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A Study of Oral Hymecromone to Treat Adults With Primary Sclerosing Cholangitis

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ClinicalTrials.gov Identifier: NCT02780752
Recruitment Status : Not yet recruiting
First Posted : May 23, 2016
Last Update Posted : February 8, 2018
Sponsor:
Information provided by (Responsible Party):
Kenneth L. Cox, Stanford University

Brief Summary:
Primary sclerosing cholangitis (PSC) is a chronic inflammatory disease of the biliary ducts in the liver resulting in destruction and fibrosis. PSC is associated with increased serum levels of hyaluronan (HA), an extracellular matrix polymer known to drive inflammation in multiple other tissues and disease models. Inhibition of HA synthesis with oral hymecromone has been shown to prevent progression of autoimmunity in several animal models. This is a prospective, open-label, single-center, pilot safety study of hymecromone in adults 18 years of age or older diagnosed with primary sclerosing cholangitis without evidence of hepatic fibrosis. Participants will be treated for 6 months with study drug. Safety and biomarkers of response will be monitored during therapy.

Condition or disease Intervention/treatment Phase
Primary Sclerosing Cholangitis Drug: hymecromone Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study of Oral Hymecromone to Treat Adults With Primary Sclerosing Cholangitis
Estimated Study Start Date : October 2018
Estimated Primary Completion Date : September 2020
Estimated Study Completion Date : September 2020


Arm Intervention/treatment
Experimental: Hymecromone
Hymecromone 300 mg orally three times per day for 3 months followed by hymecromone 600 mg orally three times per day for an additional 3 months (i.e. total 6 months of drug treatment).
Drug: hymecromone
hymecromone treatment as described
Other Name: 4-Methylumbelliferone




Primary Outcome Measures :
  1. Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 12 months (6 months on treatment and 6 months post treatment) ]

Secondary Outcome Measures :
  1. Number of participants with a qualitative improvement in the anatomy of the biliary tree as assessed by Magnetic Resonance Cholangiopancreatography [ Time Frame: Month 0 (baseline) and Month 6 ]
  2. Hyaluronan serum levels [ Time Frame: Month 0 (baseline) and 3 ]
  3. Hymecromone drug levels [ Time Frame: Month 3 and Month 6 ]
  4. Number of participants with gamma-glutamyl transferase <1.5 times the upper limit of normal [ Time Frame: Month 0 (baseline), 3, 6, 9, 12 ]
  5. Number of participants with Alkaline phosphatase <1.5 times the upper limit of normal [ Time Frame: Month 0 (baseline), 3, 6, 9, 12 ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 18 years or older
  • Diagnosis of primary sclerosing cholangitis confirmed by liver biopsy and/or imaging study (MRI cholangiography or endoscopic retrograde cholangiopancreatography)
  • Gamma-glutamyl transferase (GGT) and Alkaline phosphatase >1.5 times the upper limit of normal
  • If history of endoscopically confirmed inflammatory bowel disease, currently stable based on Harvey-Bradshaw index ≤ 2, normal inflammatory markers (ESR, C-reactive protein, and fecal calprotectin) and stable non-excluded medical therapy for at least 6 months.
  • Not currently taking vancomycin for at least 3 months prior to enrollment. Patients on vancomycin can stop for 3 months and then be screened for enrollment.
  • Not currently taking ursodiol for at least 3 months prior to enrollment. Patients on ursodiol can stop for 3 months and then be screened for enrollment.

Exclusion Criteria:

  • Currently receiving biologic therapies (i.e. infliximab, adalimumab, golimumab, etc.) or steroid medications for inflammatory bowel disease
  • Known allergy to hymecromone
  • Established hepatic or biliary fibrosis
  • Cholangiocarcinoma
  • Pregnancy
  • Physician concern that participant may not adhere to the study protocol, based on prior behavior

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): NCT02780752


Contacts
Contact: Paul Bollyky, MD (650) 723-7823 pbollyky@stanford.edu
Contact: Adam Frymoyer, MD frymoyer@stanford.edu

Locations
United States, California
Stanford University Not yet recruiting
Palo Alto, California, United States, 94304
Contact: Adam Frymoyer, MD       frymoyer@stanford.edu   
Sponsors and Collaborators
Stanford University
Investigators
Principal Investigator: Kenneth Cox, MD Stanford University