Primary Sclerosing Cholangitis
Conditions
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.
Interventions
DRUGHymecromone
Hymecromone 400 mg 3 times daily by mouth.
Sponsors
Aparna Goel
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
14 Years to 75 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in serum gamma-glutamyltransferase (GGT) levels | Baseline to Month 6 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Fibrotic effect is the amount of liver with fatty change |
| Change in biliary tree anatomy (e.g. strictures) based on FibroScan | Baseline to Month 6 | — |
| Change in serum Alkaline Phosphatase (ALP) levels | Baseline to Month 6 | — |
| 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-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 | — |
| Plasma drug levels of 4-MUS | Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits | — |
| 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. |
Countries
United States
Outcome results
None listed