An Upcoming Clinical Study to Measure the Safety and Impact of a Drug Called Macitentan in Teenage and Adult Fontan Patients.
Condition: Congenital Heart Disease With Fontan Circulation
MeSH terms: Heart Diseases; Heart Defects, Congenital
Intervention: macitentan 10 mg
Intervention description: macitentan 10 mg, film-coated tablet, oral use
Intervention MeSH terms: Macitentan
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Intervention model description: The study is designed as an open-label (OL), single-arm, multicenter long-term trial in which all adolescent (≥ 12 years) and adult male and female subjects who had previously completed in the parent RUBATO DB study (AC-055H301, NCT03153137) will enroll. The primary objective of the study is to assess the long-term safety and tolerability of macitentan. All efficacy endpoints including the ones listed below are considered as exploratory in nature.
Masking: None (Open Label)
Enrollment: 112.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No
Keywords: Congenital Heart Failure; macitentan; Univentricular Heart; Cavopulmonary Anastomosis; Fontan circulation

Summary:

      The aim of this open-label (OL) trial is to study the long-term use of macitentan for up to 2
      years in Fontan-palliated adult and adolescent patients beyond the 52 weeks of treatment in
      the parent RUBATO double-blind (DB) study (AC-055H301, NCT03153137). This OL trial studies
      the long-term effect of macitentan in Fontan-palliated patients as it is not known if the
      effect of macitentan is sustained beyond 52 weeks (end of the parent RUBATO DB study). In
      addition, the trial also studies the long-term safety of macitentan as this is also unknown.
      Furthermore, the opportunity will be given to patients who were on placebo in the parent
      RUBATO DB study to receive macitentan 10 mg and benefit from a potentially active treatment.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Written informed consent/assent from the subject and/or a legal representative prior
             to initiation of any study-mandated procedures.

          -  Subjects who have completed Week 52 of the parent AC-055H301/RUBATO DB study
             (NCT03153137)

          -  Women of childbearing potential must:

               1. have a negative serum pregnancy test prior to first intake of OL study drug, and,

               2. agree to perform monthly pregnancy tests up to the end of the safety follow up
                  (S-FU) period, and,

               3. use reliable methods of contraception from enrollment up to at least 30 days
                  after study treatment discontinuation.

        Exclusion Criteria:

          -  Clinical worsening leading to medical interventions including reoperation of Fontan
             circulation (Fontan take-down) during the enrollment period

          -  Systolic blood pressure < 90 mmHg (< 85 mmHg for subjects < 18 years old and < 150 cm
             of height) at rest

          -  Criteria related to macitentan use

          -  Any known factor or disease that may interfere with treatment compliance or full
             participation in the study