Clinical trial NCT05295680: Oral Hymecromone to Treat Adolescents and Adults With Primary Sclerosing Cholangitis.
Official title: A Study of Oral Hymecromone to Treat Adolescents and Adults With Primary Sclerosing Cholangitis(HAAPS Study).
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Primary Sclerosing Cholangitis
Lead sponsor: Stanford University
Sponsor class: OTHER
First posted: 2022-03-25
Enrollment: 24
Sex: ALL
Minimum age (years): 14
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Change in serum gamma-glutamyltransferase (GGT) levels
timeFrame: Baseline to Month 6
Secondary outcomes:
measure: Change in serum Alkaline Phosphatase (ALP) levels
timeFrame: Baseline to Month 6
measure: Change in serum hyaluronan levels
timeFrame: Baseline to Month 6
measure: Change in T-cell count
timeFrame: Baseline to Month 6
measure: Change in fibrotic effect based on FibroScan
timeFrame: Baseline to Month 6
measure: Change in biliary tree anatomy (e.g. strictures) based on FibroScan
timeFrame: Baseline to Month 6
measure: Change in serum inflammatory cytokine profile
timeFrame: Baseline to Month 6
measure: Change in lymphocyte immunophenotype
timeFrame: Baseline to Month 6
measure: Plasma drug levels of 4-MU
timeFrame: Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
measure: Plasma drug levels of 4-MUG
timeFrame: Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
measure: Plasma drug levels of 4-MUS
timeFrame: Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits
Other outcomes:
Locations:
city: Redwood City
country: United States
facility: Stanford Clinic
state: California
city: Stanford
country: United States
facility: Stanford Clinic
state: California
Study officials:
affiliation: Stanford University
name: Aparna Goel, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Stanford University
name: Leina Alrabadi, MD
role: PRINCIPAL_INVESTIGATOR
References: