Trial Outcomes & Findings for Gastrointestinal Microbiota in Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin (NCT NCT01322386)
NCT ID: NCT01322386
Last Updated: 2016-05-18
Results Overview
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.
COMPLETED
PHASE1
32 participants
Within 3 months of therapy
2016-05-18
Participant Flow
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.
11 PSC patients whose consent to participate was obtained did not participate in the Study.
Participant milestones
| Measure |
Oral Vancomycin-Biliary Atresia
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.
|
Oral Vancomycin/ Primary Sclerosing Cholangitis
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.
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
11
|
|
Overall Study
COMPLETED
|
10
|
9
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
Reasons for withdrawal
| Measure |
Oral Vancomycin-Biliary Atresia
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.
|
Oral Vancomycin/ Primary Sclerosing Cholangitis
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.
|
|---|---|---|
|
Overall Study
Protocol Violation
|
0
|
1
|
|
Overall Study
Physician Decision
|
0
|
1
|
Baseline Characteristics
Gastrointestinal Microbiota in Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin
Baseline characteristics by cohort
| Measure |
Oral Vancomycin-BIliary Atresia
n=10 Participants
Enrolled- 10, Participated - 10,Completed - 10 (Based on Annual Progress Report in Oct 2010)
|
Oral Vancomycin - PSC
n=11 Participants
Enrolled- 11, Participated - 10 ,Completed - 9 (Based on Annual Progress Report in Oct 2010)
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
1-3 months
|
10 participants
n=5 Participants
|
0 participants
n=7 Participants
|
10 participants
n=5 Participants
|
|
Age, Customized
2-4 years
|
0 participants
n=5 Participants
|
3 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Age, Customized
9-11 years
|
0 participants
n=5 Participants
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Age, Customized
12-16 years
|
0 participants
n=5 Participants
|
6 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
5 participants
n=5 Participants
|
10 participants
n=7 Participants
|
15 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic
|
3 participants
n=5 Participants
|
1 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
African American
|
2 participants
n=5 Participants
|
0 participants
n=7 Participants
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Within 3 months of therapyPopulation: 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.
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.
Outcome measures
| Measure |
Liver Blood Test
n=19 Participants
BA -10/10 , PSC - 9/9 - All had improvement on Vancomycin therapy.
|
MRI / MRCP
n=7 Participants
BA-N/A, PSC - 7/7 showed improvement on Vancomycin therapy.
|
Liver Biopsy
n=4 Participants
BA - N/A , PSC - 4/4 who had Liver biopsies showed improvement on Vancomycin therapy.
|
Colon Biopsies
n=8 Participants
BA - N/A , PSC - 8/8- who had colonoscopies with colon biopsies showed improvement on Vancomycin therapy.
|
Liver Biopsy and/or MRI
n=9 Participants
BA-N/A , PSC- 8/9 participants showed improvement of liver biopsies and/or MRI on Vancomycin.
|
|---|---|---|---|---|---|
|
Determine the Benefit of Oral Vancomycin Therapy for Primary Sclerosing Cholangitis and Biliary Atresia
"BA (n=10,0,0,0,0)"
|
10 participants
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Determine the Benefit of Oral Vancomycin Therapy for Primary Sclerosing Cholangitis and Biliary Atresia
"PSC (n=9,7,4,8,8)"
|
9 participants
|
7 participants
|
4 participants
|
8 participants
|
8 participants
|
Adverse Events
BA/PSC -Oral Vancomycin
Serious adverse events
| Measure |
BA/PSC -Oral Vancomycin
n=21 participants at risk
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.
|
|---|---|
|
Nervous system disorders
Non-malignant Spinal Cord Tumor
|
4.8%
1/21 • Number of events 1 • 2 years
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.
|
Other adverse events
Adverse event data not reported
Additional Information
Kenneth L Cox, MD / Principal Investigator
Stanford University
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place