Sulfasalazine for the Treatment of Primary Sclerosing Cholangitis

NCT03561584 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: 2018P000019
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief 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. We are recruiting remotely throughout the United States so an individual anywhere in the US with PSC and IBD can be enrolled.

Conditions

Primary Sclerosing Cholangitis

Keywords

Sclerosing; Cholangitis

Outcome Measures

Primary Outcomes (2)

• Reduction in Mean Alkaline Phosphatase (ALP)

  Proportion of patients with reduction of mean ALP \< 1.5 x ULN at end of treatment

  Baseline through the end of the Study at Week 22

• Normalization of ALP below the upper limit of normal

  Assessment in number of patients whose ALP normalizes

  Baseline through the end of the Study at Week 22

Secondary Outcomes (6)

• Overall changes in ALP levels

  Baseline through the end of the Study at Week 22

• Changes in blood tests

  Baseline through the end of the Study at Week 22

• Adverse Events

  Baseline through the end of the Study at Week 22

• Changes in Mayo PSC risk score

  Baseline through the end of the Study at Week 22

• Changes in Modified Fatigue Scale (MFS)

  Baseline through the end of the Study at Week 22

Study Arms (2)

Active Drug (Sulfasalazine)

ACTIVE COMPARATOR

Drug: Sulfasalazine

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Sulfasalazine DRUG

Patients will be initiated on a low dose of sulfasalazine (500 mg) twice daily (bid). Dosage will be increased throughout the study.

Also known as: Azulfidine

Active Drug (Sulfasalazine)

Placebo DRUG

Patients will be initiated on 1 placebo tablet twice daily (bid). Dosage will be increased throughout the study.

Placebo

Eligibility Criteria

• Age: 15 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 15-80
• 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.
• ALP \> 1.67 times the upper limit of normal (ULN) at screening
• Inflammatory bowel disease
• 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).
• We are recruiting remotely throughout the United States so an individual anywhere in the US with PSC and IBD can be enrolled.

You may not qualify if:

• Anticipated need for liver transplant within one year as determined by Mayo PSC risk score treatment
• Evidence of decompensated liver disease such as variceal bleeding, ascites, or hepatic encephalopathy.
• Evidence of advanced liver disease including MELD score \> 10, bilirubin \> 3.0, platelet count \< 100,000; or INR \> 1.4
• 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
• Secondary causes of sclerosing cholangitis
• Known intolerance to sulfasalazine (including but not limited to allergy to sulfa or mesalamine) or folic acid
• History of cholangiocarcinoma or colon cancer within 5 years
• History of colectomy with \> 1/3 bowel resected
• Treatment with any investigational agents, within two months or 5 half-lives of the investigational product, whichever is longer.
• Active illicit drug or alcohol abuse
• Current or past use of sulfasalazine within 6 months of enrollment.
• Need for chronic use of antibiotics
• Evidence of bacterial cholangitis within 6 months of enrollment
• In patients with Ulcerative Colitis, simple clinical colitis activity index of \> 4 or, if Crohn's disease, a Harvey-Bradshaw index of \> 5
• Chronic kidney injury (eGFR \< 59)

Sponsors & Collaborators

• Brigham and Women's Hospital: lead

Study Sites (1)

Brigham and Women's Hospital

Chestnut Hill, Massachusetts, 02467, United States

Location

MeSH Terms

Conditions

Cholangitis, Sclerosing; Cholangitis

Interventions

Sulfasalazine

Condition Hierarchy (Ancestors)

Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases

Intervention Hierarchy (Ancestors)

Sulfonamides; Amides; Organic Chemicals; Sulfones; Sulfur Compounds

Study Officials

• Joshua R Korzenik, MD

  Brigham and Women's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, CARE PROVIDER
• Masking Details: Participants and Providers will be masked until Week 14. If a subject continues past week 14, the study becomes Open-Label and participants are given the option to continue on the active drug for an additional 8 weeks. We are recruiting remotely throughout the United States so an individual anywhere in the US with PSC and IBD can be enrolled.
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Crohn's and Colitis Center

Model Details: There are two arms in this trial: active drug and placebo. We are recruiting remotely throughout the United States so an individual anywhere in the US with PSC and IBD can be enrolled.

Study Record Dates

• First Submitted: May 14, 2018
• First Posted: June 19, 2018
• Study Start: July 1, 2018
• Primary Completion: March 3, 2026
• Study Completion: March 3, 2026
• Last Updated: March 12, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)