Trial Outcomes & Findings for Safety and Efficacy of Pioglitazone as an Anti-inflammatory for the Treatment of Cystic Fibrosis (CF) Lung Disease (NCT NCT00322868)
NCT ID: NCT00322868
Last Updated: 2018-02-23
Results Overview
The total number of white cells log 10 cells/mL
COMPLETED
NA
21 participants
Day 0 and Day 29
2018-02-23
Participant Flow
All patients who will be approached for participation in this trial will first be identified using either Port CF (CFF Registry) or CF Center database. Once potentials are identified a chart review will be conducted to determine eligibility. Patients will approached at clinic for possible participation in the study.
Washout: A 4 week washout from chronic use of NSAIDs, corticosteroids and oral meds to treat diabetes will be required. Subjects will be excluded if they take these medication within 72 hours of Visit 1. Subjects using alternate month tobramycin for inhalation (TOBI) will need to be on an off cycle to participate in this study.
Participant milestones
| Measure |
Pioglitazone (30 mg)
After screening eligibility requirements were met, cystic fibrosis subjects received pioglitazone orally, 30 mg, once daily, for 28 days
|
|---|---|
|
Overall Study
STARTED
|
21
|
|
Overall Study
COMPLETED
|
20
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Pioglitazone (30 mg)
After screening eligibility requirements were met, cystic fibrosis subjects received pioglitazone orally, 30 mg, once daily, for 28 days
|
|---|---|
|
Overall Study
disease progression
|
1
|
Baseline Characteristics
1 subject withdrawn due to disease progression
Baseline characteristics by cohort
| Measure |
Pioglitazone (30 mg)
n=21 Participants
After screening eligibility requirements were met, cystic fibrosis subjects received pioglitazone orally, 30 mg, once daily, for 28 days
|
|---|---|
|
Age, Continuous
|
30 years
STANDARD_DEVIATION 9.28 • n=20 Participants • 1 subject withdrawn due to disease progression
|
|
Sex: Female, Male
Female
|
8 Participants
n=20 Participants • 1 subject was withdrawn and no samples analyzed
|
|
Sex: Female, Male
Male
|
12 Participants
n=20 Participants • 1 subject was withdrawn and no samples analyzed
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=20 Participants • 1 subject withdrawn due to disease progression. no samples analyzed.
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
20 Participants
n=20 Participants • 1 subject withdrawn due to disease progression. no samples analyzed.
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=20 Participants • 1 subject withdrawn due to disease progression. no samples analyzed.
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=20 Participants • 1 subject withdrawn due to disease progression no samples analyzed
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=20 Participants • 1 subject withdrawn due to disease progression no samples analyzed
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=20 Participants • 1 subject withdrawn due to disease progression no samples analyzed
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=20 Participants • 1 subject withdrawn due to disease progression no samples analyzed
|
|
Race (NIH/OMB)
White
|
18 Participants
n=20 Participants • 1 subject withdrawn due to disease progression no samples analyzed
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=20 Participants • 1 subject withdrawn due to disease progression no samples analyzed
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=20 Participants • 1 subject withdrawn due to disease progression no samples analyzed
|
|
Region of Enrollment
United States
|
20 Participants
n=20 Participants • 1 subject was withdrawn due to disease progression. No samples analyzed.
|
|
Total white cell count
|
6.93 log 10 (cells/mL)
STANDARD_DEVIATION 0.40 • n=20 Participants • 1 subject withdrawn due to disease progression. No samples analyzed.
|
|
Total neutrophil count
|
6.88 Log 10 (PMN/mL)
STANDARD_DEVIATION 0.39 • n=20 Participants • 1 subject withdrawn due to disease progression. No samples analyzed.
|
|
percent neutrophils
|
82.47 percent of white blood cells
STANDARD_DEVIATION 11.88 • n=20 Participants • 1 subject withdrawn due to disease progression. No samples analyzed
|
PRIMARY outcome
Timeframe: Day 0 and Day 29Population: Of the 20 enrolled subjects, one did not produce sputum at Baseline and a second subject's Post-Treatment sputum sample was lost due to a lab error. This reduced the number of subjects per group to 19. Of these 19 subjects, 18 subjects had both Baseline and Post-Treatment sputum samples.
The total number of white cells log 10 cells/mL
Outcome measures
| Measure |
Baseline
n=19 Participants
Results pre-Pioglitazone dosing
|
Post-Treatment
n=19 Participants
Results following 28 days of Pioglitazone (oral, 30mg, once daily)
|
|---|---|---|
|
Sputum White Cell Count
|
6.93 log 10 (cells/mL)
Standard Deviation 0.40
|
6.81 log 10 (cells/mL)
Standard Deviation 0.41
|
PRIMARY outcome
Timeframe: Day 0 and Day 29Population: Of the 20 enrolled subjects, one did not produce sputum at Baseline and a second subject's Post-Treatment sputum sample was lost due to a lab error. This reduced the number of subjects per group to 19. Of these 19 subjects, 18 subjects had both Baseline and Post-Treatment sputum samples.
sputum neutrophils log 10 (cells/mL)
Outcome measures
| Measure |
Baseline
n=19 Participants
Results pre-Pioglitazone dosing
|
Post-Treatment
n=19 Participants
Results following 28 days of Pioglitazone (oral, 30mg, once daily)
|
|---|---|---|
|
Sputum Neutrophil Count
|
6.88 log 10 (cells/mL)
Standard Deviation 0.39
|
6.75 log 10 (cells/mL)
Standard Deviation 0.42
|
PRIMARY outcome
Timeframe: Day 0 and Day 29Population: Of the 20 enrolled subjects, one did not produce sputum at Baseline and a second subject's Post-Treatment sputum sample was lost due to a lab error. This reduced the number of subjects per group to 19. Of these 19 subjects, 18 subjects had both Baseline and Post-Treatment sputum samples.
Neutrophils as a percent of the total white cells.
Outcome measures
| Measure |
Baseline
n=19 Participants
Results pre-Pioglitazone dosing
|
Post-Treatment
n=19 Participants
Results following 28 days of Pioglitazone (oral, 30mg, once daily)
|
|---|---|---|
|
Sputum Neutrophil Percent
|
82.47 Percent (%) of white blood cells
Standard Deviation 11.88
|
74.44 Percent (%) of white blood cells
Standard Deviation 17.07
|
PRIMARY outcome
Timeframe: Day 0 and Day 29Population: Of the 20 enrolled subjects, one did not produce sputum at Baseline and a second subject's Post-Treatment sputum sample was lost due to a lab error. This reduced the number of subjects per group to 19. Of these 19 subjects, 18 subjects had both Baseline and Post-Treatment sputum samples.
Log 10 of Concentration of active Elastase in mcg/mL
Outcome measures
| Measure |
Baseline
n=19 Participants
Results pre-Pioglitazone dosing
|
Post-Treatment
n=19 Participants
Results following 28 days of Pioglitazone (oral, 30mg, once daily)
|
|---|---|---|
|
Sputum Active Elastase
|
2.03 log 10 (mcg/mL)
Standard Deviation 0.36
|
1.96 log 10 (mcg/mL)
Standard Deviation 0.36
|
PRIMARY outcome
Timeframe: Day 0 and Day 29Population: Of the 20 enrolled subjects, one did not produce sputum at Baseline and a second subject's Post-Treatment sputum sample was lost due to a lab error. This reduced the number of subjects per group to 19. Of these 19 subjects, 18 subjects had both Baseline and Post-Treatment sputum samples.
The concentration of Tumor Necrosis Factor-α (TNFα) log 10 (pg/mL)
Outcome measures
| Measure |
Baseline
n=19 Participants
Results pre-Pioglitazone dosing
|
Post-Treatment
n=19 Participants
Results following 28 days of Pioglitazone (oral, 30mg, once daily)
|
|---|---|---|
|
Sputum TNFα
|
1.74 log 10 (pg/mL)
Standard Deviation 0.49
|
1.69 log 10 (pg/mL)
Standard Deviation 0.59
|
PRIMARY outcome
Timeframe: Day 0 and Day 29Population: Of the 20 enrolled subjects, one did not produce sputum at Baseline and a second subject's Post-Treatment sputum sample was lost due to a lab error. This reduced the number of subjects per group to 19. Of these 19 subjects, 18 subjects had both Baseline and Post-Treatment sputum samples.
The concentration of Interleukin-1ß (IL-1ß) log 10 (pg/mL)
Outcome measures
| Measure |
Baseline
n=19 Participants
Results pre-Pioglitazone dosing
|
Post-Treatment
n=19 Participants
Results following 28 days of Pioglitazone (oral, 30mg, once daily)
|
|---|---|---|
|
Sputum IL-1ß
|
4.05 log 10 (pg/mL)
Standard Deviation 0.42
|
3.99 log 10 (pg/mL)
Standard Deviation 0.52
|
PRIMARY outcome
Timeframe: Day 0 and Day 29Population: Of the 20 enrolled subjects, one did not produce sputum at Baseline and a second subject's Post-Treatment sputum sample was lost due to a lab error. This reduced the number of subjects per group to 19. Of these 19 subjects, 18 subjects had both Baseline and Post-Treatment sputum samples.
The concentration of Interleukin-6 (IL-6) log 10 (pg/mL)
Outcome measures
| Measure |
Baseline
n=19 Participants
Results pre-Pioglitazone dosing
|
Post-Treatment
n=19 Participants
Results following 28 days of Pioglitazone (oral, 30mg, once daily)
|
|---|---|---|
|
Sputum IL-6
|
1.38 log 10 (pg/mL)
Standard Deviation 0.33
|
1.41 log 10 (pg/mL)
Standard Deviation 0.32
|
PRIMARY outcome
Timeframe: Day 0 and Day 29Population: Of the 20 enrolled subjects, one did not produce sputum at Baseline and a second subject's Post-Treatment sputum sample was lost due to a lab error. This reduced the number of subjects per group to 19. Of these 19 subjects, 18 subjects had both Baseline and Post-Treatment sputum samples.
Concentration of Interleukin-8 log 10 (pg/mL)
Outcome measures
| Measure |
Baseline
n=19 Participants
Results pre-Pioglitazone dosing
|
Post-Treatment
n=19 Participants
Results following 28 days of Pioglitazone (oral, 30mg, once daily)
|
|---|---|---|
|
Sputum IL-8
|
5.13 log 10 (pg/mL)
Standard Deviation 0.35
|
5.10 log 10 (pg/mL)
Standard Deviation 0.34
|
Adverse Events
Pioglitazone (30 mg)
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Pioglitazone (30 mg)
n=20 participants at risk
After screening eligibility requirements were met, cystic fibrosis subjects received pioglitazone orally,30 mg, once daily for 28 days
|
|---|---|
|
Endocrine disorders
hypoglycemic event
|
5.0%
1/20
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place