Investigation of Vancomycin Efficacy in Patients With Ulcerative Colitis and Primary Sclerosing Cholangitis
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Recruitment rate
研究概览
简要总结
This clinical trial tests if oral vancomycin can safely treat active ulcerative colitis (UC) in adults who also have primary sclerosing cholangitis (PSC), a liver condition. The main questions it aims to answer are:
- Can oral vancomycin improve UC symptoms as measured by Mayo score at 4 weeks?
- Is oral vancomycin safe and tolerable in this patient group?
Participants will be compared to see if vancomycin works better than placebo. Participants will:
- Take oral vancomycin (250 mg twice daily) or identical placebo capsules for 4 weeks
- Have the option for 4 more weeks of open-label vancomycin after the blinded phase
- Attend clinic visits at baseline, week 4, and follow-up for Mayo scoring, endoscopy, blood/stool tests, and safety checks
- Track treatment adherence and side effects
The study primarily assesses if the trial can recruit 14 participants, retain them, achieve good adherence, and follow protocol procedures (feasibility). Secondary goals include safety (adverse events) and early signs of benefit in UC activity, liver tests, and gut bacteria balance. This pilot will guide larger future studies.
详细描述
This is an investigator-initiated feasibility study designed to rigorously characterize clinical response signals, safety parameters, and feasibility metrics in adults with co-existing PSC and UC, a population where microbiome-targeted immune modulation is hypothesized to influence disease activity.
Randomization & Allocation Governance
- Randomization will be implemented using validated computer-generated randomization software, with the allocation sequence generated by a study statistician not involved in clinical assessments.
- Allocation concealment will be maintained through centralized pharmacy control. An independent pharmacist will assign treatment codes and release sequentially numbered study medication containers.
- Blinding integrity will be preserved through visual and packaging equivalence, sequential drug accountability logs, and restricted access to treatment codes until database lock.
- Emergency unblinding is permitted only when required for clinical management, and all unblinding events will be documented, timestamped, and reviewed by the DSMC.
Clinical Visit & Assessment Schedule
Participants will complete a structured study visit pathway:
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adults aged ≥18 years.
- •Confirmed diagnosis of UC and PSC.
- •Moderate to severe UC disease activity (total Mayo score ≥5; endoscopic subscore ≥2).
- •Informed consent provided.
排除标准
- •Diagnosis of Crohn's disease or indeterminate colitis.
- •Fulminant colitis or need for immediate surgical intervention.
- •Use of antibiotics or probiotics within the past 4 weeks (for microbiome substudy).
- •Decompensated liver disease (Child-Pugh B/C).
- •Severe Renal Impairment (CrCl <30mL/min)
- •Active untreated infection.
- •Pregnancy or lactation.
- •Prior allergy or intolerance to Vancomycin
- •History of hearing loss or current hearing problems
研究组 & 干预措施
Placebo Comparator
4 weeks blinded placebo followed by optional 4 weeks open-label vancomycin.
干预措施: Placebo (blinded) (Drug)
Placebo Comparator
4 weeks blinded placebo followed by optional 4 weeks open-label vancomycin.
干预措施: Vancomycin (open-label extension) (Drug)
Vancomycin
4 weeks blinded oral vancomycin followed by optional additional 4 weeks open-label vancomycin.
干预措施: Vancomycin (blinded) (Drug)
Vancomycin
4 weeks blinded oral vancomycin followed by optional additional 4 weeks open-label vancomycin.
干预措施: Vancomycin (open-label extension) (Drug)
结局指标
主要结局
Recruitment rate
时间窗: 12 months
Proportion of eligible participants who provide consent and are enrolled in the study (number enrolled ÷ number eligible approached), with a target of 14 participants within 12 months.
Retention rate
时间窗: 12 months
Proportion of enrolled participants who complete all protocol-specified study visits and assessments at 12 months (number completing all follow-up visits ÷ number enrolled).
Treatment adherence
时间窗: 12 months
Proportion of prescribed oral vancomycin doses taken over the treatment period, assessed by pill counts and/or medication diary (number of doses taken ÷ number of doses prescribed).
Protocol compliance with study procedures
时间窗: 12 months
Proportion of participants with all required visits, laboratory tests, and assessments completed per protocol (number fully compliant ÷ number enrolled); may also include rate of major protocol deviations.
次要结局
- Incidence of adverse events and serious adverse events(12 months)
- Severity and type of adverse events(12 months)
- Relationship of adverse events to oral vancomycin(12 months)
- Patient-reported tolerability of oral vancomycin(12 months)