Trial Outcomes & Findings for Ursodiol in Treating Patients With Barrett Esophagus and Low-Grade Dysplasia (NCT NCT01097304)
NCT ID: NCT01097304
Last Updated: 2017-12-19
Results Overview
8OHdG will be assessed by percentage of positively stained nuclear area. A paired t-test at a one-sided 0.05 significance level will be used to assess change during intervention. The observed results will be reported along with the corresponding confidence intervals.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
36 participants
Primary outcome timeframe
Baseline to 6 months
Results posted on
2017-12-19
Participant Flow
Participant milestones
| Measure |
Treatment (Ursodiol)
Patients receive ursodiol PO (13 to 15 mg/kg/day) BID for 6 months in the absence of disease progression or unacceptable toxicity.
Ursodiol: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Overall Study
STARTED
|
36
|
|
Overall Study
COMPLETED
|
29
|
|
Overall Study
NOT COMPLETED
|
7
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Ursodiol in Treating Patients With Barrett Esophagus and Low-Grade Dysplasia
Baseline characteristics by cohort
| Measure |
Treatment (Ursodiol)
n=36 Participants
Patients receive ursodiol PO (13 to 15 mg/kg/day) BID for 6 months in the absence of disease progression or unacceptable toxicity.
Ursodiol: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
20 Participants
n=93 Participants
|
|
Age, Categorical
>=65 years
|
16 Participants
n=93 Participants
|
|
Age, Continuous
|
62.8 years
STANDARD_DEVIATION 10.4 • n=93 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
30 Participants
n=93 Participants
|
|
Region of Enrollment
United States
|
36 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Baseline to 6 monthsPopulation: participants with fewer than 500 total nuclei in longitudinally sectioned crypts opening to the lumen were excluded from analysis
8OHdG will be assessed by percentage of positively stained nuclear area. A paired t-test at a one-sided 0.05 significance level will be used to assess change during intervention. The observed results will be reported along with the corresponding confidence intervals.
Outcome measures
| Measure |
Treatment (Ursodiol)
n=25 Participants
Patients receive ursodiol PO BID for 6 months in the absence of disease progression or unacceptable toxicity.
Ursodiol: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Reversal of Oxidative DNA Damage as Assessed by Changes in 8-hydroxy-2' -Deoxyguanosine (8OHdG) Immunostaining
|
1.62 % of strongly/moderately stained nuclei
Standard Deviation 13.13
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: analysis was limited to participants with baseline and 6-month gastric fluid
Outcome measures
| Measure |
Treatment (Ursodiol)
n=28 Participants
Patients receive ursodiol PO BID for 6 months in the absence of disease progression or unacceptable toxicity.
Ursodiol: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Changes in Gastric Bile Acid Composition (Change in Percent of Total Bile Acid Present as Ursodeoxycholic Acid and Its Glycine/Taurine Conjugates) Measured by Liquid Chromatography-tandem Mass Spectrometry, From Baseline to Post-intervention
|
66.28 % of total bile acid
Interval 36.29 to 77.09
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: analysis was limited to participants with baseline and 6-month gastric fluid
Outcome measures
| Measure |
Treatment (Ursodiol)
n=28 Participants
Patients receive ursodiol PO BID for 6 months in the absence of disease progression or unacceptable toxicity.
Ursodiol: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Changes in Gastric Bile Acid Composition (Change in Percent of Total Bile Acid Present as Deoxycholic Acid and Its Glycine/Taurine Conjugates) Measured by Liquid Chromatography-tandem Mass Spectrometry, From Baseline to Post-intervention
|
-13.31 % of total bile acid
Interval -46.98 to 0.4
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: tissue slides with fewer than 500 total nuclei in longitudinally sectioned crypts opening to the lumen were excluded from analysis
Results will be analyzed using paired t-tests. Results (mean values and changes during intervention) will be reported along with the corresponding confidence intervals.
Outcome measures
| Measure |
Treatment (Ursodiol)
n=29 Participants
Patients receive ursodiol PO BID for 6 months in the absence of disease progression or unacceptable toxicity.
Ursodiol: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Changes in Cell Proliferation in BE Epithelium From Baseline to Post-intervention as Assessed by Proliferation-related Ki-67 Antigen (Ki67) Immunostaining, Percentage of Positively Stained Nuclei, in BE Tissue Sections
|
1.36 % change
Standard Deviation 13.13
|
Adverse Events
Treatment (Ursodiol)
Serious events: 5 serious events
Other events: 31 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment (Ursodiol)
n=36 participants at risk
Patients receive ursodiol PO BID for 6 months in the absence of disease progression or unacceptable toxicity.
Ursodiol: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Cardiac disorders
Hypotension
|
2.8%
1/36
|
|
Cardiac disorders
Ischemia/Infarction
|
2.8%
1/36
|
|
Cardiac disorders
Atrial Fibrillation
|
2.8%
1/36
|
|
Gastrointestinal disorders
Enteritis
|
2.8%
1/36
|
|
Gastrointestinal disorders
Barrett's esophagus with at least intramucosal carcinoma
|
2.8%
1/36
|
|
Gastrointestinal disorders
Pancreatitis
|
2.8%
1/36
|
|
Musculoskeletal and connective tissue disorders
Post surgical pain
|
2.8%
1/36
|
|
Nervous system disorders
CNS cerebrovascular ischemia
|
2.8%
1/36
|
|
Nervous system disorders
Altered mental status
|
2.8%
1/36
|
|
Cardiac disorders
Chest/Thorax pain
|
2.8%
1/36
|
|
Renal and urinary disorders
Renal failure
|
2.8%
1/36
|
Other adverse events
| Measure |
Treatment (Ursodiol)
n=36 participants at risk
Patients receive ursodiol PO BID for 6 months in the absence of disease progression or unacceptable toxicity.
Ursodiol: Given PO
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Cardiac disorders
hypertension
|
5.6%
2/36
|
|
General disorders
Fatigue
|
11.1%
4/36
|
|
General disorders
Fever
|
5.6%
2/36
|
|
Investigations
Weight loss
|
5.6%
2/36
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
5.6%
2/36
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.3%
3/36
|
|
Gastrointestinal disorders
Colitis
|
5.6%
2/36
|
|
Gastrointestinal disorders
Constipation
|
11.1%
4/36
|
|
Gastrointestinal disorders
Diarrhea
|
27.8%
10/36
|
|
Gastrointestinal disorders
Distention/Bloating
|
5.6%
2/36
|
|
Gastrointestinal disorders
Flatulence
|
5.6%
2/36
|
|
Gastrointestinal disorders
Heartburn
|
8.3%
3/36
|
|
Gastrointestinal disorders
Nausea
|
19.4%
7/36
|
|
Gastrointestinal disorders
Phytobezoar
|
5.6%
2/36
|
|
Gastrointestinal disorders
Vomiting
|
16.7%
6/36
|
|
Infections and infestations
Upper airway infection
|
16.7%
6/36
|
|
Metabolism and nutrition disorders
Hyponatremia
|
5.6%
2/36
|
|
Nervous system disorders
Dizziness
|
16.7%
6/36
|
|
Gastrointestinal disorders
Abdomen pain
|
11.1%
4/36
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.3%
3/36
|
|
Nervous system disorders
Headache
|
16.7%
6/36
|
|
Musculoskeletal and connective tissue disorders
Joint pain
|
8.3%
3/36
|
|
Gastrointestinal disorders
Stomach pain
|
8.3%
3/36
|
|
Vascular disorders
Phlebitis
|
5.6%
2/36
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60