Trial Outcomes & Findings for An Efficacy and Safety Study of SPX-101 Inhalation Solution in Subjects With Cystic Fibrosis (NCT NCT03229252)
NCT ID: NCT03229252
Last Updated: 2019-12-30
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
91 participants
Primary outcome timeframe
Baseline and Day 28
Results posted on
2019-12-30
Participant Flow
Participant milestones
| Measure |
Placebo
Placebo Inhalation solution twice daily for 28 days.
Placebo Inhalation Solution: Normal Saline Inhalation Solution
|
SPX-101 Low Dose (60mg BID)
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
SPX-101 High Dose (120mg BID)
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
|---|---|---|---|
|
Overall Study
STARTED
|
31
|
15
|
45
|
|
Overall Study
COMPLETED
|
29
|
15
|
39
|
|
Overall Study
NOT COMPLETED
|
2
|
0
|
6
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
An Efficacy and Safety Study of SPX-101 Inhalation Solution in Subjects With Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Placebo
n=31 Participants
Placebo Inhalation solution twice daily for 28 days.
Placebo Inhalation Solution: Normal Saline Inhalation Solution
|
SPX-101 Low Dose (60mg BID)
n=15 Participants
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
SPX-101 High Dose (120mg BID)
n=45 Participants
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
Total
n=91 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
31 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
45 Participants
n=5 Participants
|
91 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Continuous
|
30.8 years
STANDARD_DEVIATION 7.82 • n=5 Participants
|
33.7 years
STANDARD_DEVIATION 8.62 • n=7 Participants
|
31.9 years
STANDARD_DEVIATION 8.49 • n=5 Participants
|
31.8 years
STANDARD_DEVIATION 8.25 • n=4 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
22 Participants
n=5 Participants
|
37 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
54 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
29 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
43 Participants
n=5 Participants
|
86 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
26 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
44 Participants
n=5 Participants
|
82 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Region of Enrollment
Canada
|
3 participants
n=5 Participants
|
5 participants
n=7 Participants
|
8 participants
n=5 Participants
|
16 participants
n=4 Participants
|
|
Region of Enrollment
Italy
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
3 participants
n=5 Participants
|
4 participants
n=4 Participants
|
|
Region of Enrollment
United Kingdom
|
14 participants
n=5 Participants
|
7 participants
n=7 Participants
|
15 participants
n=5 Participants
|
36 participants
n=4 Participants
|
|
Region of Enrollment
France
|
12 participants
n=5 Participants
|
2 participants
n=7 Participants
|
13 participants
n=5 Participants
|
27 participants
n=4 Participants
|
|
Region of Enrollment
Portugal
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
6 participants
n=5 Participants
|
8 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Baseline and Day 28Outcome measures
| Measure |
Placebo
n=31 Participants
Placebo Inhalation solution twice daily for 28 days.
Placebo Inhalation Solution: Normal Saline Inhalation Solution
|
SPX-101 Low Dose (60mg BID)
n=15 Participants
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
SPX-101 High Dose (120mg BID)
n=45 Participants
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
|---|---|---|---|
|
Change in Percent Predicted FEV1
|
1.633 change from baseline in ppFEV1
Standard Deviation 6.3302
|
0.800 change from baseline in ppFEV1
Standard Deviation 6.5049
|
0.890 change from baseline in ppFEV1
Standard Deviation 6.7061
|
SECONDARY outcome
Timeframe: Day 1 through Day 28Outcome measures
| Measure |
Placebo
n=31 Participants
Placebo Inhalation solution twice daily for 28 days.
Placebo Inhalation Solution: Normal Saline Inhalation Solution
|
SPX-101 Low Dose (60mg BID)
n=15 Participants
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
SPX-101 High Dose (120mg BID)
n=45 Participants
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
|---|---|---|---|
|
Number of Participants With Adverse Events
|
20 Participants
|
11 Participants
|
30 Participants
|
SECONDARY outcome
Timeframe: Day 1 through Day 28Chemistry, Hematology, Urinalysis
Outcome measures
| Measure |
Placebo
n=31 Participants
Placebo Inhalation solution twice daily for 28 days.
Placebo Inhalation Solution: Normal Saline Inhalation Solution
|
SPX-101 Low Dose (60mg BID)
n=15 Participants
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
SPX-101 High Dose (120mg BID)
n=45 Participants
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
|---|---|---|---|
|
Change From Baseline Through Day 28 in Clinical Laboratory Tests
Blood Electrolytes (Potassium) Low
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Change From Baseline Through Day 28 in Clinical Laboratory Tests
Blood Electrolytes (Potassium) Normal
|
29 Participants
|
13 Participants
|
40 Participants
|
|
Change From Baseline Through Day 28 in Clinical Laboratory Tests
Blood Electrolytes (Potassium) High
|
0 Participants
|
1 Participants
|
0 Participants
|
Adverse Events
Placebo
Serious events: 1 serious events
Other events: 20 other events
Deaths: 0 deaths
SPX-101 Low Dose (60mg BID)
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
SPX-101 High Dose (120mg BID)
Serious events: 3 serious events
Other events: 29 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=31 participants at risk
Placebo Inhalation solution twice daily for 28 days.
Placebo Inhalation Solution: Normal Saline Inhalation Solution
|
SPX-101 Low Dose (60mg BID)
n=15 participants at risk
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
SPX-101 High Dose (120mg BID)
n=45 participants at risk
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
|---|---|---|---|
|
Infections and infestations
Infective Pulmonary Exacerbation of Cystic Fibrosis
|
0.00%
0/31 • 28 Days
|
0.00%
0/15 • 28 Days
|
2.2%
1/45 • Number of events 1 • 28 Days
|
|
Gastrointestinal disorders
Intestinal Obstuction
|
3.2%
1/31 • Number of events 1 • 28 Days
|
0.00%
0/15 • 28 Days
|
0.00%
0/45 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Cough and Dyspnea
|
0.00%
0/31 • 28 Days
|
0.00%
0/15 • 28 Days
|
2.2%
1/45 • Number of events 1 • 28 Days
|
|
Cardiac disorders
Tachycardia
|
0.00%
0/31 • 28 Days
|
0.00%
0/15 • 28 Days
|
2.2%
1/45 • Number of events 1 • 28 Days
|
Other adverse events
| Measure |
Placebo
n=31 participants at risk
Placebo Inhalation solution twice daily for 28 days.
Placebo Inhalation Solution: Normal Saline Inhalation Solution
|
SPX-101 Low Dose (60mg BID)
n=15 participants at risk
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
SPX-101 High Dose (120mg BID)
n=45 participants at risk
Inhalation solution twice daily for 28 days.
SPX-101: SPX-101 Inhalation Solution
|
|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
19.4%
6/31 • Number of events 6 • 28 Days
|
26.7%
4/15 • Number of events 4 • 28 Days
|
13.3%
6/45 • Number of events 6 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Sputum Increased
|
6.5%
2/31 • Number of events 2 • 28 Days
|
20.0%
3/15 • Number of events 3 • 28 Days
|
8.9%
4/45 • Number of events 4 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.00%
0/31 • 28 Days
|
6.7%
1/15 • Number of events 2 • 28 Days
|
8.9%
4/45 • Number of events 7 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
0.00%
0/31 • 28 Days
|
0.00%
0/15 • 28 Days
|
11.1%
5/45 • Number of events 5 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
3.2%
1/31 • Number of events 1 • 28 Days
|
0.00%
0/15 • 28 Days
|
8.9%
4/45 • Number of events 4 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/31 • 28 Days
|
6.7%
1/15 • Number of events 1 • 28 Days
|
4.4%
2/45 • Number of events 2 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
0.00%
0/31 • 28 Days
|
20.0%
3/15 • Number of events 3 • 28 Days
|
0.00%
0/45 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Lower Respiratory Tract Congestion
|
6.5%
2/31 • Number of events 2 • 28 Days
|
0.00%
0/15 • 28 Days
|
0.00%
0/45 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Increased Viscosity of Bronchial Secretion
|
0.00%
0/31 • 28 Days
|
6.7%
1/15 • Number of events 1 • 28 Days
|
0.00%
0/45 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Sputum Discolored
|
0.00%
0/31 • 28 Days
|
6.7%
1/15 • Number of events 1 • 28 Days
|
0.00%
0/45 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Infective Pulmonary Exacerbation of Cystic Fibrosis
|
12.9%
4/31 • Number of events 4 • 28 Days
|
6.7%
1/15 • Number of events 1 • 28 Days
|
6.7%
3/45 • Number of events 3 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Nasopharyngitis
|
6.5%
2/31 • Number of events 2 • 28 Days
|
0.00%
0/15 • 28 Days
|
6.7%
3/45 • Number of events 3 • 28 Days
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Tract Infection
|
0.00%
0/31 • 28 Days
|
13.3%
2/15 • Number of events 2 • 28 Days
|
0.00%
0/45 • 28 Days
|
|
Gastrointestinal disorders
Diarrhea
|
3.2%
1/31 • Number of events 1 • 28 Days
|
6.7%
1/15 • Number of events 1 • 28 Days
|
4.4%
2/45 • Number of events 2 • 28 Days
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/31 • 28 Days
|
6.7%
1/15 • Number of events 1 • 28 Days
|
4.4%
2/45 • Number of events 2 • 28 Days
|
|
Gastrointestinal disorders
Frequent Bowel Movements
|
0.00%
0/31 • 28 Days
|
6.7%
1/15 • Number of events 1 • 28 Days
|
0.00%
0/45 • 28 Days
|
|
Gastrointestinal disorders
Tongue Coated
|
0.00%
0/31 • 28 Days
|
6.7%
1/15 • Number of events 1 • 28 Days
|
0.00%
0/45 • 28 Days
|
|
Nervous system disorders
Headache
|
3.2%
1/31 • Number of events 1 • 28 Days
|
0.00%
0/15 • 28 Days
|
8.9%
4/45 • Number of events 4 • 28 Days
|
|
Nervous system disorders
Dysgeusia
|
6.5%
2/31 • Number of events 2 • 28 Days
|
0.00%
0/15 • 28 Days
|
0.00%
0/45 • 28 Days
|
|
Nervous system disorders
Migraine
|
0.00%
0/31 • 28 Days
|
6.7%
1/15 • Number of events 1 • 28 Days
|
0.00%
0/45 • 28 Days
|
|
General disorders
Chest Discomfort
|
6.5%
2/31 • Number of events 2 • 28 Days
|
13.3%
2/15 • Number of events 2 • 28 Days
|
4.4%
2/45 • Number of events 3 • 28 Days
|
|
General disorders
Fatigue
|
6.5%
2/31 • Number of events 2 • 28 Days
|
0.00%
0/15 • 28 Days
|
0.00%
0/45 • 28 Days
|
|
General disorders
Pyrexia
|
0.00%
0/31 • 28 Days
|
6.7%
1/15 • Number of events 1 • 28 Days
|
0.00%
0/45 • 28 Days
|
|
Investigations
Forced Expiratory Volume Decreased
|
0.00%
0/31 • 28 Days
|
0.00%
0/15 • 28 Days
|
8.9%
4/45 • Number of events 4 • 28 Days
|
|
Skin and subcutaneous tissue disorders
Eczema
|
0.00%
0/31 • 28 Days
|
6.7%
1/15 • Number of events 1 • 28 Days
|
0.00%
0/45 • 28 Days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place