Join a Current Trial

National Institutes of Health PSC Trials

The National Institutes of Health (NIH) offers a web site of clinical trials specific to PSC. You can visit the site by clicking here.

At this time the following research trials are recruiting participants who have been diagnosed with PSC. These trials may or may not be related to the NIH trials.

Oral Vancomycin Trial at Stanford University Medical Center Now Recruiting

Title: Treatment of Primary Sclerosing Cholangitis in Inflammatory Bowel Disease Patients With Oral Vancomycin by the Study of Its Antimicrobial and Immunomodulating Effects

The purpose of this study is to evaluate changes in the fecal and salivary microbiota during vancomycin treatment of children and adults with Primary Sclerosing Cholangitis (PSC), identify features of the host microbiota that are associated with disease activity and/or response to treatment and further delineate the immunological effects of oral vancomycin treatment of PSC. This study will correlate changes in microbiota with the immunological effects of oral vancomycin in children and adults with PSC. The results of this proposal will lead to new and validated targets for diagnosis and treatment of PSC that will have high impact in the short and long term for patients and their families. 

Determine the benefit of oral vancomycin therapy for Primary Sclerosing Cholangitis within 3 months of therapy.  Determine the benefit of oral vancomycin therapy for Primary Sclerosing Cholangitis. Blood tests (liver enzymes - ALT and GGT), imaging studies (MRI, ERCP) and/or liver biopsy changes before and while on oral vancomycin will assess the benefit of the therapy.

More information on this study, including contact information, may be found at: www.clinicaltrials.gov/ct2/show/NCT01802073

Multicenter Trial of UDCA in Pediatric PSC Now Recruiting

Title: Ursodeoxycholic Acid Therapy in Pediatric Primary Sclerosing Cholangitis: A pilot Withdrawal/Reinstitution Trial (WUP PSC)

Primary sclerosing cholangitis (PSC), a devastating and insidiously progressive cholestatic liver disease, results from advancing inflammation, fibrosis and obliteration of the intra- and extrahepatic bile ducts, leading to cirrhosis and end-stage liver disease. PSC is an uncommon disorder (prevalence in the US of 8-14/100,000 with even lower prevalence in children). Although prognosis in children may be somewhat better, approximately one third of pediatric patients require transplantation by adulthood. Other than transplantation, there is to date no therapy conclusively proven to improve the long-term outcome. Ursodeoxycholic acid (UDCA) improves biochemical markers of liver disease, although in high doses does not clearly improve the long-term outcome in adults. Furthermore, a recent large adult trial of high-dose UDCA therapy suggested a higher incidence of serious adverse events and poor outcomes with UDCA treatment, leading many centers to discontinue UDCA therapy in adult patients. Childhood PSC is different from the adult disease including a stronger association with both autoimmune markers and histologic features and a trend to higher transaminases at diagnosis. Furthermore, in response to intermediate-dose UDCA therapy, there is a more striking and prompt improvement in biochemistries as compared to adults. In light of the prompt normalization of liver enzymes and the fact that UDCA is well tolerated in children, pediatric hepatologists are reluctant to generalize the adult UDCA study results to children and to stop UDCA therapy. This presents a significant dilemma: Should UDCA therapy be stopped in pediatric PSC patients to avoid a possible adverse influence on long-term prognosis at the risk of losing a possible beneficial effect on disease progression in children? Additional factors in children with PSC/autoimmune hepatitis (AIH) overlap are the long-term adverse effects of corticosteroids and azathioprine use. If UDCA therapy is effective as monotherapy, these complications may be avoided. Therefore, we are conducting a preliminary UDCA withdrawal and reinstitution trial sponsored by the FDA Office of Orphan Product Development in pediatric PSC patients to collect data to support the design of a larger, longer-term randomized, placebo-controlled trial of UDCA therapy in childhood PSC. This pilot study, which utilizes the infrastructure and participating centers of the STOPSC (Studies of Primary Sclerosing Cholangitis) consortium, will test the following hypotheses: 1) UDCA therapy yields a rapid biochemical response in children with PSC, thus withdrawal would lead to increased biochemical evidence of disease. 2) UDCA therapy suppresses liver and biliary inflammation in children with PSC, thus withdrawal of therapy would result in a burst of inflammatory activity and an increase in serum cytokine biomarkers, 3) Biochemical control of childhood PSC with histologic features of AIH is dependent upon treatment with immunosuppression in addition to UDCA, therefore childhood PSC without histologic features of AIH will worsen significantly with UDCA withdrawal compared to PSC with histological features of AIH.

More information on this study, including a list of participating centers and contact information, may be found at:
www.clinicaltrials.gov/ct2/show/NCT01088607

For now, participants must live near a participating center.

Noninvasive Assessment of Disease Progression in Primary Sclerosing Cholangitis

Dr. Everson and his colleagues at the University of Colorado Denver have pioneered the development of noninvasive tests to assess liver function as a way to identify the patients with more severe disease or patients whose disease is progressing. The tests have previously been studied primarily in patients with chronic hepatitis C. The main test involves oral and intravenous administration of naturally-occurring compounds, cholates, that are labeled with stable (not radioactive) isotopes (13C or 2H). Five blood samples are obtained over 90 minutes after the administration of cholates and serum analyzed for the concentrations of cholates. Hepatic blood flow, portal blood flow, and portal systemic shunt are estimated from the data. In patients with PSC the fibrosis accumulating in the liver causes portal hypertension, splenic enlargement, and varices. Changes in the portal circulation likely occur very early in the course of PSC and can be quantified by the cholate test. For this reason, the goal of the research is to study the cholate test in patients with PSC to determine if cholate testing can identify the patients with more severe disease and track disease progression. Participants in the research will be studied twice within one month (baseline) to define reproducibility of the test and then studied once more after one year to define the rate of disease progression. This research could provide a basis for considering noninvasive testing in the assessment of disease severity and tracking disease progression in patients with PSC.

If you think you might like to participate in this research study, please call Jennifer DeSanto RN at 303-724-1861.

PROGRESS (PSC Resource Of Genetic Risk, Environment and Synergy Studies)

The Mayo Clinic is conducting a trial that is looking for participants who have PSC. The research website lists goals and participation requirements; click here for more information.

The study asks for a small blood sample, and asks participants to fill out a questionnaire, all to examine genetic and environmental factors relating to PSC.  For more information about this trial, please contact Erik Schlicht at Schlicht.erik@mayo.edu or call 507-284-4312.

PSC Study at University of California/Davis

Dr. Eric Gershwin and Christopher Bowlus of the University of California are beginning a new study for PSC and PBC patients: Prevalence And Clinical Utility Of pANCA, ASCA, ANTI-OMPC, AND ANTI-CBIR1 In Patients With Primary Sclerosing Cholangitis.

To participate, subjects must be 18 years or older, have a diagnosis of PSC and not have had a transplant. They must provide a release of medical information along with a colonoscopy report and MRI or ERC documenting their PSC. Participation also requires a blood sample to be shipped to the researchers. The team has other studies that require fresh blood for immunologic study. Any patients in Northern California that would like to participate can contact Elizabeth Pickett Mathis at

• elizabeth.pickett@ucdmc.ucdavis.edu

• Call 916-734-8246 to arrange for an appointment at the center:
  University of California, Davis Medical Center
  TICON I Building
  2000 Stockton Blvd. Suite 100
  Sacramento, CA 95817

The university study does not have funding for collecting blood at other locations. The participant must be able to travel to Sacramento, unless the patient's doctor is interested in coordinating the patient's blood donation with Dr. Bowlus.

Different T-cells have specialized receptors to match specific antigens. Dr. Bowlus and Dr. Gershwin will study T-cells in PSC (and PBC) patients to determine if their blood displays a high concentration of T-cells with the same receptors. The goal of the study is to explore and identify a correlation, if any, between certain types of T-cells and PBC and PSC.

This is particularly important research. If the doctors identify a specific type of T-cell that is especially concentrated in PSC patients, we will be one big step closer to finding the cause of and perhaps the cure for PSC.

PSC in Twins

Genetically identical twin pairs in which one or both siblings have PSC are an enormous resource to determine the environmental factors contributing to the disease. Researchers at the Division of Rheumatology at UC Davis are searching for patients with PSC that have a twin, whether identical or fraternal. Dr. Carlo Selmi is leading a study on these issues and is interested in such twins whether they both have PSC or not and whether they are identical or not. The search for twins is ongoing throughout the world, if you know of anybody who is a patient with PSC and has a twin, please contact us using the email below. The study will only include a blood draw and a simple questionnaire for both twins.

• cfselmi@ucdavis.edu