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Trial record 18 of 29 for:    Recruiting, Not yet recruiting Studies | Primary Sclerosing Cholangitis

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

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ClinicalTrials.gov Identifier: NCT03359174
Recruitment Status : Recruiting
First Posted : December 2, 2017
Last Update Posted : June 29, 2018
Sponsor:
Information provided by (Responsible Party):
Yale University

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.

Condition or disease Intervention/treatment Phase
Cholangitis, Sclerosing Drug: All-trans retinoic acid Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Efficacy Trial of Low Dose All-trans Retinoic Acid (ATRA) in Patients With Primary Sclerosing Cholangitis
Actual Study Start Date : May 29, 2018
Estimated Primary Completion Date : December 1, 2020
Estimated Study Completion Date : January 1, 2021


Arm Intervention/treatment
Experimental: All-trans retinoic acid (ATRA) therapy
Fixed low dose of ATRA 10 mg twice daily for 24 weeks.
Drug: All-trans retinoic acid
Fixed low dose of ATRA 10 mg twice daily for 24 weeks.




Primary Outcome Measures :
  1. Percent change of serum alkaline phosphatase (ALP) [ Time Frame: Baseline to week 24. ]
    Blood will be drawn at each time point to compare pre- and post-treatment values for each individual.


Secondary Outcome Measures :
  1. The percent of patients who have normalization of serum ALP or reduction to less than 1.5 x upper limit of normal (ULN) [ Time Frame: Baseline to week 24. ]
    Blood is drawn at each time point to assess the outcome.

  2. The percent of patients who have reduction of serum C4 by 50% [ Time Frame: Baseline to week 24. ]
    Blood is drawn at each time point to assess the outcome.

  3. The percent of patients who have reduction of serum bile acids by 50% [ Time Frame: Baseline to week 24. ]
    Blood is drawn at each time point to assess the outcome.

  4. The percent of patients who have reduction of serum alanine aminotransferase (ALT) by 50% [ Time Frame: Baseline to week 24. ]
    Blood is drawn at each time point to assess the outcome.

  5. The percent of patients who have reduction of serum enhanced liver fibrosis score (ELF) by 10% [ Time Frame: Baseline to week 24. ]
    Blood is drawn at each time point to assess the outcome.

  6. The percent of patients who have improvement in fibrosis per Transient Elastography by at least 1 stage [ Time Frame: Baseline to week 24. ]
    Transient Elastography will be performed at baseline and week 24 to assess the outcome.



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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03359174


Contacts
Contact: James L. Boyer, MD 203-785-5279 james.boyer@yale.edu

Locations
United States, Connecticut
Yale University Recruiting
New Haven, Connecticut, United States, 06510
Sponsors and Collaborators
Yale University
Investigators
Principal Investigator: James Boyer, MD Yale University

Responsible Party: Yale University
ClinicalTrials.gov Identifier: NCT03359174     History of Changes
Other Study ID Numbers: 2000021447
First Posted: December 2, 2017    Key Record Dates
Last Update Posted: June 29, 2018
Last Verified: June 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Cholangitis
Cholangitis, Sclerosing
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Tretinoin
Antineoplastic Agents
Keratolytic Agents
Dermatologic Agents