Impact of Therapy Using Colesevelam Treatment Reducing Bile Acids in Patients With Fontan Circulation (MYSTIC) Pilot Study.
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 50
- 主要终点
- Drug safety
研究概览
简要总结
This is the first pilot study proposing a novel therapeutic option treating patients with Fontan circulation (FC), a high-risk condition that has no definite treatment options available, other than heart or heart-liver transplantation. The investigator's identification of elevated BA and their association with adverse clinical - investigational features in Fontan patients are novel.
详细描述
The investigators propose a prospective, double-blind, placebo cross-over, pilot study evaluating colesevelam (intestinal BA scavenger) therapy and placebo in 25 stable adult FC participants. These participants will be recruited from the cardiology clinics at the St. Boniface Hospital, specifically the Manitoba Adult Congenital Heart (MACH) Clinic. Participants will be randomized to be treated with either colesevelam (625 mg tablets - 3 tables twice per day) or placebo (3 tablets twice per day) for 6 weeks, interspersed by 8 week of washout time. Local research and ethics board approval will be obtained. Informed consent will be obtained from all participants.
A further 25 age- and sex- matched healthy control participants will be recruited from advertising at St. Boniface Hospital and University of Manitoba. These healthy control participants will be comparators to the randomized Fontan participants at baseline. Recruitment of this group will be completed after all Fontan participants have been screened, consented, and randomized to ensure accuracy of age and sex matching. Age and sex matched participants will be +/- 3 years and not pregnant or nursing at time of study visit.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
盲法说明
Participants, investigators, and everyone involved in study conduct or analysis or with any other interest in this double-blind study will remain blinded with regard to the randomization treatment assignments until after data collection completion. The access to the randomization code will be kept restricted until its release for analysis.
The DSMB will be provided with unblinded data in order to allow them to review efficacy and safety and to fulfill their tasks as outlined in the DSMB charter.
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Fontan participants
Participants will be randomized to be treated with either colesevelam (625 mg tablets - 3 tables twice per day) or placebo (3 tablets twice per day) for 6 weeks, interspersed by 8 week of washout time.
干预措施: Colesevelam (Drug)
Healthy control participants
These healthy control participants will be comparators to the randomized Fontan participants. Healthy controls will only undergo the baseline visit and will not receive the investigational drug or placebo.
干预措施: Control Arm (Other)
结局指标
主要结局
Drug safety
时间窗: 20 Weeks
This outcome will be evaluated by identifying side effects experienced by any of the study participants throughout the study period. This information will be collected during biweekly phone calls to each of the study participants by the study coordinator. The study participants will also have the contact number of the study coordinator to notify them of any issues. The investigators have also established a "Data and drug safety monitoring board" which will review each of the side effects experienced by the study participants.
Impact of colesevelam on plasma & stool bile acid concentration.
时间窗: 20 Weeks
Plasma \& stool bile acid concentration (total and their subcomponents) will be measured at baseline as well as after medication (placebo \& colesevelam) use.
Drug compliance
时间窗: 20 weeks
This will be evaluated by checking with each study participant about their drug compliance and identifying the number of days, they have taken tablets as recommended.
次要结局
- Impact of colesevelam on gut microbiome.(20 Weeks)
- Impact of colesevelam on hemodynamics.(20 Weeks)
- Gut microbiome comparison between healthy subjects and Fontan patients.(Baseline)
- Impact of colesevelam on blood & urine metabolites.(20 Weeks)
研究者
Dr. Ashish Shah
Qualified Investigator
St. Boniface Hospital