Combination Therapy With Ursodeoxycholic Acid (UDCA) and All-Trans Retinoic Acid (ATRA) for Treatment of Primary Sclerosing Cholangitis

• Register: ClinicalTrials.gov
• Last refreshed on: 8 August 2016
• Main ID: NCT01456468
• Date of registration: 14/10/2011
• Prospective Registration: No
• Primary sponsor: Yale University
• Public title: Combination Therapy With Ursodeoxycholic Acid (UDCA) and All-Trans Retinoic Acid (ATRA) for Treatment of Primary Sclerosing Cholangitis
• Scientific title: Combination Therapy With Ursodeoxycholic Acid (UDCA) and All-Trans Retinoic Acid (ATRA) for Treatment of Primary Sclerosing Cholangitis - A Human Pilot Study
• Date of first enrolment: October 2011
• Target sample size: 19
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01456468
• Study type: Interventional
• Study design: Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: James L Boyer, MD
• Affiliation: Yale University

Key inclusion & exclusion criteria

Inclusion Criteria:

- Diagnosis of PSC for at least 6 months, made by clinical evaluation in addition to
one of the following: a prior endoscopic retrograde cholangiography (ERC), magnetic
resonance cholangiography (MRC, also termed MRI/MRCP) or liver biopsy.

- Progressing disease or stable disease with persistent elevation in AP despite
treatment with UDCA (15 mg/kg/day) for at least 6 months.

- Measures of progressing disease:

1. Cholangitis within the past 12 months.

2. Presence or progression of biliary abnormalities on MRI/MRC.

3. Elevated liver tests (alkaline phosphatase, bilirubin, aspartate
aminotransferase [AST], alanine aminotransferase [ALT]).

- Age between 18 and 80.

Exclusion Criteria:

- Pregnancy or planned pregnancy during study period and within 6 months of study
completion.

- Common Terminology Criteria for Adverse Events (CTCAE) grade 3 or higher cardiac
disease, hyperlipidemia, hypertriglyceridemia, hepatic injury, or adverse event
related to administration of UDCA or ATRA.

- Prior intolerance to UDCA or ATRA (or related oral vitamin A compounds).

- Evidence of decompensated cirrhosis within the past 6 months (i.e. variceal bleeding,
uncontrolled ascites, hepatic encephalopathy, jaundice).

- Estimated need for liver transplantation within 1 year.

- Any evidence of hepatocellular carcinoma, cholangiocarcinoma, or other malignancy.



Age minimum: 18 Years
Age maximum: 80 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Cholangitis, Sclerosing

Intervention(s)

Drug: Oral all-trans retinoic acid (ATRA)

Primary Outcome(s)

Improvement in serum alkaline phosphatase levels [Time Frame: Baseline and after 3 months of treatment.]

Secondary ID(s)

1012007734

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Mayo Clinic