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Impact of Therapy Using Colesevelam Treatment Reducing Bile Acids in Patients With Fontan Circulation.

Phase 1
Not yet recruiting
Conditions
Fontan Circulation
Interventions
Other: Control Arm
Registration Number
NCT06197763
Lead Sponsor
St. Boniface Hospital
Brief Summary

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.

Detailed Description

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.

Recruitment & Eligibility

Status
NOT_YET_RECRUITING
Sex
All
Target Recruitment
50
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
CROSSOVER
Arm && Interventions
GroupInterventionDescription
Healthy control participantsControl ArmThese 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.
Fontan participantsColesevelamParticipants 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.
Primary Outcome Measures
NameTimeMethod
Drug safety20 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.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 compliance20 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.

Secondary Outcome Measures
NameTimeMethod
Impact of colesevelam on gut microbiome.20 Weeks

Stool samples before and after each medication (placebo and colesevelam) use will be analyzed to determine gut microbiome composition at baseline, and the impact of the colesevelam therapy on this composition.

Impact of colesevelam on hemodynamics.20 Weeks

Each study participant will be evaluated by total body-impedance-based, non-invasive cardiac system (NICaS) derived hemodynamic parameters (cardiac output, cardiac index, stroke volume, stroke index, cardiac power output), before and after medication (placebo \& colesevelam) use.

Gut microbiome comparison between healthy subjects and Fontan patients.Baseline

The gut microbiome will be compared between healthy subjects and patients with Fontan circulation at baseline.

Impact of colesevelam on blood & urine metabolites.20 Weeks

Plasma and urine metabolites will be measured before and after the drug (colesevelam) and placebo therapy.

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