Trial Outcomes & Findings for Phase II Study of Digitoxin to Treat Cystic Fibrosis (NCT NCT00782288)
NCT ID: NCT00782288
Last Updated: 2022-04-21
Results Overview
The Il-8 measurements of sputum digitoxin levels for each group is shown for 5 days (Days 1, 14, 21, 28 and 42).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
24 participants
Primary outcome timeframe
42 days (Day 1 to Day 42)
Results posted on
2022-04-21
Participant Flow
All participants were recruited from the Johns Hopkins University in Baltimore, Maryland. The first participant was enrolled on October 13, 2010 and the last participant was enrolled on May 6, 2014. All study visits were completed as of June 2014. There was a DSMB safety review prior to enrolling any subjects into the higher dose group (0.1 mg).
Participants were all consented on a Johns Hopkins IRB approved consent form and screened 2 or 4 weeks prior to enrollment. Enrollment was contingent on meeting all inclusion/exclusion criteria.
Participant milestones
| Measure |
Placebo
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05mg Digitoxin
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Overall Study
STARTED
|
8
|
8
|
8
|
|
Overall Study
COMPLETED
|
8
|
8
|
8
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase II Study of Digitoxin to Treat Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=8 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=8 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
Total
n=24 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=93 Participants
|
8 Participants
n=4 Participants
|
8 Participants
n=27 Participants
|
24 Participants
n=483 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Age, Continuous
|
24.8 years
n=93 Participants
|
23.5 years
n=4 Participants
|
30.3 years
n=27 Participants
|
26.2 years
n=483 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=93 Participants
|
5 Participants
n=4 Participants
|
4 Participants
n=27 Participants
|
13 Participants
n=483 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
4 Participants
n=27 Participants
|
11 Participants
n=483 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=93 Participants
|
8 Participants
n=4 Participants
|
8 Participants
n=27 Participants
|
24 Participants
n=483 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Region of Enrollment
United States
|
8 participants
n=93 Participants
|
8 participants
n=4 Participants
|
8 participants
n=27 Participants
|
24 participants
n=483 Participants
|
PRIMARY outcome
Timeframe: 42 days (Day 1 to Day 42)Population: Sputum was collected for Il-8 biomarkers on Days 1,14, 21, 28 during the treatment phase and at Day 42. Measures were compared between Baseline, Treatment and Recovery periods to determine if changes from baseline differed between placebo and the two doses.
The Il-8 measurements of sputum digitoxin levels for each group is shown for 5 days (Days 1, 14, 21, 28 and 42).
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=8 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=8 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Effect of Digitoxin on IL-8 (Interleukin 8) in Induced Sputum in Stable Cystic Fibrosis (CF) Patients.
IL-8 Day 21
|
5.08 log 10 (pg/mL)
Interval 4.21 to 5.38
|
4.77 log 10 (pg/mL)
Interval 4.64 to 4.89
|
5.17 log 10 (pg/mL)
Interval 4.66 to 5.42
|
|
Effect of Digitoxin on IL-8 (Interleukin 8) in Induced Sputum in Stable Cystic Fibrosis (CF) Patients.
IL-8 Day 28
|
4.66 log 10 (pg/mL)
Interval 4.49 to 5.38
|
4.79 log 10 (pg/mL)
Interval 4.49 to 4.91
|
5.20 log 10 (pg/mL)
Interval 4.68 to 5.34
|
|
Effect of Digitoxin on IL-8 (Interleukin 8) in Induced Sputum in Stable Cystic Fibrosis (CF) Patients.
IL-8 Day 42
|
4.59 log 10 (pg/mL)
Interval 4.22 to 5.06
|
4.74 log 10 (pg/mL)
Interval 4.62 to 5.04
|
4.97 log 10 (pg/mL)
Interval 4.43 to 5.25
|
|
Effect of Digitoxin on IL-8 (Interleukin 8) in Induced Sputum in Stable Cystic Fibrosis (CF) Patients.
IL-8 Day 1
|
4.59 log 10 (pg/mL)
Interval 4.15 to 5.12
|
4.96 log 10 (pg/mL)
Interval 4.4 to 5.18
|
5.04 log 10 (pg/mL)
Interval 4.58 to 5.1
|
|
Effect of Digitoxin on IL-8 (Interleukin 8) in Induced Sputum in Stable Cystic Fibrosis (CF) Patients.
IL-8 Day 14
|
4.94 log 10 (pg/mL)
Interval 4.21 to 5.34
|
4.89 log 10 (pg/mL)
Interval 4.5 to 5.01
|
5.01 log 10 (pg/mL)
Interval 4.66 to 5.45
|
PRIMARY outcome
Timeframe: 28 days (Day 28 minus Day 1)Population: For change in Il-8 from Day 28 minus Day 1, a Kruskal-Wallis equality of populations rank was performed.
Change in Il-8 values are expressed as a change in log 10 Il-8 pg/mL from Day 28 minus Day 1.
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=8 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=8 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Change in Il-8 (Interleukin 8) Levels From Day 28 Minus Day 1 (Treatment Period).
|
0.13 log10 (pg/mL)
Interval 0.06 to 0.4
|
-0.13 log10 (pg/mL)
Interval -0.23 to 0.16
|
0.04 log10 (pg/mL)
Interval -0.24 to 0.41
|
PRIMARY outcome
Timeframe: 42 days (Day 1- Day 42)Population: Neutrophil cell counts were compared between Baseline, Treatment and Recovery periods to determine if changes from baseline differed between the placebo and the two digitoxin dose levels.
The neutrophil counts were measured in the induced sputum of participants in each group on study 5 days (Days 1, 14, 21, 28 and 42).
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=7 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=7 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Effect of Digitoxin on Neutrophil Counts in Induced Sputum in Stable Cystic Fibrosis (CF) Patients.
Day 1
|
2.58 log 10 (neutrophil cells/mL)
Interval 1.89 to 3.0
|
2.82 log 10 (neutrophil cells/mL)
Interval 1.88 to 3.08
|
2.93 log 10 (neutrophil cells/mL)
Interval 2.35 to 3.04
|
|
Effect of Digitoxin on Neutrophil Counts in Induced Sputum in Stable Cystic Fibrosis (CF) Patients.
Day 14
|
2.89 log 10 (neutrophil cells/mL)
Interval 2.18 to 2.92
|
2.26 log 10 (neutrophil cells/mL)
Interval 1.35 to 2.91
|
2.71 log 10 (neutrophil cells/mL)
Interval 2.26 to 3.1
|
|
Effect of Digitoxin on Neutrophil Counts in Induced Sputum in Stable Cystic Fibrosis (CF) Patients.
Day 21
|
2.86 log 10 (neutrophil cells/mL)
Interval 2.44 to 3.35
|
2.23 log 10 (neutrophil cells/mL)
Interval 1.52 to 2.62
|
2.88 log 10 (neutrophil cells/mL)
Interval 1.9 to 3.14
|
|
Effect of Digitoxin on Neutrophil Counts in Induced Sputum in Stable Cystic Fibrosis (CF) Patients.
Day 28
|
2.71 log 10 (neutrophil cells/mL)
Interval 2.16 to 3.06
|
2.16 log 10 (neutrophil cells/mL)
Interval 0.83 to 2.64
|
2.77 log 10 (neutrophil cells/mL)
Interval 1.97 to 3.01
|
|
Effect of Digitoxin on Neutrophil Counts in Induced Sputum in Stable Cystic Fibrosis (CF) Patients.
Day 42
|
2.55 log 10 (neutrophil cells/mL)
Interval 2.27 to 2.81
|
2.06 log 10 (neutrophil cells/mL)
Interval 1.58 to 2.35
|
2.82 log 10 (neutrophil cells/mL)
Interval 2.0 to 2.96
|
PRIMARY outcome
Timeframe: 28 days (Day 28 minus Day 1)Population: The change in neutrophil cell count from Day 1 to Day 28 was performed using a Kruskal-Wallis equality of populations rank test. The cells counts are expressed as log 10 (neutrophil cell/mL).
The change in log 10 neutrophil cell count from Day 28 minus Day 1 (during the treatment period) expressed as log (10\^4 neutrophil/mL).
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=7 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=7 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Change in Neutrophil Cell Count Day 28 Minus Day 1 (Treatment Period).
|
0.19 log 10 (neutrophil cell/mL)
Interval -0.24 to 0.82
|
-0.26 log 10 (neutrophil cell/mL)
Interval -0.41 to 0.29
|
-0.02 log 10 (neutrophil cell/mL)
Interval -0.33 to 0.26
|
SECONDARY outcome
Timeframe: Serum PK on Days 1 (pre-dose), 7, 14, 21 and 42Population: Subjects had serum digitoxin levels drawn at Day 1 (pre-dose) with no levels observed, as expected. Digitoxin was drawn on Day 7, 14, 21 and 42 to determine the number of subjects with a detectable level of digitoxin (ng/mL) in serum by group.
Digitoxin serum levels were drawn on Days 1 (pre-dose), 7, 14, 21 and 42. The range of digitoxin levels for each visit is listed and the number of subjects who had that level are marked by group (placebo, low dose and high dose).
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=8 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=8 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 7, digitoxin level 6.9 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 7, digitoxin level 5.6 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 7, digitoxin level 6 ng/mL
|
0 participants
|
1 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 7, digitoxin level 6.4 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 7, digitoxin level 7.8 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 7, digitoxin level none detected
|
8 participants
|
7 participants
|
3 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 14, digitoxin level 10 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 14, digitoxin level 11 ng/mL
|
0 participants
|
1 participants
|
0 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 14, digitoxin level 13 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 14, digitoxin level 6.8 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 14, digitoxin level 6.9 ng/mL
|
0 participants
|
1 participants
|
0 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 14, digitoxin level 7.3 ng/mL
|
0 participants
|
1 participants
|
0 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 14, digitoxin level 7.5 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 14, digitoxin level 8.1 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 14, digitoxin level 9.3 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 14, digitoxin level 9.4 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 14, digitoxin level none detected
|
8 participants
|
5 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 10 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 11 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 12 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 13 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 14 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 15 ng/mL
|
0 participants
|
1 participants
|
0 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 4 ng/mL
|
0 participants
|
1 participants
|
0 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 5 ng/mL
|
0 participants
|
1 participants
|
0 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 6 ng/mL
|
0 participants
|
1 participants
|
0 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 6.4 ng/mL
|
0 participants
|
1 participants
|
0 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 6.5 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 6.9 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level 7.5 ng/mL
|
0 participants
|
0 participants
|
1 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 21, digitoxin level none detected
|
8 participants
|
3 participants
|
0 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 42, digitoxin level 5 ng/mL
|
0 participants
|
1 participants
|
0 participants
|
|
Pharmacokinetics (PK) of Digitoxin in Serum in Stable CF Patients.
Day 42, digitoxin level none detected
|
8 participants
|
7 participants
|
8 participants
|
SECONDARY outcome
Timeframe: Baseline and Day 28Population: Median changes in FEV1 in L by group for the 28 days of treatment.
Safety indices included changes in FEV1 (forced expiratory volume in 1 second), changes in WBC (white blood cell count), alterations in ECG and sputum microbiology. The median changes in FEV1 are reported.
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=8 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=8 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Safety Indices Including Change in FEV1 in Stable CF Patients.
|
-0.00 liters
Interval -0.37 to 0.22
|
0.10 liters
Interval 0.05 to 0.13
|
-0.02 liters
Interval -0.14 to 0.07
|
SECONDARY outcome
Timeframe: Baseline and Day 42Population: Mean change in CFQ-R scores from Visit 1 to Visit 6, by domain.
CFQ-R is a disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being and symptoms. Developed specifically for use in patients with a diagnosis of cystic fibrosis. There are 9 Quality of life domains: physical, role/school, vitality, emotion, social, body image, eating, treatment burden, health perceptions and 3 symptom scales: weight, respiratory, and digestion. Scaling of items is done via 5 distinct 4-point Likert scales. Scores for each domain; after recoding, each item is summed to generate a domain score and standardized. Scores range from 0 to 100, with higher scores indicating better health. Based on clinician judgment of global clinical change, a moderate change was a standardized effect size of 0.50 units and an important change was a standardized effect size of 0.80 units evaluating pre- and post-treatment for CF exacerbation. Mean changes in CFQ-R Scores by Group were evaluated between Visit 6 and Visit 1, by Domain.
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=8 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=8 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Mean Change in Quality of Life Scores, for Each Domain, From Day 1 to Day 42 Using the Cystic Fibrosis Questionnaire Revised (CFQ-R).
Physical Domain
|
-5.21 mean change of scores on a scale
Standard Deviation 8.55
|
-14.58 mean change of scores on a scale
Standard Deviation 17.11
|
-2.60 mean change of scores on a scale
Standard Deviation 11.34
|
|
Mean Change in Quality of Life Scores, for Each Domain, From Day 1 to Day 42 Using the Cystic Fibrosis Questionnaire Revised (CFQ-R).
Role Domain
|
0.00 mean change of scores on a scale
Standard Deviation 7.72
|
-1.04 mean change of scores on a scale
Standard Deviation 5.34
|
1.04 mean change of scores on a scale
Standard Deviation 8.26
|
|
Mean Change in Quality of Life Scores, for Each Domain, From Day 1 to Day 42 Using the Cystic Fibrosis Questionnaire Revised (CFQ-R).
Vitality Domain
|
-2.08 mean change of scores on a scale
Standard Deviation 5.89
|
4.17 mean change of scores on a scale
Standard Deviation 6.30
|
1.04 mean change of scores on a scale
Standard Deviation 20.62
|
|
Mean Change in Quality of Life Scores, for Each Domain, From Day 1 to Day 42 Using the Cystic Fibrosis Questionnaire Revised (CFQ-R).
Emotional Domain
|
-5.83 mean change of scores on a scale
Standard Deviation 12.57
|
-5.00 mean change of scores on a scale
Standard Deviation 17.73
|
4.17 mean change of scores on a scale
Standard Deviation 13.30
|
|
Mean Change in Quality of Life Scores, for Each Domain, From Day 1 to Day 42 Using the Cystic Fibrosis Questionnaire Revised (CFQ-R).
Social Domain
|
-2.08 mean change of scores on a scale
Standard Deviation 9.36
|
-5.56 mean change of scores on a scale
Standard Deviation 6.64
|
-4.17 mean change of scores on a scale
Standard Deviation 15.92
|
|
Mean Change in Quality of Life Scores, for Each Domain, From Day 1 to Day 42 Using the Cystic Fibrosis Questionnaire Revised (CFQ-R).
Body Domain
|
6.94 mean change of scores on a scale
Standard Deviation 14.47
|
-4.17 mean change of scores on a scale
Standard Deviation 22.17
|
8.33 mean change of scores on a scale
Standard Deviation 11.50
|
|
Mean Change in Quality of Life Scores, for Each Domain, From Day 1 to Day 42 Using the Cystic Fibrosis Questionnaire Revised (CFQ-R).
Eating Domain
|
-1.39 mean change of scores on a scale
Standard Deviation 11.01
|
-1.39 mean change of scores on a scale
Standard Deviation 3.93
|
-1.39 mean change of scores on a scale
Standard Deviation 3.93
|
|
Mean Change in Quality of Life Scores, for Each Domain, From Day 1 to Day 42 Using the Cystic Fibrosis Questionnaire Revised (CFQ-R).
Treatment Domain
|
1.39 mean change of scores on a scale
Standard Deviation 9.27
|
6.94 mean change of scores on a scale
Standard Deviation 11.79
|
5.56 mean change of scores on a scale
Standard Deviation 8.40
|
|
Mean Change in Quality of Life Scores, for Each Domain, From Day 1 to Day 42 Using the Cystic Fibrosis Questionnaire Revised (CFQ-R).
Health Domain
|
1.39 mean change of scores on a scale
Standard Deviation 9.27
|
4.17 mean change of scores on a scale
Standard Deviation 5.75
|
-4.17 mean change of scores on a scale
Standard Deviation 11.79
|
|
Mean Change in Quality of Life Scores, for Each Domain, From Day 1 to Day 42 Using the Cystic Fibrosis Questionnaire Revised (CFQ-R).
Weight Domain
|
0.00 mean change of scores on a scale
Standard Deviation 0.00
|
12.50 mean change of scores on a scale
Standard Deviation 17.25
|
-0.00 mean change of scores on a scale
Standard Deviation 17.82
|
|
Mean Change in Quality of Life Scores, for Each Domain, From Day 1 to Day 42 Using the Cystic Fibrosis Questionnaire Revised (CFQ-R).
Respiratory Domain
|
-2.78 mean change of scores on a scale
Standard Deviation 13.61
|
2.78 mean change of scores on a scale
Standard Deviation 13.28
|
-6.94 mean change of scores on a scale
Standard Deviation 22.17
|
|
Mean Change in Quality of Life Scores, for Each Domain, From Day 1 to Day 42 Using the Cystic Fibrosis Questionnaire Revised (CFQ-R).
Digestion Domain
|
-9.72 mean change of scores on a scale
Standard Deviation 12.51
|
4.17 mean change of scores on a scale
Standard Deviation 10.18
|
-5.56 mean change of scores on a scale
Standard Deviation 16.80
|
SECONDARY outcome
Timeframe: Baseline and Day 28Population: Safety indices included changes in WBC during treatment for each group.
Safety indices included changes in FEV1 (lung function), changes in WBC, alterations in ECG and sputum microbiology. There were no significant alterations in the ECG in any subjects over the course of the study. There were no subjects who acquired multiple resistant changes in microbiology of sputum and no acquisition of B. cepacia in any subjects. Therefore, these data were not analyzed. The median changes in FEV1 and median changes in WBC, ESR and CRP are reported.
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=8 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=8 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Change in WBC (White Blood Cell) Count by Group During Treatment Period
|
-510.28 K/cu mm
Interval -2775.0 to 350.92
|
-0.98 K/cu mm
Interval -680.0 to 930.0
|
-0.13 K/cu mm
Interval -2130.63 to 1.87
|
SECONDARY outcome
Timeframe: Baseline and Day 28Population: All subjects had blood labs for CRP drawn on Visits 1,3,4 and 5. The changes in median CRP were calculated during the Treatment Period (28 days).
Median changes in CRP and IQR were assessed from serum samples collected at Visits 1,3, 4 and 5.
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=8 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=8 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Changes in C Reactive Protein (CRP) During Treatment.
|
-0.20 mg/dL
Interval -0.5 to 0.15
|
0.05 mg/dL
Interval 0.0 to 0.4
|
-0.05 mg/dL
Interval -0.2 to 0.05
|
SECONDARY outcome
Timeframe: Baseline and Day 28Population: All subjects had blood labs for ESR drawn on Visits 1,3,4 and 5. The changes in median ESR were calculated during the Treatment Period (28 days).
Median change in serum ESR (mm/hr) and IQR was calculated from Treatment Period (28 days).
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=8 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=8 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Changes in Erythrocyte Sedimentation Rate (ESR) During Treatment Period.
|
-1.00 mm/hr
Interval -5.5 to 0.5
|
3.50 mm/hr
Interval 0.05 to 11.5
|
0.00 mm/hr
Interval -3.5 to 5.0
|
SECONDARY outcome
Timeframe: Day 0 and Day 28Population: The full set of microarray data has been deposited in the National Center for Biotechnology Information (NCBI) Gene Expression Omnibus (GEO). Results can be found under the accession number GSE76347. One subject's pair of specimens in the placebo group was not collected.
Rhinoprobe was used to collect nasal epithelial cells. The cells were collected pre and post-treatment and placed in Trizol for RNA isolation then used to measure the effect of digitoxin on gene expression. The full set of microarray data has been deposited in the National Center for Biotechnology Information (NCBI) Gene Expression Omnibus (GEO). The accession number for that data is GSE76347 (data available Dec 2018).
Outcome measures
| Measure |
Placebo
n=23 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Number of CF Subjects With Microarray Results From Nasal Epithelial Cells to Measure the Effect of Digitoxin on Gene Expression.
|
23 participants
|
—
|
—
|
SECONDARY outcome
Timeframe: 28 daysPopulation: Count of clinically significant alterations in the ECG in all subjects during the Treatment Phase of the study.
Safety indices included an assessment for the number of clinically significant alterations in the subjects ECG readings from Day 1 to Day 28.
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=8 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=8 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Clinically Significant Alterations in ECG Readings
|
0 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 28 daysPopulation: There were no significant changes in the microbiology of sputum in any subjects over the course of treatment.
Count of clinically significant changes in sputum microbiology during the Treatment phase (Day 1 to Day 28) to include any new acquisition of B. cepacia or new acquisition of any multiple resistant organism.
Outcome measures
| Measure |
Placebo
n=8 Participants
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=8 Participants
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=8 Participants
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Clinically Significant Changes in the Microbiology of Sputum in Subjects
|
0 participants
|
0 participants
|
0 participants
|
Adverse Events
Placebo
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Low Dose 0.05 mg Digitoxin
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Higher Dose 0.1mg Digitoxin
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Placebo
n=8 participants at risk
placebo given daily for 28 days
placebo: pill taken once daily for 28 days
|
Low Dose 0.05 mg Digitoxin
n=8 participants at risk
low dose 0.05mg digitoxin given once daily for 28 days
digitoxin: 0.05mg tabs, once daily for 28 days
|
Higher Dose 0.1mg Digitoxin
n=8 participants at risk
higher dose 0.1mg digitoxin daily for 28 days
digitoxin: 0.1mg pills, once daily for 28 days.
|
|---|---|---|---|
|
Gastrointestinal disorders
abdominal pain
|
25.0%
2/8 • Number of events 4 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Nervous system disorders
anxiety
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Infections and infestations
chills
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
25.0%
2/8 • Number of events 2 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
25.0%
2/8 • Number of events 2 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
25.0%
2/8 • Number of events 2 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Respiratory, thoracic and mediastinal disorders
crackles
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 2 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Respiratory, thoracic and mediastinal disorders
decrease breath sounds
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Respiratory, thoracic and mediastinal disorders
decreased lung function
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Gastrointestinal disorders
diarrhea
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 2 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Injury, poisoning and procedural complications
epistaxis
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
25.0%
2/8 • Number of events 2 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Injury, poisoning and procedural complications
erythema, nares
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
General disorders
fatigue
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 2 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Infections and infestations
fever
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Gastrointestinal disorders
GERD
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 2 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
General disorders
headache
|
37.5%
3/8 • Number of events 3 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
50.0%
4/8 • Number of events 8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
37.5%
3/8 • Number of events 3 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Injury, poisoning and procedural complications
hemoptysis
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Respiratory, thoracic and mediastinal disorders
increased sputum
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Renal and urinary disorders
increased urination
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Reproductive system and breast disorders
menstrual cramps
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Musculoskeletal and connective tissue disorders
muscle pain
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
50.0%
4/8 • Number of events 4 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Injury, poisoning and procedural complications
pain, nares
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 2 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Gastrointestinal disorders
nausea
|
25.0%
2/8 • Number of events 3 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Infections and infestations
night sweats
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Respiratory, thoracic and mediastinal disorders
pulmonary exacerbation
|
25.0%
2/8 • Number of events 2 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Skin and subcutaneous tissue disorders
rash
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Respiratory, thoracic and mediastinal disorders
rhinitis
|
25.0%
2/8 • Number of events 2 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Respiratory, thoracic and mediastinal disorders
rhonchi
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Respiratory, thoracic and mediastinal disorders
shortness of breath
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Respiratory, thoracic and mediastinal disorders
sinusitis
|
37.5%
3/8 • Number of events 3 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Infections and infestations
sore throat
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Musculoskeletal and connective tissue disorders
sprain
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Nervous system disorders
syncope
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Nervous system disorders
tonic clonic seizure
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Reproductive system and breast disorders
upper respiratory infection
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
25.0%
2/8 • Number of events 2 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Renal and urinary disorders
urinary tract infection
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
General disorders
weight loss
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Respiratory, thoracic and mediastinal disorders
wheeze
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
|
Infections and infestations
vaginal yeast infection
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
12.5%
1/8 • Number of events 1 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
0.00%
0/8 • Adverse events were collected from Screening through Day 42 or the Early Withdraw Visit.
Physical exams by study physician at each visit and documented in the subject case report forms. All Adverse Events were subsequently followed up until resolution.
|
Additional Information
Pamela L. Zeitlin, M.D., Ph.D., Professor and Director of SOM Pediatric Pulmonary
Johns Hopkins University
Phone: 443-287-8981
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place