Sulfasalazine for the Treatment of Primary Sclerosing Cholangitis

• Status: Recruiting
• Trial end date: 2020-12-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a multicenter, randomized, double-blinded placebo controlled trial to assess the benefit of sulfasalazine in the treatment of PSC. The specific objectives of this study are to determine if sulfasalazine treatment 1) results in reduced serum ALP and other biomarkers of liver injury in PSC; 2) improves PSC patient symptoms; and 3) is safe in patients with PSC.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Brigham and Women's Hospital
• Treatments: Sulfasalazine

Criteria

Inclusion Criteria:

1. Age 15-80

2. A diagnosis of PSC for at least 6 months based upon cholangiography (ERCP or MRCP) demonstrating intrahepatic and/or extrahepatic biliary strictures, beading or irregularity consistent with PSC.

3. ALP > 1.67 times the upper limit of normal (ULN) at screening

4. Inflammatory bowel disease

5. Subject must either be on a stable dose of ursodeoxycholic acid for > 6 months prior to screening or have been discontinued > 4 weeks prior to screening (enrollment of patients who are on UDCA will be limited to 50% of all enrolled patients).

Exclusion Criteria:

1. Anticipated need for liver transplant within one year as determined by Mayo PSC risk score treatment

2. Evidence of decompensated liver disease such as variceal bleeding, ascites, or hepatic encephalopathy.

3. Evidence of advanced liver disease including MELD score > 10, bilirubin > 3.0, platelet count < 100,000; or INR > 1.4

4. Concomitant chronic liver disease including alcohol related liver disease, chronic hepatitis B or C infection, haemochromatosis, Wilson's disease, alpha1-antitrypsin deficiency, non-alcoholic steatohepatitis, autoimmune hepatitis, or primary biliary cholangitis

5. Secondary causes of sclerosing cholangitis

6. Known intolerance to sulfasalazine (including but not limited to allergy to sulfa or mesalamine) or folic acid

7. History of cholangiocarcinoma or colon cancer within 5 years

8. History of colectomy with > 1/3 bowel resected

9. Treatment with any investigational agents, within two months or 5 half-lives of the investigational product, whichever is longer.

10. Active illicit drug or alcohol abuse

11. Current or past use of sulfasalazine within 6 months of enrollment.

12. Treatment with antibiotics within 3 months of start of medication

13. Need for chronic use of antibiotics

14. Evidence of bacterial cholangitis within 6 months of enrollment

15. In patients with Ulcerative Colitis, simple clinical colitis activity index of > 4 or, if Crohn's disease, a Harvey-Bradshaw index of > 5

16. Chronic kidney injury (eGFR < 59)

17. Pregnancy or lactation