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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Hymecromone | Experimental | Participants will receive Hymecromone for six months + Standard Of Care (SOC), and will be followed for an additional nine months. |
|
| Standard Of Care (SOC) | No Intervention | Participants will receive Standard Of Care (SOC), and will be followed for 15 months. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hymecromone | Drug | Hymecromone 400 mg 3 times daily by mouth. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in serum gamma-glutamyltransferase (GGT) levels | Baseline to Month 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in serum Alkaline Phosphatase (ALP) levels | Baseline to Month 6 | |
| Change in serum hyaluronan levels | Baseline to Month 6 | |
| Change in T-cell count |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Aparna Goel, MD | Stanford University | Principal Investigator |
| Leina Alrabadi, MD | Stanford University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford Clinic | Recruiting | Redwood City | California | 94063 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37055910 | Derived | 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. |
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| ID | Term |
|---|---|
| D015209 | Cholangitis, Sclerosing |
| ID | Term |
|---|---|
| D002761 | Cholangitis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
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| ID | Term |
|---|---|
| D006923 | Hymecromone |
| ID | Term |
|---|---|
| D014468 | Umbelliferones |
| D003374 | Coumarins |
| D001578 | Benzopyrans |
| D011714 | Pyrans |
| D006573 |
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| Baseline to Month 6 |
| Change in fibrotic effect based on FibroScan | Fibrotic effect is the amount of liver with fatty change | Baseline to Month 6 |
| Change in biliary tree anatomy (e.g. strictures) based on FibroScan | 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. | 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. | Baseline to Month 6 |
| 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 |
| Stanford Clinic | Recruiting | Stanford | California | 94305 | United States |
|
| Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |