Fontan CirculationPhase 1/2MYSTICNCT06197763
Impact of Therapy Using Colesevelam Treatment Reducing Bile Acids in Patients With Fontan Circulation.
A Phase 1/2 study testing Colesevelam in people with Fontan Circulation, run by St. Boniface Hospital.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Fontan Circulation
- What is being tested?
- Colesevelam
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- 1 Jun 2024to 1 May 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Of full age of consent (at least ≥ 18 years of age) at screening
- Signed and dated written informed consent in accordance with ICF-GCP and local regulations prior to admission to the study
- Male or female participants. Women of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control that result in a low failure rate of less than 1% per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in the participant informed consent form.
- Diagnosis of Fontan circulation documented in the participant's medical record
- Of full age of consent (at least ≥ 18 years of age) at screening
- Signed and dated written informed consent in accordance with ICF-GCP and local regulations prior to admission to the study
- Male or female participants
Who cannot join
- Has previously received a heart or heart-liver transplant
- Contraindication for using colesevelam, including allergy
- Any physical or mental condition significantly affecting the participant's ability to participate in the investigator's opinion
- Women who are pregnant, nursing, or who plan to become pregnant while in the trial
- Any known medical condition
- Any physical or mental condition significantly affecting the participant's ability to participate in the investigator's opinion
- Women who are pregnant or nursing at time of study visit
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Impact of Therapy Using Colesevelam Treatment Reducing Bile Acids in Patients With Fontan Circulation (MYSTIC) Pilot Study.
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.
Read the 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.
- Conditions
- Fontan Circulation
- Being tested
- Colesevelam, Control Arm
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ColesevelamDrug
625 mg tablets - 3 tables twice per day of Colesevelam or placebo for 6 weeks (crossover), interspersed by 8 week of washout time.
Control ArmOther
No Treatment: baseline assessment only
| Study group | Role | What they receive |
|---|---|---|
| Fontan participants | Experimental | 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. Receives: Drug: Colesevelam |
| Healthy control participants | Sham comparator | 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. Receives: Other: Control Arm |
What is being measured
The primary outcome is the main question the study is designed to answer.
Drug safety
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.
Measured: 20 Weeks
Drug compliance
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.
Measured: 20 weeks
Impact of colesevelam on plasma & stool bile acid concentration.
Plasma \& stool bile acid concentration (total and their subcomponents) will be measured at baseline as well as after medication (placebo \& colesevelam) use.
Measured: 20 Weeks
Secondary outcomes (4)
Impact of colesevelam on blood & urine metabolites.
Plasma and urine metabolites will be measured before and after the drug (colesevelam) and placebo therapy.
Measured: 20 Weeks
Impact of colesevelam on gut microbiome.
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.
Measured: 20 Weeks
Impact of colesevelam on hemodynamics.
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.
Measured: 20 Weeks
Gut microbiome comparison between healthy subjects and Fontan patients.
The gut microbiome will be compared between healthy subjects and patients with Fontan circulation at baseline.
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2024
- Study start1 Jun 2024 (estimated)
- Primary completion1 Nov 2025 (estimated)
- Study completion1 May 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fontan CirculationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- 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. A further 25 age- and sex- matched healthy control participants will be recruited from advertising at St. Boniface Hospital and University of Manitoba. Healthy controls will only undergo the baseline visit and will not receive the investigational drug or placebo.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- St. Boniface Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dr. Ashish Shah, St. Boniface Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06197763
- Sponsor's protocol ID
- CTA/2023/001
- Acronym
- MYSTIC
- Status last verified
- Apr 2024
- First submitted
- 4 Oct 2023
- Last updated
- 17 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Shah AH, Armstrong HK, Mittal I, Reimer A, Kunutsor SK, Ducas RA, Alizadeh K, Tam JW, Ravandi A, Dhingra S. IMpact of therapY using coleSevelam treatment reducing bile acids in patients with fonTan cIrCulation (MYSTIC): Rationale and study design. Am Heart J. 2026 Jan;291:81-88. doi: 10.1016/j.ahj.2025.08.011. Epub 2025 Aug 19.
PubMed 40840821
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Impact of Therapy Using Colesevelam Treatment Reducing Bile Acids in Patients With Fontan Circulation (MYSTIC) Pilot Study.
- ClinicalTrials.gov ID
- NCT06197763
- Acronym / sponsor ID
- MYSTIC / CTA/2023/001
- Conditions
- Fontan Circulation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- St. Boniface HospitalUniversity, hospital or other
- Study start
- 1 Jun 2024 (estimated)
- Primary completion
- 1 Nov 2025 (estimated)
- Study completion
- 1 May 2026 (estimated)
- First posted
- 9 Jan 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- 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. A further 25 age- and sex- matched healthy control participants will be recruited from advertising at St. Boniface Hospital and University of Manitoba. Healthy controls will only undergo the baseline visit and will not receive the investigational drug or placebo.
| Arm | Type | Receives |
|---|---|---|
| Fontan participants | Experimental | Drug: ColesevelamParticipants 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. |
| Healthy control participants | Sham comparator | Other: Control 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Of full age of consent (at least ≥ 18 years of age) at screening
- Signed and dated written informed consent in accordance with ICF-GCP and local regulations prior to admission to the study
- Male or female participants. Women of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control that result in a low failure rate of less than 1% per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in the participant informed consent form.
- Diagnosis of Fontan circulation documented in the participant's medical record
- Of full age of consent (at least ≥ 18 years of age) at screening
Key exclusion criteria (7 listed)
- Has previously received a heart or heart-liver transplant
- Contraindication for using colesevelam, including allergy
- Any physical or mental condition significantly affecting the participant's ability to participate in the investigator's opinion
- Women who are pregnant, nursing, or who plan to become pregnant while in the trial
- Any known medical condition
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Colesevelam625 mg tablets - 3 tables twice per day of Colesevelam or placebo for 6 weeks (crossover), interspersed by 8 week of washout time. | Drug | Fontan participants |
| Control ArmNo Treatment: baseline assessment only | Other | Healthy control participants |
Primary outcome
Drug safety
Time frame: 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.
Drug compliance
Time frame: 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.
Impact of colesevelam on plasma & stool bile acid concentration.
Time frame: 20 Weeks
Plasma \& stool bile acid concentration (total and their subcomponents) will be measured at baseline as well as after medication (placebo \& colesevelam) use.
4 secondary outcomes
Impact of colesevelam on blood & urine metabolites.
Time frame: 20 Weeks
Impact of colesevelam on gut microbiome.
Time frame: 20 Weeks
Impact of colesevelam on hemodynamics.
Time frame: 20 Weeks
Gut microbiome comparison between healthy subjects and Fontan patients.
Time frame: Baseline
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Apr 2024.
- Derived from this study: PubMed 40840821Shah AH, Armstrong HK, Mittal I, Reimer A, Kunutsor SK, Ducas RA, Alizadeh K, Tam JW, Ravandi A, Dhingra S. IMpact of therapY using coleSevelam treatment reducing bile acids in patients with fonTan cIrCulation (MYSTIC): Rationale and study design. Am Heart J. 2026 Jan;291:81-88. doi: 10.1016/j.ahj.2025.08.011. Epub 2025 Aug 19.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Colesevelam in people with Fontan Circulation, run by St. Boniface Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 17 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ashish Shah, M.D. (204-237-2023, ashah5@sbgh.mb.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Drug safety (time frame: 20 Weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06197763, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Apr 2024.