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

NCT ID: NCT05295680

Last Updated: 2025-06-13

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-05-10
• Study Completion Date: 2027-12-31

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Hymecromone

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

Group Type: EXPERIMENTAL

Hymecromone

Intervention Type: DRUG

Hymecromone 400 mg 3 times daily by mouth.

Standard Of Care (SOC)

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

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Hymecromone

Hymecromone 400 mg 3 times daily by mouth.

Intervention Type: DRUG

Other Intervention Names

Isochol

Eligibility Criteria

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

• Minimum Eligible Age: 14 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Aparna Goel

OTHER

Sponsor Role: lead

Responsible Party

Aparna Goel

Associate Professor of Medicine

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Aparna Goel, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Leina Alrabadi, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Stanford Clinic

Redwood City, California, United States

Site Status: RECRUITING

Stanford Clinic

Stanford, California, United States

Site Status: RECRUITING

Countries

United States

Facility Contacts

Aparna Goel, MD

Role: primary

goela21@stanford.edu

(650) 498-7878

Leina Alrabadi, MD

Role: primary

alrabadi@stanford.edu

(650) 721-2250

References

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.

Reference Type: DERIVED

PMID: 37055910 (View on PubMed)

Other Identifiers

64030

Identifier Type: -

Identifier Source: org_study_id