A Research Study to Evaluate Safety and Efficacy of DUR-928 in Subjects With Primary Sclerosing Cholangitis (PSC)

Study Overview
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Brief Summary
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This is a research trial testing DUR-928 (an experimental medication). The purpose of the trial is to assess whether treatment with DUR-928 has any effect on the treatment of Primary Sclerosing Cholangitis (PSC). This trial will also assess safety (side effects).

Official Title
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A Randomized, Open-label, Phase 2 Study to Evaluate Safety and Efficacy of DUR-928 in Subjects With Primary Sclerosing Cholangitis (PSC)

Conditions
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Primary Sclerosing Cholangitis

Intervention / Treatment
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* Drug: DUR-928


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Verified diagnosis of primary sclerosing cholangitis (PSC) for at least 12 months, with or without inflammatory bowel disease (IBD). Serum alkaline phosphatase (ALP) ≥ 1.5 times ULN and with no >15% fluctuation in the past 3 months. In subjects receiving treatment with ursodeoxycholic acid (UDCA), therapy must be stable for at least 3 months and at a dose not greater than 20 mg/kg/day. Subjects of childbearing potential must agree to use a medically acceptable method of contraceptive to prevent pregnancy in the subject and/or the partner for the duration of their participation in the trial up to 2 months after the last study drug dosing. Exclusion Criteria: Presence of documented secondary sclerosing cholangitis or small duct PSC Bacterial cholangitis within 30 days prior to Screening Presence of percutaneous drain or endoscopic bile duct stent History of, or suspicion of cholangiocarcinoma. Prior liver transplantation, or currently listed for liver transplantation Presence of other concomitant liver diseases Moderate to Severe active IBD or flare in colitis activity within the last 3 months Any severe and/or untreated concomitant cardiovascular, renal, endocrine or psychiatric disorder Any active malignant disease (within 3 years), other than non-melanomatous skin cancer Human immunodeficiency virus (HIV) infection Existing or intended pregnancy, or breast feeding Has received medication from another clinical trial within the past 30 days

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: DUR-928 10 mg<br>10 mg oral suspension | Drug: DUR-928<br>* oral suspension daily for 28 days<br>|
| Experimental: DUR-928 50 mg<br>50 mg oral suspension | Drug: DUR-928<br>* oral suspension daily for 28 days<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent Change of Alkaline Phosphatase (ALP) From Baseline |  | Day 28 (end of treatment) and Day 56 (end of study/early termination) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent Change of Liver Enzymes and Serum Bile Acids (sBA) | Liver enzymes include alanine transaminase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT), bilirubin. Percent change from baseline through the end of study treatment (Day 28) and throughout the follow-up period (Day 56). | Day 28 and Day 56 | 
| Percent of Subjects With Reduction of Serum Alkaline Phosphatase (ALP) From Baseline |  | Day 28 (end of treatment) and Day 56 (end of follow-up) | 
| Percent Change of Selected Biomarkers From Baseline Through the End of Study Treatment and Throughout the Follow-up Period. |  | Day 28 and Day 56 |