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

Phase 2

Recruiting

• Conditions: Primary Sclerosing Cholangitis
• Interventions: Drug: Hymecromone

• Registration Number: NCT05295680
• Lead Sponsor: Aparna Goel

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.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2022-03-04
• Study First Submitted QC: 2022-03-15
• Study First Posted: 2022-03-25
• Study Start: 2023-05-10
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-24
• Last Update Posted: 2024-11-27
• Primary Completion: 2025-05
• Study Completion: 2025-05

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 24

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

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Exclusion Criteria

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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Change in serum gamma-glutamyltransferase (GGT) levels	Baseline to Month 6

Secondary Outcome Measures

Name	Time	Method
Change in serum Alkaline Phosphatase (ALP) levels	Baseline to Month 6
Change in serum hyaluronan levels	Baseline to Month 6
Change in biliary tree anatomy (e.g. strictures) based on FibroScan	Baseline to Month 6
Change in T-cell count	Baseline to Month 6
Change in serum inflammatory cytokine profile	Baseline to Month 6	This outcome measure will assess pro-inflammatory cytokines previously associated with biliary inflammation and other autoimmune diseases including IFNg, IL-6, and TNF.
Change in fibrotic effect based on FibroScan	Baseline to Month 6	Fibrotic effect is the amount of liver with fatty change
Change in lymphocyte immunophenotype	Baseline to Month 6	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.
Plasma drug levels of 4-MUS	Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
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

Trial Locations

Locations (1)

Stanford Clinic; 🇺🇸 Stanford, California, United States