An Efficacy Trial of Low Dose All-trans Retinoic Acid in Patients With Primary Sclerosing Cholangitis

NCT ID: NCT03359174

Last Updated: 2021-08-09

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 2 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-05-29
• Study Completion Date: 2021-01-01

Brief Summary

The purpose of this research study is to determine whether a low dose of ATRA will improve laboratory tests of liver and bile duct inflammation in patients with PSC. The investigators will also look for changes to other blood tests which are related to inflammation, scarring, and the immune system.

Conditions

Cholangitis, Sclerosing

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

All-trans retinoic acid (ATRA) therapy

Fixed low dose of ATRA 10 mg twice daily for 24 weeks.

Group Type: EXPERIMENTAL

All-trans retinoic acid

Intervention Type: DRUG

Fixed low dose of ATRA 10 mg twice daily for 24 weeks.

Interventions

All-trans retinoic acid

Fixed low dose of ATRA 10 mg twice daily for 24 weeks.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Males and females ages 18-80
• Diagnosis of large-duct PSC based on ERCP or MRCP, or liver biopsy findings without alternative explanation for findings, for at least 6 months.
• Serum ALP levels persistently more than 1.5 x upper limit of normal over the past 6 months.
• Ursodeoxycholic acid therapy must be discontinued for at least 3 months.
• At least 2 forms of barrier protection for males and females of child-bearing age.

Exclusion Criteria

• Small duct PSC, overlap with autoimmune hepatitis, IgG4 disease or secondary sclerosing cholangitis.
• Any malignancy, presently or within the past 5 years, except adequately treated non-melanoma skin cancer, carcinoma in situ of the cervix or in situ prostate cancer.
• Viral hepatitis including hepatitis A, B, C, D, E.
• Decompensated cirrhosis, or planned liver transplantation.
• Recent diagnostic or therapeutic biliary manipulation (endoscopic, radiologic) within the past 3 months.
• Ascending Cholangitis requiring antibiotics within the past 3 months.
• Uncontrolled IBD, or IBD requiring the use of steroids.
• Acute or Chronic Kidney Disease with serum creatinine > 2 mg/dL.
• Allergy to ATRA or vitamin A compounds.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Yale University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

James Boyer, MD

Role: PRINCIPAL_INVESTIGATOR

Yale University

Locations

Yale University

New Haven, Connecticut, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

2000021447

Identifier Type: -

Identifier Source: org_study_id