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Primary Sclerosing Cholangitis With Oral Vancomycin by the Study of Its Antimicrobial and Immunomodulating Effects (PSC)

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ClinicalTrials.gov Identifier: NCT01802073
Recruitment Status : Completed
First Posted : March 1, 2013
Results First Posted : September 21, 2018
Last Update Posted : September 21, 2018
Sponsor:
Information provided by (Responsible Party):
Kenneth L. Cox, Stanford University

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Primary Sclerosing Cholangitis
Intervention Drug: Oral Vancomycin
Enrollment 34
Recruitment Details  
Pre-assignment Details Two participants in the adult cohort completed the study but were not analyzed for outcomes (1 post-liver transplant patient; 1 completed the study but followed a diet that could influence results analysis). Two adult participants who consented but withdrew prior to treatment were not analyzed for outcomes.
Arm/Group Title Oral Vancomycin-Children Oral Vancomycin-Adults
Hide Arm/Group Description

For children who weight < or = 30 kg, the vancomycin dose will be 50 mg/kg/day given orally 3 times per day for the 1st month and continue with the same dose for subsequent months if the clinical laboratory studies improved and are normal. If the laboratory studies are not normal the dose will be increased to 75 mg/kg/day given orally 3 times per day for the 2nd month and 100mg/kg/day given orally 3 times per day the 3rd month. If the laboratory studies do not improve by the end of the 3rd month since starting the vancomycin, the vancomycin will be stopped and the child will not continue the study.

Oral Vancomycin

For adults and children who weigh >30 kg, the vancomycin dose will be 500 mg given orally 3 times per day for the 1st month and continue with this dose if the clinical laboratory studies improve and are normal. If the laboratory studies are not normal the dose will be increased to 750 mg 3 times per day for the 2nd month and 1000 mg 3 times per day the 3rd month.

Oral Vancomycin

Period Title: Overall Study
Started 14 20
Completed 9 9
Not Completed 5 11
Reason Not Completed
Lost to Follow-up             0             1
Non-compliant             4             2
Withdrawal by Subject             1             1
Physician Decision             0             7
Arm/Group Title Oral Vancomycin-Children Oral Vancomycin-Adults Total
Hide Arm/Group Description

For children who weight < or = 30 kg, the vancomycin dose will be 50 mg/kg/day given orally 3 times per day for the 1st month and continue with the same dose for subsequent months if the clinical laboratory studies improved and are normal. If the laboratory studies are not normal the dose will be increased to 75 mg/kg/day given orally 3 times per day for the 2nd month and 100mg/kg/day given orally 3 times per day the 3rd month. If the laboratory studies do not improve by the end of the 3rd month since starting the vancomycin, the vancomycin will be stopped and the child will not continue the study.

Oral Vancomycin

For adults and children who weigh >30 kg, the vancomycin dose will be 500 mg given orally 3 times per day for the 1st month and continue with this dose if the clinical laboratory studies improve and are normal. If the laboratory studies are not normal the dose will be increased to 750 mg 3 times per day for the 2nd month and 1000 mg 3 times per day the 3rd month.

Oral Vancomycin

Total of all reporting groups
Overall Number of Baseline Participants 14 20 34
Hide Baseline Analysis Population Description
Participants who completed the protocol are included in the analysis
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants 20 participants 34 participants
0-6 years
2
  14.3%
0
   0.0%
2
   5.9%
7-12 years
5
  35.7%
0
   0.0%
5
  14.7%
13-17 years
6
  42.9%
0
   0.0%
6
  17.6%
18-64 years
1
   7.1%
20
 100.0%
21
  61.8%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants 20 participants 34 participants
Female
6
  42.9%
2
  10.0%
8
  23.5%
Male
8
  57.1%
18
  90.0%
26
  76.5%
1.Primary Outcome
Title Count of Participants With Elevated Alanine Aminotransferase (ALT) at Baseline and With Clinically Significant Improvement at Month 3
Hide Description Clinically significant improvement was determined by investigator assessment per participant based on their medical history and disease stage. Elevated ALT was any value greater than the upper limit of the standard reference range used by patient's laboratory.
Time Frame Baseline; Month 3
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with ALT elevated at baseline are included in the analysis.
Arm/Group Title Oral Vancomycin-Children Oral Vancomycin-Adults
Hide Arm/Group Description:

For children who weight < or = 30 kg, the vancomycin dose will be 50 mg/kg/day given orally 3 times per day for the 1st month and continue with the same dose for subsequent months if the clinical laboratory studies improved and are normal. If the laboratory studies are not normal the dose will be increased to 75 mg/kg/day given orally 3 times per day for the 2nd month and 100mg/kg/day given orally 3 times per day the 3rd month. If the laboratory studies do not improve by the end of the 3rd month since starting the vancomycin, the vancomycin will be stopped and the child will not continue the study.

Oral Vancomycin

For adults and children who weigh >30 kg, the vancomycin dose will be 500 mg given orally 3 times per day for the 1st month and continue with this dose if the clinical laboratory studies improve and are normal. If the laboratory studies are not normal the dose will be increased to 750 mg 3 times per day for the 2nd month and 1000 mg 3 times per day the 3rd month.

Oral Vancomycin

Overall Number of Participants Analyzed 10 9
Measure Type: Count of Participants
Unit of Measure: Participants
10
 100.0%
6
  66.7%
2.Primary Outcome
Title Count of Participants With Elevated Gamma-glutamyltransferase (GGT) at Baseline and With Clinically Significant Improvement at Month 3
Hide Description Clinically significant improvement was determined by investigator assessment per participant based on their medical history and disease stage. Elevated GGT was any value greater than the upper limit of the standard reference range used by patient's laboratory.
Time Frame Baseline; Month 3
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with GGT elevated at baseline are included in the analysis.
Arm/Group Title Oral Vancomycin-Children Oral Vancomycin-Adults
Hide Arm/Group Description:

For children who weight < or = 30 kg, the vancomycin dose will be 50 mg/kg/day given orally 3 times per day for the 1st month and continue with the same dose for subsequent months if the clinical laboratory studies improved and are normal. If the laboratory studies are not normal the dose will be increased to 75 mg/kg/day given orally 3 times per day for the 2nd month and 100mg/kg/day given orally 3 times per day the 3rd month. If the laboratory studies do not improve by the end of the 3rd month since starting the vancomycin, the vancomycin will be stopped and the child will not continue the study.

Oral Vancomycin

For adults and children who weigh >30 kg, the vancomycin dose will be 500 mg given orally 3 times per day for the 1st month and continue with this dose if the clinical laboratory studies improve and are normal. If the laboratory studies are not normal the dose will be increased to 750 mg 3 times per day for the 2nd month and 1000 mg 3 times per day the 3rd month.

Oral Vancomycin

Overall Number of Participants Analyzed 8 9
Measure Type: Count of Participants
Unit of Measure: Participants
8
 100.0%
6
  66.7%
3.Primary Outcome
Title Count of Participants With Elevated ALT and/or GGT at Baseline and With Clinically Significant Improvement at Month 3
Hide Description Clinically significant improvement was determined by investigator assessment per participant based on their medical history and disease stage. Elevated ALT (and GGT) was any value greater than the upper limit of the standard reference range used by patient's laboratory.
Time Frame Baseline; Month 3
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with ALT and/or GGT elevated at baseline are included in the analysis.
Arm/Group Title Oral Vancomycin-Children Oral Vancomycin-Adults
Hide Arm/Group Description:

For children who weight < or = 30 kg, the vancomycin dose will be 50 mg/kg/day given orally 3 times per day for the 1st month and continue with the same dose for subsequent months if the clinical laboratory studies improved and are normal. If the laboratory studies are not normal the dose will be increased to 75 mg/kg/day given orally 3 times per day for the 2nd month and 100mg/kg/day given orally 3 times per day the 3rd month. If the laboratory studies do not improve by the end of the 3rd month since starting the vancomycin, the vancomycin will be stopped and the child will not continue the study.

Oral Vancomycin

For adults and children who weigh >30 kg, the vancomycin dose will be 500 mg given orally 3 times per day for the 1st month and continue with this dose if the clinical laboratory studies improve and are normal. If the laboratory studies are not normal the dose will be increased to 750 mg 3 times per day for the 2nd month and 1000 mg 3 times per day the 3rd month.

Oral Vancomycin

Overall Number of Participants Analyzed 10 10
Measure Type: Count of Participants
Unit of Measure: Participants
10
 100.0%
8
  80.0%
4.Primary Outcome
Title Count of Participants With Abnormal Magnetic Resonance Cholangiopancreatography (MRCP) Imaging at Baseline and With Clinically Significant Improvement at Year 1
Hide Description Clinically significant improvement was determined by investigator assessment per participant based on their medical history and disease stage. MRCP imaging was abnormal if it included biliary beading, biliary strictures, dilated bile duct, and/or liver fibrosis.
Time Frame Baseline; Year 1
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with abnormal MRCP at baseline are included in the analysis.
Arm/Group Title Oral Vancomycin-Children Oral Vancomycin-Adults
Hide Arm/Group Description:

For children who weight < or = 30 kg, the vancomycin dose will be 50 mg/kg/day given orally 3 times per day for the 1st month and continue with the same dose for subsequent months if the clinical laboratory studies improved and are normal. If the laboratory studies are not normal the dose will be increased to 75 mg/kg/day given orally 3 times per day for the 2nd month and 100mg/kg/day given orally 3 times per day the 3rd month. If the laboratory studies do not improve by the end of the 3rd month since starting the vancomycin, the vancomycin will be stopped and the child will not continue the study.

Oral Vancomycin

For adults and children who weigh >30 kg, the vancomycin dose will be 500 mg given orally 3 times per day for the 1st month and continue with this dose if the clinical laboratory studies improve and are normal. If the laboratory studies are not normal the dose will be increased to 750 mg 3 times per day for the 2nd month and 1000 mg 3 times per day the 3rd month.

Oral Vancomycin

Overall Number of Participants Analyzed 8 8
Measure Type: Count of Participants
Unit of Measure: Participants
6
  75.0%
4
  50.0%
5.Primary Outcome
Title Count of Participants With Abnormal Liver Biopsies at Baseline and With Clinically Significant Improvement at Year 1
Hide Description Clinically significant improvement was determined by investigator assessment per participant based on their medical history and disease stage. Liver pathology was considered abnormal if the biopsy was S1 or greater on the liver fibrosis staging scale (S0 no fibrosis, S1 mild fibrosis, S2 moderate fibrosis, S3 sever fibrosis, S4 cirrhosis).
Time Frame Baseline; Year 1
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with abnormal liver biopsy at baseline and who had a post-treatment liver biopsy are included in the analysis.
Arm/Group Title Oral Vancomycin-Children Oral Vancomycin-Adults
Hide Arm/Group Description:

For children who weight < or = 30 kg, the vancomycin dose will be 50 mg/kg/day given orally 3 times per day for the 1st month and continue with the same dose for subsequent months if the clinical laboratory studies improved and are normal. If the laboratory studies are not normal the dose will be increased to 75 mg/kg/day given orally 3 times per day for the 2nd month and 100mg/kg/day given orally 3 times per day the 3rd month. If the laboratory studies do not improve by the end of the 3rd month since starting the vancomycin, the vancomycin will be stopped and the child will not continue the study.

Oral Vancomycin

For adults and children who weigh >30 kg, the vancomycin dose will be 500 mg given orally 3 times per day for the 1st month and continue with this dose if the clinical laboratory studies improve and are normal. If the laboratory studies are not normal the dose will be increased to 750 mg 3 times per day for the 2nd month and 1000 mg 3 times per day the 3rd month.

Oral Vancomycin

Overall Number of Participants Analyzed 3 0
Measure Type: Count of Participants
Unit of Measure: Participants
3
 100.0%
0
6.Primary Outcome
Title Count of Participants With Abnormal MRCP and/or Liver Biopsy at Baseline and With Clinically Significant Improvement at Year 1
Hide Description Clinically significant improvement was determined by investigator assessment per participant based on their medical history and disease stage. MRCP imaging was abnormal if it included biliary beading, biliary strictures, dilated bile duct, and/or liver fibrosis. Liver pathology was considered abnormal if the biopsy was S1 or greater on the liver fibrosis staging scale (S0 no fibrosis, S1 mild fibrosis, S2 moderate fibrosis, S3 sever fibrosis, S4 cirrhosis).
Time Frame Baseline; Year 1
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with abnormal MRCP and/or liver biopsy at baseline are included in the analysis.
Arm/Group Title Oral Vancomycin-Children Oral Vancomycin-Adults
Hide Arm/Group Description:

For children who weight < or = 30 kg, the vancomycin dose will be 50 mg/kg/day given orally 3 times per day for the 1st month and continue with the same dose for subsequent months if the clinical laboratory studies improved and are normal. If the laboratory studies are not normal the dose will be increased to 75 mg/kg/day given orally 3 times per day for the 2nd month and 100mg/kg/day given orally 3 times per day the 3rd month. If the laboratory studies do not improve by the end of the 3rd month since starting the vancomycin, the vancomycin will be stopped and the child will not continue the study.

Oral Vancomycin

For adults and children who weigh >30 kg, the vancomycin dose will be 500 mg given orally 3 times per day for the 1st month and continue with this dose if the clinical laboratory studies improve and are normal. If the laboratory studies are not normal the dose will be increased to 750 mg 3 times per day for the 2nd month and 1000 mg 3 times per day the 3rd month.

Oral Vancomycin

Overall Number of Participants Analyzed 10 9
Measure Type: Count of Participants
Unit of Measure: Participants
10
 100.0%
4
  44.4%
Time Frame Up to 2 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Oral Vancomycin-Children Oral Vancomycin-Adults
Hide Arm/Group Description

For children who weight < or = 30 kg, the vancomycin dose will be 50 mg/kg/day given orally 3 times per day for the 1st month and continue with the same dose for subsequent months if the clinical laboratory studies improved and are normal. If the laboratory studies are not normal the dose will be increased to 75 mg/kg/day given orally 3 times per day for the 2nd month and 100mg/kg/day given orally 3 times per day the 3rd month. If the laboratory studies do not improve by the end of the 3rd month since starting the vancomycin, the vancomycin will be stopped and the child will not continue the study.

Oral Vancomycin

For adults and children who weigh >30 kg, the vancomycin dose will be 500 mg given orally 3 times per day for the 1st month and continue with this dose if the clinical laboratory studies improve and are normal. If the laboratory studies are not normal the dose will be increased to 750 mg 3 times per day for the 2nd month and 1000 mg 3 times per day the 3rd month.

Oral Vancomycin

All-Cause Mortality
Oral Vancomycin-Children Oral Vancomycin-Adults
Affected / at Risk (%) Affected / at Risk (%)
Total   0/14 (0.00%)   0/20 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Oral Vancomycin-Children Oral Vancomycin-Adults
Affected / at Risk (%) Affected / at Risk (%)
Total   1/14 (7.14%)   0/20 (0.00%) 
Hepatobiliary disorders     
Biliary stricture requiring stent   1/14 (7.14%)  0/20 (0.00%) 
Progression of PSC   1/14 (7.14%)  0/20 (0.00%) 
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Oral Vancomycin-Children Oral Vancomycin-Adults
Affected / at Risk (%) Affected / at Risk (%)
Total   0/14 (0.00%)   0/20 (0.00%) 
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Kenneth Cox
Organization: Stanford University
Phone: (650) 723-5070
EMail: KCox@stanfordchildrens.org
Layout table for additonal information
Responsible Party: Kenneth L. Cox, Stanford University
ClinicalTrials.gov Identifier: NCT01802073     History of Changes
Other Study ID Numbers: 22591
First Submitted: February 21, 2013
First Posted: March 1, 2013
Results First Submitted: July 20, 2018
Results First Posted: September 21, 2018
Last Update Posted: September 21, 2018