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

NCT05295680 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 64030
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Primary Sclerosing Cholangitis

Outcome Measures

Primary Outcomes (1)

• Change in serum gamma-glutamyltransferase (GGT) levels

  Baseline to Month 6

Secondary Outcomes (10)

• 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

  Baseline to Month 6

• Change in biliary tree anatomy (e.g. strictures) based on FibroScan

  Baseline to Month 6

Study Arms (2)

Hymecromone

EXPERIMENTAL

Participants will receive Hymecromone for six months + Standard Of Care (SOC), and will be followed for an additional nine months.

Drug: Hymecromone

Standard Of Care (SOC)

NO INTERVENTION

Participants will receive Standard Of Care (SOC), and will be followed for 15 months.

Interventions

Hymecromone DRUG

Hymecromone 400 mg 3 times daily by mouth.

Also known as: Isochol

Hymecromone

Eligibility Criteria

• Age: 14 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Aparna Goel: lead

Study Sites (2)

Stanford Clinic

Redwood City, California, 94063, United States

RECRUITING

Stanford Clinic

Stanford, California, 94305, United States

RECRUITING

Related Publications (1)

• Salman L, Martinez L, Faddoul G, Manning C, Ali K, Salman M, Vazquez-Padron R. Hyaluronan Inhibition as a Therapeutic Target for Diabetic Kidney Disease: What Is Next? Kidney360. 2023 Jun 1;4(6):e851-e860. doi: 10.34067/KID.0000000000000126. Epub 2023 Apr 14.

  PMID: 37055910 DERIVED

MeSH Terms

Conditions

Cholangitis, Sclerosing

Interventions

Hymecromone

Condition Hierarchy (Ancestors)

Cholangitis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases

Intervention Hierarchy (Ancestors)

Umbelliferones; Coumarins; Benzopyrans; Pyrans; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Aparna Goel, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

• Leina Alrabadi, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Associate Professor of Medicine

Study Record Dates

• First Submitted: March 4, 2022
• First Posted: March 25, 2022
• Study Start: May 10, 2023
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: June 13, 2025

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)