Oral Hymecromone to Treat Adolescents and Adults With Primary Sclerosing Cholangitis. (HAAPS)

A Study of Oral Hymecromone to Treat Adolescents and Adults With Primary Sclerosing Cholangitis(HAAPS Study).

Primary objective: To evaluate the efficacy of hymecromone plus standard of care compared with standard of care alone in the treatment of adolescents and adults with primary sclerosing cholangitis (PSC).

Secondary objectives: To evaluate the change in Alkaline Phosphatase (ALP) from baseline to 6 months post-treatment following treatment with hymecromone plus standard of care compared with standard of care.

To evaluate changes in biomarkers of PSC disease during hymecromone treatment, namely: (a) fibrotic effect (FibroScan); (b) inflammatory biomarkers (serum Hyaluronan (HA)); and, (c) T-cell count.

Study Overview

• Status: Recruiting
• Conditions: Primary Sclerosing Cholangitis
• Intervention / Treatment: Drug: Hymecromone

• Study Type: Interventional
• Enrollment (Estimated): 24
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Redwood City, California, United States, 94063
      • Recruiting
      • Stanford Clinic
      • Contact: Aparna Goel, MD; Phone Number: (650) 498-7878; Email: goela21@stanford.edu
    • Stanford, California, United States, 94305
      • Recruiting
      • Stanford Clinic
      • Contact: Leina Alrabadi, MD; Phone Number: (650) 721-2250; Email: alrabadi@stanford.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 10 years to 71 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Diagnosis of primary sclerosing cholangitis confirmed by liver biopsy and/or imaging study
• If history of endoscopically confirmed inflammatory bowel disease, currently stable based on Mayo Score / Disease Activity Index (DAI) for Ulcerative Colitis Score ≤ 1, normal inflammatory markers (ESR, CRP and fecal calprotectin) and stable non-excluded medical therapy for at least 6 months

Exclusion Criteria:

• Currently receiving biologic therapies
• Known allergy to hymecromone
• Cholangiocarcinoma
• Pregnancy
• Serious liver disease

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Hymecromone; Participants will receive Hymecromone for six months + Standard Of Care (SOC), and will be followed for an additional nine months.	Drug: Hymecromone; Hymecromone 400 mg 3 times daily by mouth.; Other Names: Isochol
No Intervention: Standard Of Care (SOC); Participants will receive Standard Of Care (SOC), and will be followed for 15 months.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change in serum gamma-glutamyltransferase (GGT) levels	Baseline to Month 6

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in serum Alkaline Phosphatase (ALP) levels		Baseline to Month 6
Change in serum hyaluronan levels		Baseline to Month 6
Change in T-cell count		Baseline to Month 6
Change in fibrotic effect based on FibroScan	Fibrotic effect is the amount of liver with fatty change	Baseline to Month 6
Change in biliary tree anatomy (e.g. strictures) based on FibroScan		Baseline to Month 6
Change in serum inflammatory cytokine profile	This outcome measure will assess pro-inflammatory cytokines previously associated with biliary inflammation and other autoimmune diseases including IFNg, IL-6, and TNF.	Baseline to Month 6
Change in lymphocyte immunophenotype	Single cell analysis technique will be used to assess the lymphocytes (B- and T-cells) present in serum samples, including FoxP3+ regulatory T-cells, a tolerogenic lymphocyte subset with important roles in immune tolerance.	Baseline to Month 6
Plasma drug levels of 4-MU		Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Plasma drug levels of 4-MUG		Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Plasma drug levels of 4-MUS		Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits

Collaborators and Investigators

• Sponsor: Aparna Goel
• Investigators: Principal Investigator: Aparna Goel, MD, Stanford University; Principal Investigator: Leina Alrabadi, MD, Stanford University

Study record dates

Study Major Dates

• Study Start (Actual): May 10, 2023
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: March 4, 2022
• First Submitted That Met QC Criteria: March 15, 2022
• First Posted (Actual): March 25, 2022

Study Record Updates

• Last Update Posted (Actual): June 13, 2025
• Last Update Submitted That Met QC Criteria: June 11, 2025
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Biliary Tract Diseases; Bile Duct Diseases; Cholangitis; Cholangitis, Sclerosing
• Other Study ID Numbers: 64030

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No