Trial Outcomes & Findings for A Pilot Study of Vancomycin or Metronidazole in Patients With Primary Sclerosing Cholangitis (NCT NCT01085760)
NCT ID: NCT01085760
Last Updated: 2013-09-19
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
35 participants
Primary outcome timeframe
baseline, 12 weeks
Results posted on
2013-09-19
Participant Flow
A total of 35 adult patients with primary sclerosing cholangitis (PSC) were enrolled between February 2010 and November 2011 at Mayo Clinic, Rochester MN.
Participant milestones
| Measure |
Low Dose Vancomycin
Vancomycin 125 mg 4 times a day
|
High Dose Vancomycin
Vancomycin 250 mg 4 times a day
|
Low Dose Metronidazole
Metronidazole 250 mg 3 times a day
|
High Dose Metronidazole
metronidazole 500 mg 3 times a day
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
8
|
9
|
9
|
9
|
|
Overall Study
COMPLETED
|
7
|
8
|
7
|
6
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
2
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Pilot Study of Vancomycin or Metronidazole in Patients With Primary Sclerosing Cholangitis
Baseline characteristics by cohort
| Measure |
Low Dose Vancomycin
n=8 Participants
Vancomycin 125 mg 4 times a day
|
High Dose Vancomycin
n=9 Participants
Vancomycin 250 mg 4 times a day
|
Low Dose Metronidazole
n=9 Participants
Metronidazole 250 mg 3 times a day
|
High Dose Metronidazole
n=9 Participants
metronidazole 500 mg 3 times a day
|
Total
n=35 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
9 Participants
n=4 Participants
|
32 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
3 Participants
n=21 Participants
|
|
Age Continuous
|
40 years
n=5 Participants
|
42 years
n=7 Participants
|
35 years
n=5 Participants
|
40 years
n=4 Participants
|
40 years
n=21 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
21 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
14 Participants
n=21 Participants
|
|
Region of Enrollment
United States
|
8 participants
n=5 Participants
|
9 participants
n=7 Participants
|
9 participants
n=5 Participants
|
9 participants
n=4 Participants
|
35 participants
n=21 Participants
|
PRIMARY outcome
Timeframe: baseline, 12 weeksPopulation: The number of participants was based on a per protocol analysis.
Outcome measures
| Measure |
Low Dose Vancomycin
n=7 Participants
Vancomycin 125 mg 4 times a day
|
High Dose Vancomycin
n=8 Participants
Vancomycin 250 mg 4 times a day
|
Low Dose Metronidazole
n=7 Participants
Metronidazole 250 mg 3 times a day
|
High Dose Metronidazole
n=6 Participants
Metronidazole 500 mg 3 times a day
|
|---|---|---|---|---|
|
Change From Baseline in Alkaline Phosphatase Following 12 Weeks of Treatment
|
-117 U/L
Interval -304.0 to 165.0
|
-49 U/L
Interval -315.0 to 18.0
|
-62 U/L
Interval -502.0 to 212.0
|
-36 U/L
Interval -450.0 to 42.0
|
SECONDARY outcome
Timeframe: baseline, 12 weeksPopulation: Per protocol analysis done
Outcome measures
| Measure |
Low Dose Vancomycin
n=7 Participants
Vancomycin 125 mg 4 times a day
|
High Dose Vancomycin
n=8 Participants
Vancomycin 250 mg 4 times a day
|
Low Dose Metronidazole
n=7 Participants
Metronidazole 250 mg 3 times a day
|
High Dose Metronidazole
n=6 Participants
Metronidazole 500 mg 3 times a day
|
|---|---|---|---|---|
|
Change From Baseline in Total Bilirubin Following 12 Weeks Treatment
|
-0.4 mg/dl
Interval -0.9 to 0.2
|
0.05 mg/dl
Interval -0.8 to 9.7
|
-0.3 mg/dl
Interval -0.5 to 0.0
|
0.05 mg/dl
Interval -0.5 to 0.4
|
SECONDARY outcome
Timeframe: baseline, 12 weeksPopulation: Per protocol analysis
The Mayo PSC risk score was calculated for each patient at baseline and at 12 weeks, where Risk = 0.03 (age \[years\]) + 0.54 Ln (total bilirubin \[mg/dL\]) + 0.54 Ln (AST \[IU/L\]) + 1.24 (variceal bleeding) - 0.84 (albumin \[g/dL\]). There is no range, minimum, or maximum value but greater values indicate worse disease.
Outcome measures
| Measure |
Low Dose Vancomycin
n=7 Participants
Vancomycin 125 mg 4 times a day
|
High Dose Vancomycin
n=8 Participants
Vancomycin 250 mg 4 times a day
|
Low Dose Metronidazole
n=7 Participants
Metronidazole 250 mg 3 times a day
|
High Dose Metronidazole
n=6 Participants
Metronidazole 500 mg 3 times a day
|
|---|---|---|---|---|
|
Change From Baseline in Mayo PSC Risk Score Following 12 Weeks of Treatment
|
-0.65 units on a scale
Interval -1.13 to 0.21
|
-0.01 units on a scale
Interval -0.71 to 1.15
|
-0.26 units on a scale
Interval -0.75 to 0.09
|
-0.20 units on a scale
Interval -0.64 to 0.09
|
SECONDARY outcome
Timeframe: baseline, 12 weeksPopulation: Per protocol analysis done
Outcome measures
| Measure |
Low Dose Vancomycin
n=7 Participants
Vancomycin 125 mg 4 times a day
|
High Dose Vancomycin
n=8 Participants
Vancomycin 250 mg 4 times a day
|
Low Dose Metronidazole
n=7 Participants
Metronidazole 250 mg 3 times a day
|
High Dose Metronidazole
n=6 Participants
Metronidazole 500 mg 3 times a day
|
|---|---|---|---|---|
|
Change From Baseline in C-Reactive Protein Following 12 Weeks of Treatment
|
-2.2 mg/L
Interval -47.3 to 0.0
|
1 mg/L
Interval -5.6 to 28.6
|
-3.6 mg/L
Interval -17.4 to 0.0
|
0 mg/L
Interval -1.9 to 2.2
|
Adverse Events
Low Dose Vancomycin
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
High Dose Vancomycin
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Low Dose Metronidazole
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
High Dose Metronidazole
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Low Dose Vancomycin
n=8 participants at risk
Vancomycin 125 mg 4 times a day
|
High Dose Vancomycin
n=9 participants at risk
Vancomycin 250 mg 4 times a day
|
Low Dose Metronidazole
n=9 participants at risk
Metronidazole 250 mg 3 times a day
|
High Dose Metronidazole
n=9 participants at risk
metronidazole 500 mg 3 times a day
|
|---|---|---|---|---|
|
Gastrointestinal disorders
diarrhea
|
12.5%
1/8 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
0.00%
0/9
|
11.1%
1/9 • Number of events 1
|
|
Gastrointestinal disorders
nausea
|
0.00%
0/8
|
11.1%
1/9 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
66.7%
6/9 • Number of events 6
|
|
Gastrointestinal disorders
anorexia
|
0.00%
0/8
|
0.00%
0/9
|
0.00%
0/9
|
22.2%
2/9 • Number of events 2
|
|
Gastrointestinal disorders
metallic taste
|
0.00%
0/8
|
0.00%
0/9
|
22.2%
2/9 • Number of events 2
|
22.2%
2/9 • Number of events 2
|
|
Nervous system disorders
numbness and tingling of extremities
|
12.5%
1/8 • Number of events 1
|
0.00%
0/9
|
0.00%
0/9
|
33.3%
3/9 • Number of events 3
|
|
Gastrointestinal disorders
stomach pain
|
0.00%
0/8
|
0.00%
0/9
|
11.1%
1/9 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
|
Eye disorders
burning in eyes
|
0.00%
0/8
|
0.00%
0/9
|
0.00%
0/9
|
11.1%
1/9 • Number of events 1
|
|
Metabolism and nutrition disorders
weight loss
|
0.00%
0/8
|
0.00%
0/9
|
0.00%
0/9
|
11.1%
1/9 • Number of events 1
|
|
General disorders
increased fatigue
|
0.00%
0/8
|
11.1%
1/9 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
|
General disorders
malaise
|
0.00%
0/8
|
0.00%
0/9
|
11.1%
1/9 • Number of events 1
|
0.00%
0/9
|
|
Hepatobiliary disorders
increased bilirubin
|
0.00%
0/8
|
22.2%
2/9 • Number of events 2
|
0.00%
0/9
|
0.00%
0/9
|
|
Gastrointestinal disorders
dry mouth
|
0.00%
0/8
|
11.1%
1/9 • Number of events 1
|
0.00%
0/9
|
0.00%
0/9
|
|
Nervous system disorders
vertigo
|
12.5%
1/8 • Number of events 1
|
0.00%
0/9
|
0.00%
0/9
|
0.00%
0/9
|
|
Skin and subcutaneous tissue disorders
increased itching
|
12.5%
1/8 • Number of events 1
|
0.00%
0/9
|
0.00%
0/9
|
0.00%
0/9
|
|
Nervous system disorders
headaches
|
12.5%
1/8 • Number of events 1
|
0.00%
0/9
|
0.00%
0/9
|
0.00%
0/9
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place