CompletedPhase 3

Clinical Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Subjects

United States, Australia, Canada142 participantsStarted 2017-08-14

Plain-language summary

The primary objective is to assess the effect of macitentan 10 mg as compared to placebo on exercise capacity through cardiopulmonary exercise testing.

Who can participate

Age range

12 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Written informed consent/assent from the subject and/or a legal representative prior to initiation of any study-mandated procedures * Fontan-palliated subjects with either intra-atrial lateral tunnel total cavopulmonary connection (LT-TCPC), or extra cardiac tunnel TCPC (EC-TCPC) surgery \> 1 year before Screening. Either LT- or EC-TCPC can be primary or secondary to atrio-pulmonary connection * New York Heart Association (NYHA) functional class (FC) II or III (assessed by the investigator using the Specific Activity Scale * Women of childbearing potential must have a negative serum pregnancy test use reliable contraception Exclusion Criteria: * Pattern of Fontan circulation severity * Deterioration of the Fontan-palliated condition. * Limitations to Cardiopulmonary exercise testing (CPET) * Peak VO2 \< 15 mL/kg/min. * Any known factor or disease that may interfere with treatment compliance or full participation in the study

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change From Baseline in Peak Oxygen Uptake/Consumption (VO2) Up to Week 16

Timeframe: Baseline up to Week 16

Trial details

NCT IDNCT03153137

SponsorActelion

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-06-30

Results submitted2022-06-30

View on ClinicalTrials.gov More Congenital Heart Disease trials

Who can participate

Age range

12 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.