gastrointestinal disorders
ABDOMINAL PAIN UPPER
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
General disorders
CHEST PAIN
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
General disorders
CHILLS
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
General disorders
PAIN
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
|
General disorders
PYREXIA
|
0.00%
0/10 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
20.0%
2/10 • Number of events 2 • 3 weeks
|
|
Infections and infestations
OTITIS EXTERNA
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
|
Infections and infestations
OTITIS MEDIA
|
0.00%
0/10 • 3 weeks
|
10.0%
1/10 • Number of events 1 • 3 weeks
|
0.00%
0/10 • 3 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Site may publish results from the Study, after providing Sponsor thirty days' notice prior to submitting a manuscript or other materials related to the Study to any outside party. At Sponsors' request, Site will remove any Confidential Information (other than Study results), and Site will upon Sponsors' request, delay publication or presentation for a period of up to one hundred twenty days to allow Sponsor to protect its interests in any Sponsor Inventions.
- Publication restrictions are in place
Restriction type: OTHER