A Study of Oral Hymecromone to Treat Adolescents and Adults With Primary Sclerosing Cholangitis(HAAPS Study).
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Change in serum gamma-glutamyltransferase (GGT) levels
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 14 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •Currently receiving biologic therapies
- •Known allergy to hymecromone
- •Cholangiocarcinoma
- •Pregnancy
- •Serious liver disease
研究组 & 干预措施
Hymecromone
Participants will receive Hymecromone for six months + Standard Of Care (SOC), and will be followed for an additional nine months.
干预措施: Hymecromone (Drug)
结局指标
主要结局
Change in serum gamma-glutamyltransferase (GGT) levels
时间窗: Baseline to Month 6
次要结局
- Change in serum Alkaline Phosphatase (ALP) levels(Baseline to Month 6)
- Change in serum hyaluronan levels(Baseline to Month 6)
- Change in biliary tree anatomy (e.g. strictures) based on FibroScan(Baseline to Month 6)
- Change in T-cell count(Baseline to Month 6)
- Change in serum inflammatory cytokine profile(Baseline to Month 6)
- Change in fibrotic effect based on FibroScan(Baseline to Month 6)
- Change in lymphocyte immunophenotype(Baseline to Month 6)
- Plasma drug levels of 4-MUS(Single blood draw at baseline, week 2, and months 1, 3, and 6 study visits)
- 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)
研究者
Aparna Goel
Associate Professor of Medicine
Stanford University