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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
24
Hymecromone 400 mg 3 times daily by mouth.
Stanford Clinic
Redwood City, California, United States
RECRUITINGStanford Clinic
Stanford, California, United States
RECRUITINGChange in serum gamma-glutamyltransferase (GGT) levels
Time frame: Baseline to Month 6
Change in serum Alkaline Phosphatase (ALP) levels
Time frame: Baseline to Month 6
Change in serum hyaluronan levels
Time frame: Baseline to Month 6
Change in T-cell count
Time frame: Baseline to Month 6
Change in fibrotic effect based on FibroScan
Fibrotic effect is the amount of liver with fatty change
Time frame: Baseline to Month 6
Change in biliary tree anatomy (e.g. strictures) based on FibroScan
Time frame: 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.
Time frame: 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.
Time frame: Baseline to Month 6
Plasma drug levels of 4-MU
Time frame: Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Plasma drug levels of 4-MUG
Time frame: Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Plasma drug levels of 4-MUS
Time frame: Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
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