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

Prospective, multi-center, double-blind, randomized, placebo-controlled, parallel-group study assessing the efficacy and safety of macitentan in Fontan-palliated adult and adolescent subjects. - RUBATO

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-003320-23-GB

Enrollment

134

Registered

2017-05-08

Start date

2017-10-11

Completion date

Unknown

Last updated

2020-09-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Congenital Heart Failure MedDRA version: 20.0 Level: PT Classification code 10045545 Term: Univentricular heart System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 21.1 Level: PT Classification code 10065950 Term: Cavopulmonary anastomosis System Organ Class: 10042613 - Surgical and medical procedures

Interventions

Trade Name: Opsumit® Product Name: macitentan Product Code: ACT-064992 Pharmaceutical Form: Film-coated tablet INN or Proposed INN: MACITENTAN CAS Number: 441798-33-0 Current Sponsor code: ACT-064992

Eligibility

Sex/Gender

All

Inclusion criteria

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

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

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the effect of macitentan on exercise capacity (measured by peak oxygen uptake [VO2]) in comparison with placebo in Fontan-palliated subjects.;Secondary Objective: To assess the effect of macitentan on long-term exercise capacity (measured by peak VO2 over 52 weeks). To assess the effect of macitentan on daily Physical Activity measured by Accelometer (PA-Ac). To evaluate the safety and tolerability of macitentan.;Primary end point(s): - Change in peak VO2 (oxygen uptake);Timepoint(s) of evaluation of this end point: From Baseline (Randomization/Visit 2) to Week 16 (Visit 4)	—

Measure

Time frame

Main Objective: To assess the effect of macitentan on exercise capacity (measured by peak oxygen uptake [VO2]) in comparison with placebo in Fontan-palliated subjects.;Secondary Objective: To assess the effect of macitentan on long-term exercise capacity (measured by peak VO2 over 52 weeks). To assess the effect of macitentan on daily Physical Activity measured by Accelometer (PA-Ac). To evaluate the safety and tolerability of macitentan.;Primary end point(s): - Change in peak VO2 (oxygen uptake);Timepoint(s) of evaluation of this end point: From Baseline (Randomization/Visit 2) to Week 16 (Visit 4)

—

Secondary

Measure	Time frame
Secondary end point(s): Efficacy - Change in peak VO2 - Change in mean count per minute of daily PA-Ac Safety - Treatment-emergent AEs and SAEs - AEs leading to premature discontinuation of study treatment Pharmacokinetics (PK) - Trough (pre dose) plasma concentrations;Timepoint(s) of evaluation of this end point: Efficacy - from Baseline (Visit 2) over 52 weeks - from Baseline (Visit 2) to Week 16 (Visit 4) Safety - up to 30 days after study treatment discontinuation. PK - at Week 8 (Visit 3) and Week 16 (Visit 4), or at EOT in the case of premature study drug discontinuation.	—

Measure

Time frame

Secondary end point(s): Efficacy - Change in peak VO2 - Change in mean count per minute of daily PA-Ac Safety - Treatment-emergent AEs and SAEs - AEs leading to premature discontinuation of study treatment Pharmacokinetics (PK) - Trough (pre dose) plasma concentrations;Timepoint(s) of evaluation of this end point: Efficacy - from Baseline (Visit 2) over 52 weeks - from Baseline (Visit 2) to Week 16 (Visit 4) Safety - up to 30 days after study treatment discontinuation. PK - at Week 8 (Visit 3) and Week 16 (Visit 4), or at EOT in the case of premature study drug discontinuation.

—

Countries

Australia, China, Czech Republic, Denmark, France, Germany, Ireland, New Zealand, Poland, Taiwan, United Kingdom, United States

Contacts

Public ContactClinical Registry group

Janssen-Cilag International NV

ClinicalTrialsEU@its.jnj.com+3171 5242166

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026