ClinicalTrials.gov
ClinicalTrials.gov Menu

Gastrointestinal Microbiota in Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin (PSC)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01322386
Recruitment Status : Completed
First Posted : March 24, 2011
Results First Posted : March 4, 2016
Last Update Posted : May 18, 2016
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
Conditions Primary Sclerosing Cholangitis
Biliary Atresia
Intervention Drug: Vancomycin
Enrollment 32
Recruitment Details Participants were recruited by the Physician and research team at Lucille Packard and Stanford Hospital and Clinics from 2007 - 2010. The first participant was enrolled in November 2008, and the last participant was enrolled in October 2010.
Pre-assignment Details 11 PSC patients whose consent to participate was obtained did not participate in the Study.
Arm/Group Title Oral Vancomycin-Biliary Atresia Oral Vancomycin/ Primary Sclerosing Cholangitis
Hide Arm/Group Description Vancomycin: Oral 50mg/Kg per day for three months. Initially 10 infants with Biliary Atresia (BA) were planned for this study to determine if there was clinical response to oral vancomycin. Vancomycin: Oral 50mg/Kg per day up to maximum of 1500 mg a day for three months. Initially 10 children with Primary Sclerosing Cholangitis (PSC) were planned for this study to determine if there was clinical response to oral vancomycin.
Period Title: Overall Study
Started 10 11
Completed 10 9
Not Completed 0 2
Reason Not Completed
Protocol Violation             0             1
Physician Decision             0             1
Arm/Group Title Oral Vancomycin-BIliary Atresia Oral Vancomycin - PSC Total
Hide Arm/Group Description Enrolled- 10, Participated - 10,Completed - 10 (Based on Annual Progress Report in Oct 2010) Enrolled- 11, Participated - 10 ,Completed - 9 (Based on Annual Progress Report in Oct 2010) Total of all reporting groups
Overall Number of Baseline Participants 10 11 21
Hide Baseline Analysis Population Description
Based on the annual progress report sent dated 10/04/2010, all patients completed the study as planned except 2 children with PSC- 1 was non-compliant and the other had severe UC requiring Remicade which resulted in removal from Vancomycin study.
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 10 participants 11 participants 21 participants
1-3 months 10 0 10
2-4 years 0 3 3
9-11 years 0 2 2
12-16 years 0 6 6
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 11 participants 21 participants
Female
6
  60.0%
5
  45.5%
11
  52.4%
Male
4
  40.0%
6
  54.5%
10
  47.6%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 10 participants 11 participants 21 participants
Caucasian 5 10 15
Hispanic 3 1 4
African American 2 0 2
1.Primary Outcome
Title Determine the Benefit of Oral Vancomycin Therapy for Primary Sclerosing Cholangitis and Biliary Atresia
Hide Description Determine the benefit of oral vancomycin therapy for Primary Sclerosing Cholangitis and Biliary Atresia through improvement of Liver function tests (LFTs) within 3 months of initiating therapy. In addition for PSC, we looked at 25% reduction of abnormal ALT & GGT, reduction in biliary strictures and beading, and reduction of inflammation in liver biopsies and colon biopsies.
Time Frame Within 3 months of therapy
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Ten BA participants had surgery (Kasai portoenterostomyprocedure) at 1 week before starting the Vancomycin so we could not determine if they benefited from the therapy. On oral vancomycin, 9 PSC patients had improvement of LFTs, 8 had improvement of liver biopsies and/or MRI, and colon biopsies,and 1 pt. refused to have these additional studies.
Arm/Group Title Liver Blood Test MRI / MRCP Liver Biopsy Colon Biopsies Liver Biopsy and/or MRI
Hide Arm/Group Description:
BA -10/10 , PSC - 9/9 - All had improvement on Vancomycin therapy.
BA-N/A, PSC - 7/7 showed improvement on Vancomycin therapy.
BA - N/A , PSC - 4/4 who had Liver biopsies showed improvement on Vancomycin therapy.
BA - N/A , PSC - 8/8- who had colonoscopies with colon biopsies showed improvement on Vancomycin therapy.
BA-N/A , PSC- 8/9 participants showed improvement of liver biopsies and/or MRI on Vancomycin.
Overall Number of Participants Analyzed 19 7 4 8 9
Measure Type: Number
Unit of Measure: participants
"BA (n=10,0,0,0,0)" 10 0 0 0 0
"PSC (n=9,7,4,8,8)" 9 7 4 8 8
Time Frame 2 years
Adverse Event Reporting Description There were no adverse experiences for any body system for any participants related to the study that resulted in patients being taken off the study. Since Oral Vancomycin is poorly absorbed, no serious adverse events were anticipated for this study.
 
Arm/Group Title BA/PSC -Oral Vancomycin
Hide Arm/Group Description Oral Vancomycin is commercially available (Vancocin, ViroPharma Inc.) for treatment of gastrointestinal infection such as, Clostridium difficile infection. In fact, our published observation of using oral vancomycin in treating PSC was an incidental finding to our treatment for Cl. Difficile gastrointestinal infection in a child with PSC (J Pediatr Gastroenterol Nutr 27:580-3, 1998). Since oral vancomycin is poorly absorbed, no serious adverse events were anticipated in this study.
All-Cause Mortality
BA/PSC -Oral Vancomycin
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
BA/PSC -Oral Vancomycin
Affected / at Risk (%) # Events
Total   1/21 (4.76%)    
Nervous system disorders   
Non-malignant Spinal Cord Tumor  [1]  1/21 (4.76%)  1
Indicates events were collected by systematic assessment
[1]
One subject did have a Serious Adverse Event (SAE) that was not related to the study medication or any study procedure. The SAE was for a Non-Malignant Spinal Cord Tumor. This was identified on February 3,2011 and removed on February 18,2011.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
BA/PSC -Oral Vancomycin
Affected / at Risk (%) # Events
Total   0/21 (0.00%)    
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Kenneth L Cox, MD / Principal Investigator
Organization: Stanford University
Phone: 650-723-5070
Responsible Party: Kenneth L. Cox, Stanford University
ClinicalTrials.gov Identifier: NCT01322386     History of Changes
Other Study ID Numbers: 4751
First Submitted: February 10, 2011
First Posted: March 24, 2011
Results First Submitted: May 22, 2015
Results First Posted: March 4, 2016
Last Update Posted: May 18, 2016