Trial Outcomes & Findings for Pioglitazone for Idiopathic Gastroparesis (NCT NCT04300127)

NCT ID: NCT04300127

Last Updated: 2025-04-30

Results Overview

The effect of Pioglitazone on nausea, early satiety, postprandial fullness, and upper abdominal pain as per changes in patient scoring in The American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index (GCSI) which has been designed to assess symptoms associated with gastroparesis. Symptoms are rated as none (0), mild (1), moderate (2), severe (3), very severe (4) scale of the worst severity of the symptom over the last 24 hours. Values reported represent the mean participant scale choice.

Recruitment status

COMPLETED

Study phase

EARLY_PHASE1

Target enrollment

14 participants

Primary outcome timeframe

Baseline, later monthly up to 3 months.

Results posted on

2025-04-30

Participant Flow

Participant milestones

Participant milestones
Measure
Pioglitazone Group
Candidates who are deemed eligible after the screening period will receive Pioglitazone 30 mg once daily for 8 weeks.
Baseline
STARTED
14
Baseline
COMPLETED
14
Baseline
NOT COMPLETED
0
First Intervention - 4 Weeks Treatment
STARTED
14
First Intervention - 4 Weeks Treatment
COMPLETED
12
First Intervention - 4 Weeks Treatment
NOT COMPLETED
2
Second Intervention - 4 Weeks Treatment
STARTED
12
Second Intervention - 4 Weeks Treatment
COMPLETED
12
Second Intervention - 4 Weeks Treatment
NOT COMPLETED
0
Third Intervention - 4 Weeks of Wash Out
STARTED
12
Third Intervention - 4 Weeks of Wash Out
COMPLETED
12
Third Intervention - 4 Weeks of Wash Out
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pioglitazone for Idiopathic Gastroparesis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Pioglitazone
n=14 Participants
Candidates who after the screening period are eligible to receive Pioglitazone Pioglitazone 30 mg: Patients will received 30 mg of Pioglitazone once a day for 8 weeks
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
14 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Age, Continuous
35.31 years
STANDARD_DEVIATION 9.30 • n=5 Participants
Sex: Female, Male
Female
13 Participants
n=5 Participants
Sex: Female, Male
Male
1 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
14 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
14 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Region of Enrollment
United States
14 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline, later monthly up to 3 months.

Population: Participants who started time point are reported

The effect of Pioglitazone on nausea, early satiety, postprandial fullness, and upper abdominal pain as per changes in patient scoring in The American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index (GCSI) which has been designed to assess symptoms associated with gastroparesis. Symptoms are rated as none (0), mild (1), moderate (2), severe (3), very severe (4) scale of the worst severity of the symptom over the last 24 hours. Values reported represent the mean participant scale choice.

Outcome measures

Outcome measures
Measure
Pioglitazone Group
n=14 Participants
Candidates who are deemed eligible after the screening period will receive Pioglitazone 30 mg once daily for 8 weeks.
Severity of Gastrointestinal Symptoms as Assessed by the American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index
Baseline
2.9 score on a scale
Standard Deviation 0.9
Severity of Gastrointestinal Symptoms as Assessed by the American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index
1st month of treatment
2.3 score on a scale
Standard Deviation 1.1
Severity of Gastrointestinal Symptoms as Assessed by the American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index
2nd month of treatment
2.2 score on a scale
Standard Deviation 1.1
Severity of Gastrointestinal Symptoms as Assessed by the American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index
3rd month / washout
2.6 score on a scale
Standard Deviation 0.8

SECONDARY outcome

Timeframe: Baseline and later monthly up to 3 months

Population: Participants who started time point are reported

The Patient Assessment of Gastrointestinal Symptoms (PAGI-SYM) questionnaire is a patient-reported tool designed to evaluate the severity of upper gastrointestinal symptoms across multiple domains, including nausea/vomiting, postprandial fullness/early satiety, bloating, upper abdominal pain, and heartburn/regurgitation. Each symptom is rated on a 0 to 5 Likert scale, where 0 indicates no symptoms and 5 represents very severe symptoms, with higher scores reflecting worse symptom severity. The total PAGI-SYM score is typically calculated as the average of individual domain scores, resulting in a possible score range of 0 to 5. The mean participant scale choice is reported.

Outcome measures

Outcome measures
Measure
Pioglitazone Group
n=14 Participants
Candidates who are deemed eligible after the screening period will receive Pioglitazone 30 mg once daily for 8 weeks.
Patient Assessment of Upper Gastrointestinal Symptom Severity Index (PAGI-SYM)
Baseline
2.6 score on a scale
Standard Deviation 0.9
Patient Assessment of Upper Gastrointestinal Symptom Severity Index (PAGI-SYM)
1st month of treatment
1.9 score on a scale
Standard Deviation 0.9
Patient Assessment of Upper Gastrointestinal Symptom Severity Index (PAGI-SYM)
2nd month of treatment
1.9 score on a scale
Standard Deviation 0.9
Patient Assessment of Upper Gastrointestinal Symptom Severity Index (PAGI-SYM)
3rd month / washout
2.3 score on a scale
Standard Deviation 0.8

SECONDARY outcome

Timeframe: Baseline, later monthly up to 3 months

Population: Participants who started time point are reported

The effect of Pioglitazone on quality of life assessed by the SF-36v2 Health Survey. The SF-36v2 is a 36-item, self-report measure designed to assess quality of life in patients. This measure also provides two summary scores (physical and mental health) and eight scale scores. The eight sections are: vitality, physical functioning, bodily pain and general health perceptions, physical role functioning, emotional role functioning, social role functioning and mental health. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability, i.e., a score of zero is equivalent to maximum disability and a score of 100 is equivalent to no disability.

Outcome measures

Outcome measures
Measure
Pioglitazone Group
n=14 Participants
Candidates who are deemed eligible after the screening period will receive Pioglitazone 30 mg once daily for 8 weeks.
Functional Status as Assessed by the SF-36v2 Health Survey
Pain - 1st month
45.2 score on a scale
Standard Deviation 23.7
Functional Status as Assessed by the SF-36v2 Health Survey
Physical Functioning - Baseline
54.3 score on a scale
Standard Deviation 17.6
Functional Status as Assessed by the SF-36v2 Health Survey
Physical Functioning - Washout
64.6 score on a scale
Standard Deviation 22.1
Functional Status as Assessed by the SF-36v2 Health Survey
Role limitations due to physical activity - Baseline
40.2 score on a scale
Standard Deviation 18.6
Functional Status as Assessed by the SF-36v2 Health Survey
Role limitations due to physical activity - 1st month
51.3 score on a scale
Standard Deviation 23.2
Functional Status as Assessed by the SF-36v2 Health Survey
Role limitations due to physical activity - 2nd month
60.4 score on a scale
Standard Deviation 27.2
Functional Status as Assessed by the SF-36v2 Health Survey
Role limitations due to physical activity - 3rd month / Washout
47.2 score on a scale
Standard Deviation 30.9
Functional Status as Assessed by the SF-36v2 Health Survey
Role limitations due to emotional problems - Baseline
65.5 score on a scale
Standard Deviation 29.9
Functional Status as Assessed by the SF-36v2 Health Survey
Role limitations due to emotional problems - 1st month
68.5 score on a scale
Standard Deviation 30.3
Functional Status as Assessed by the SF-36v2 Health Survey
Role limitations due to emotional problems - 2nd month
77.8 score on a scale
Standard Deviation 25.5
Functional Status as Assessed by the SF-36v2 Health Survey
Role limitations due to emotional problems - 3rd month / Washout
68.9 score on a scale
Standard Deviation 30.9
Functional Status as Assessed by the SF-36v2 Health Survey
Pain - Baseline
62.9 score on a scale
Standard Deviation 19.3
Functional Status as Assessed by the SF-36v2 Health Survey
Pain - 2nd month
41.7 score on a scale
Standard Deviation 21.3
Functional Status as Assessed by the SF-36v2 Health Survey
Pain - 3rd month/Washout
48.5 score on a scale
Standard Deviation 23.9
Functional Status as Assessed by the SF-36v2 Health Survey
Emotional well being - Baseline
46 score on a scale
Standard Deviation 8.1
Functional Status as Assessed by the SF-36v2 Health Survey
Emotional well being - 2nd month
48.7 score on a scale
Standard Deviation 9.8
Functional Status as Assessed by the SF-36v2 Health Survey
Emotional well being - 3rd month / Washout
46.9 score on a scale
Standard Deviation 11
Functional Status as Assessed by the SF-36v2 Health Survey
Energy / fatigue - Baseline
35.7 score on a scale
Standard Deviation 7.3
Functional Status as Assessed by the SF-36v2 Health Survey
Energy / fatigue - 1st month
33.6 score on a scale
Standard Deviation 17.4
Functional Status as Assessed by the SF-36v2 Health Survey
Energy / fatigue - 2nd month
35.4 score on a scale
Standard Deviation 9.2
Functional Status as Assessed by the SF-36v2 Health Survey
Energy / fatigue - 3rd month / Washout
35.5 score on a scale
Standard Deviation 7.6
Functional Status as Assessed by the SF-36v2 Health Survey
Social functioning - Baseline
51.8 score on a scale
Standard Deviation 6.7
Functional Status as Assessed by the SF-36v2 Health Survey
Social functioning - 1st month
48.2 score on a scale
Standard Deviation 6.7
Functional Status as Assessed by the SF-36v2 Health Survey
Social functioning - 3rd month / Washout
50 score on a scale
Standard Deviation 5.6
Functional Status as Assessed by the SF-36v2 Health Survey
General health - Baseline
63.6 score on a scale
Standard Deviation 9.1
Functional Status as Assessed by the SF-36v2 Health Survey
General health - 1st month
55.7 score on a scale
Standard Deviation 20.7
Functional Status as Assessed by the SF-36v2 Health Survey
General health - 2nd month
60.8 score on a scale
Standard Deviation 10.6
Functional Status as Assessed by the SF-36v2 Health Survey
General health - 3rd month / Washout
63.2 score on a scale
Standard Deviation 7.2
Functional Status as Assessed by the SF-36v2 Health Survey
Physical Functioning - 2nd month
67.1 score on a scale
Standard Deviation 24.1
Functional Status as Assessed by the SF-36v2 Health Survey
Physical Functioning - 1st month
64.6 score on a scale
Standard Deviation 20.0
Functional Status as Assessed by the SF-36v2 Health Survey
Emotional well being - 1st month
45.7 score on a scale
Standard Deviation 20.4
Functional Status as Assessed by the SF-36v2 Health Survey
Social functioning - 2nd month
52.1 score on a scale
Standard Deviation 8.9

SECONDARY outcome

Timeframe: Baseline and 2 months after treatment initiation.

Population: Participants who started time point are reported

The effect of Pioglitazone on Inflammatory markers as per changes in the values of C reactive protein (CRP) in blood. Normal CRP levels are below 3.0 mg/L.

Outcome measures

Outcome measures
Measure
Pioglitazone Group
n=14 Participants
Candidates who are deemed eligible after the screening period will receive Pioglitazone 30 mg once daily for 8 weeks.
C- Reactive Protein Level in Blood
Baseline CRP
2.5 mg/L
Standard Deviation 3.9
C- Reactive Protein Level in Blood
8 weeks CRP
5.1 mg/L
Standard Deviation 12.1

SECONDARY outcome

Timeframe: Baseline and 2 months after treatment initiation

Population: Participants who started time point are reported

The effect of Pioglitazone on Inflammatory markers as per changes Erythrocyte Sedimentation Rate (ESR) in blood. Results are reported as the millimeters of clear fluid (plasma) that are present at the top portion of the tube after one hour. The normal range is 0 to 22 mm/hr for men and 0 to 29 mm/hr for women. Values above these ranges are considered worse.

Outcome measures

Outcome measures
Measure
Pioglitazone Group
n=14 Participants
Candidates who are deemed eligible after the screening period will receive Pioglitazone 30 mg once daily for 8 weeks.
Erythrocyte Sedimentation Rate (ESR)
Baseline ESR
14.5 mm/h
Standard Deviation 20.3
Erythrocyte Sedimentation Rate (ESR)
2 Months ESR
14.8 mm/h
Standard Deviation 13.1

SECONDARY outcome

Timeframe: Baseline, later monthly up to 3 months

Population: Participants who started time point are reported

The effect of Pioglitazone on mood as per changes in the score of Beck Depression Inventory (BDI-II). The BDI-II is a commonly used, reliable 21-item self-report measure designed to assess for depression. Individuals are asked to respond to each question based on a two-week time period. The BDI-II is widely used as an indicator of the severity of depression, but not as a diagnostic tool. BDI-II items are rated on a 4-point scale ranging from 0 to 3 based on the severity of each item. The maximum total score range is 0-63. Higher total scores indicate more severe depressive symptoms.

Outcome measures

Outcome measures
Measure
Pioglitazone Group
n=14 Participants
Candidates who are deemed eligible after the screening period will receive Pioglitazone 30 mg once daily for 8 weeks.
Mood as Assessed by the Beck Depression Inventory
Baseline BDI-II
17.9 score on a scale
Standard Deviation 11.8
Mood as Assessed by the Beck Depression Inventory
4 Weeks BDI-II
8.6 score on a scale
Standard Deviation 8.2
Mood as Assessed by the Beck Depression Inventory
8 Weeks BDI-II
8.8 score on a scale
Standard Deviation 7.4
Mood as Assessed by the Beck Depression Inventory
Washout BDI-II
12.6 score on a scale
Standard Deviation 9.4

SECONDARY outcome

Timeframe: Baseline, later monthly up to 3 months

Population: Participants who started time point are reported

The effect of Pioglitazone on mood as per changes in the score of the State-Trait Anxiety Inventory (STAI). The STAI is a 40-item self-report measure designed to assess anxiety. This measure provides two subscale scores (State and Trait). STAI is one of the first tests to assess both state and trait anxiety separately. Each type of anxiety has its own scale of 20 different questions that are scored. Scores range from 20 to 80, with higher scores correlating with greater anxiety.

Outcome measures

Outcome measures
Measure
Pioglitazone Group
n=14 Participants
Candidates who are deemed eligible after the screening period will receive Pioglitazone 30 mg once daily for 8 weeks.
Mood as Assessed by the State-Trait Anxiety Inventory
State Anxiety - 1st month
35.8 score on a scale
Standard Deviation 13.9
Mood as Assessed by the State-Trait Anxiety Inventory
Trait Anxiety - 1st month
33.9 score on a scale
Standard Deviation 15.6
Mood as Assessed by the State-Trait Anxiety Inventory
Trait Anxiety - 2nd month
38.4 score on a scale
Standard Deviation 11.2
Mood as Assessed by the State-Trait Anxiety Inventory
Trait Anxiety - 3rd month / Washout
39.7 score on a scale
Standard Deviation 13
Mood as Assessed by the State-Trait Anxiety Inventory
State Anxiety - Baseline
44.1 score on a scale
Standard Deviation 12.8
Mood as Assessed by the State-Trait Anxiety Inventory
State Anxiety - 2nd month
38.7 score on a scale
Standard Deviation 15
Mood as Assessed by the State-Trait Anxiety Inventory
State Anxiety - 3rd month / Washout
36.8 score on a scale
Standard Deviation 18.2
Mood as Assessed by the State-Trait Anxiety Inventory
Trait Anxiety - Baseline
43.3 score on a scale
Standard Deviation 14.2

Adverse Events

Pioglitazone

Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Pioglitazone
n=14 participants at risk
Candidates who after the screening period are eligible to receive Pioglitazone Pioglitazone 30 mg: Patients will received 30 mg of Pioglitazone once a day for 8 weeks
Blood and lymphatic system disorders
Thrombocytopenia
7.1%
1/14 • Number of events 1 • Up to 3.5 months
Blood and lymphatic system disorders
Anemia
7.1%
1/14 • Number of events 1 • Up to 3.5 months
Blood and lymphatic system disorders
Leucopenia
7.1%
1/14 • Number of events 1 • Up to 3.5 months
General disorders
Fatigue
7.1%
1/14 • Number of events 1 • Up to 3.5 months
Nervous system disorders
Headaches
7.1%
1/14 • Number of events 1 • Up to 3.5 months
Musculoskeletal and connective tissue disorders
Muscle cramps
7.1%
1/14 • Number of events 1 • Up to 3.5 months
Gastrointestinal disorders
Constipation
7.1%
1/14 • Number of events 1 • Up to 3.5 months
Nervous system disorders
Lightheadedness
7.1%
1/14 • Number of events 1 • Up to 3.5 months
Gastrointestinal disorders
Vomit
7.1%
1/14 • Number of events 1 • Up to 3.5 months
Gastrointestinal disorders
Nausea
7.1%
1/14 • Number of events 2 • Up to 3.5 months

Additional Information

Dr. Jay Pasricha

Mayo Clinic Arizona

Phone: (480)301-4279

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place