Clinical Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Subjects
Purpose
The primary objective is to assess the effect of macitentan 10 mg as compared to placebo on exercise capacity through cardiopulmonary exercise testing.
Condition
- Congenital Heart Disease
Eligibility
- Eligible Ages
- Over 12 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Experimental Macitentan |
Macitentan 10 mg per day; film-coated tablet; oral use |
|
Placebo Comparator Placebo |
film-coated tablet; oral use |
|
More Details
- Status
- Completed
- Sponsor
- Actelion