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

Primary Sclerosing Cholangitis Clinical Trial

— HAAPS  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05295680
• Other study ID #: 64030
• Status: Recruiting
• Phase: Phase 2
• Start date: May 10, 2023
• Est. completion date: May 2025

Study information

• Verified date: October 2023
• Source: Stanford University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 24
• Est. completion date: May 2025
• Est. primary completion date: May 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Cholangitis
• Cholangitis, Sclerosing
• Primary Sclerosing Cholangitis

Intervention

Drug:
• Hymecromone
Hymecromone 400 mg 3 times daily by mouth.

Locations

Country	Name	City	State
United States	Stanford Clinic	Redwood City	California
United States	Stanford Clinic	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Aparna Goel

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in serum gamma-glutamyltransferase (GGT) levels		Baseline to Month 6
Secondary	Change in serum Alkaline Phosphatase (ALP) levels		Baseline to Month 6
Secondary	Change in serum hyaluronan levels		Baseline to Month 6
Secondary	Change in T-cell count		Baseline to Month 6
Secondary	Change in fibrotic effect based on FibroScan	Fibrotic effect is the amount of liver with fatty change	Baseline to Month 6
Secondary	Change in biliary tree anatomy (e.g. strictures) based on FibroScan		Baseline to Month 6
Secondary	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
Secondary	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
Secondary	Plasma drug levels of 4-MU		Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Secondary	Plasma drug levels of 4-MUG		Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Secondary	Plasma drug levels of 4-MUS		Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits