Clinical study assessing the safety and efficacy of macitentan in Fontan-palliated subjects.

Phase 1

• Conditions: Congenital Heart Failure; MedDRA version: 20.0Level: PTClassification code 10045545Term: Univentricular heartSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 21.1Level: PTClassification code 10065950Term: Cavopulmonary anastomosisSystem Organ Class: 10042613 - Surgical and medical procedures; Therapeutic area: Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Surgical Procedures, Operative [E04]

• Registration Number: EUCTR2016-003320-23-DK
• Lead Sponsor: Actelion Pharmaceuticals Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-05-19
• Last Update Posted: 22 March 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 134

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
- Age range: >= 12 years old
- Women of childbearing potential must have a negative serum pregnancy test at the Randomization visit, perform monthly pregnancy tests, and use reliable contraception
Are the trial subjects under 18? yes
Number of subjects for this age range: 40
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 94
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Pattern of Fontan circulation severity
- Deterioration of the Fontan-palliated condition
- Systolic BP < 90 mmHg (< 85 mmHg for < 18 years old and < 150 cm of height) at rest or during Cardiopulmonary exercise testing at screening or baseline
- Criteria related to macitentan use
- 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

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified