An upcoming clinical study to measure the safety and impact of a drug called macitentan in teenage and adult Fontan patients

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: 20.0Level: 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: EUCTR2018-002821-45-FR
• Lead Sponsor: Actelion Pharmaceuticals Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-10-03
• Last Update Posted: 21 March 2022

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.
. Subjects who have completed Week 52 of AC-055H301 RUBATO DB.
. Women of childbearing potential must:
- Have a negative serum pregnancy test prior to first intake of OL study drug, and,
- Agree to perform monthly pregnancy tests up to the end of the safety follow up (S-FU) period, and,
- use reliable methods of contraception from enrollment up to at least 30 days after study treatment discontinuation
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

. 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.

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the long-term safety and tolerability of macitentan in Fontan-palliated adult and adolescent subjects.;Secondary Objective: - To assess the effect of macitentan on exercise capacity (measured by peak VO2).<br>- To assess the effect of macitentan on daily Physical Activity measured by Accelerometer (PA-Ac).;Primary end point(s): Safety endpoints<br>- Treatment-emergent adverse events (AEs), serious AEs and AEs leading to death.<br>- AEs leading to premature discontinuation of study treatment.<br>- Change in vital signs.<br>- Treatment-emergent marked laboratory abnormalities.<br>- Change in laboratory parameters over time;Timepoint(s) of evaluation of this end point: Up to 30 days after end of treatment or treatment discontinuation

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): All efficacy endpoints are considered as exploratory, including endpoints related to the secondary objectives:<br>- Change from baseline in peak VO2.<br>- Change from baseline in mean count per minute of daily physical<br>activity measured by accelerometer (PA-Ac).;Timepoint(s) of evaluation of this end point: -At baseline, Week 52 and Week 104 for peak Vo2.<br>-At baseline, Week 26, Week 52, Week 78 and Week 104 for PA-Ac.