Clinical Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Subjects (RUBATO)

March 28, 2025 updated by: Actelion

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

The primary objective is to assess the effect of macitentan 10 mg as compared to placebo on exercise capacity through cardiopulmonary exercise testing.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
142

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Adelaide, Australia, 5000
        • Royal Adelaide Hospital
      • Camperdown, Australia, 2050
        • Royal Prince Alfred Hospital
      • Chermside, Australia, 4032
        • The Prince Charles Hospital, Adult Congenital Heart Disease Unit
      • Parkville, Australia, 3052
        • Royal Children's Hospital
      • South Brisbane, Australia, 4101
        • Queensland Children's Hospital
      • Westmead, Australia, 2145
        • Westmead Hospital
  • Canada
      • Quebec, Canada, G1V 4G2
        • CHU de Québec Université Laval
  • China
      • Beijing, China, 100029
        • Beijing Anzhen Hospital
      • Beijing, China, 100037
        • Beijing Fuwai hospital
      • Shanghai, China, 200127
        • Shanghai Childrens Medical Center
      • Wuhan, China, 430022
        • Wuhan Asia Heart Hospital
  • Czechia
      • Praha 5, Czechia, 150 06
        • Fakultni nemocnice v Motole
  • Denmark
      • Copenhagen, Denmark, 2100
        • Rigshospitalet Kardiologisk Klinisk
  • France
      • Montpellier Cedex 5, France, 34295
        • CHU Arnaud de Villeneuve
      • Paris, France, 75015
        • Hôpital Necker - Enfants Malades
      • Pessac, France, 33604
        • Hôpital Cardiologique Du Haut-Lévêque
  • Germany
      • Berlin, Germany, 13353
        • Deutsches Herzzentrum Berlinklinik Für Angeborene Herzfehler
      • München, Germany, 80636
        • Deutsches Herzzentrum München
  • New Zealand
      • Auckland, New Zealand, 1640
        • Auckland City Hospital
  • Poland
      • Gdansk, Poland, 80 952
        • Uniwersyteckie Centrum Kliniczne
      • Krakow, Poland, 30-663
        • Uniwersytecki Szpital Dzieciecy w Krakowie
      • Krakow, Poland, 31 202
        • Krakowski Szpital Specjalityczny im Jana Pawla II Oddzial Kliniczny Chorob Serca i Naczyn
      • Wroclaw, Poland, 51 124
        • Wojewodzki Szpital Specjalistyczny we Wroclawiu
  • Taiwan
      • Taipei, Taiwan, 100
        • National Taiwan University Hospital
  • United Kingdom
      • Birmingham, United Kingdom, B15 2TH
        • Queen Elizabeth Hospital
      • Birmingham, United Kingdom, B4 6NH
        • Birmingham Children's Hospital
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233-1935
        • University of Alabama at Birmingham
    • California
      • Los Angeles, California, United States, 90095
        • UCLA
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital Heart Center
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Texas
      • Houston, Texas, United States, 77030-2303
        • Texas Children's Hospital
    • Washington
      • Spokane, Washington, United States, 99202
        • Providence Medical Research Providence Health Care

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Macitentan
Macitentan 10 mg per day; film-coated tablet; oral use
Drug: Macitentan 10 mg
film-coated tablet; oral use
Placebo Comparator: Placebo
film-coated tablet; oral use
Drug: Placebo
film-coated tablet; oral use

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Peak Oxygen Uptake/Consumption (VO2) Up to Week 16
Time Frame: Baseline up to Week 16
Change from baseline in peak VO2 up to Week 16 was reported.
Baseline up to Week 16

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Peak VO2 Up to Week 52
Time Frame: Baseline up to Week 52
Change from baseline in peak VO2 up to Week 52 was reported.
Baseline up to Week 52
Change From Baseline in Mean Count Per Minute of Daily Physical Activity Measured by Accelerometer (PA-Ac) Up to Week 16
Time Frame: Baseline up to Week 16
Change from baseline in mean count per minute of daily PA-Ac up to Week 16 was reported. The daily physical activity (counts per min) of the participant was assessed via accelerometer during daytime. The accelerometer was given to the participant at Visit 1, and data was collected for 9 consecutive daily daytime periods after Visit 1 (baseline) to Visit 4 (Week 16).
Baseline up to Week 16
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs)
Time Frame: Up to 56 weeks
SAE is any AE that results in: death, persistent or significant disability/incapacity, requires inpatient hospitalization or prolongation of existing hospitalization, is life-threatening experience, is a congenital anomaly/birth defect and may jeopardize participant and/or may require medical or surgical intervention to prevent one of the outcomes listed above.
Up to 56 weeks
Number of Participants With Treatment-emergent Adverse Events (AEs)
Time Frame: Up to 56 weeks
An adverse event is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product.
Up to 56 weeks
Number of Participants With AEs Leading to Premature Discontinuation of Study Treatment
Time Frame: Up to 56 weeks
Number of participants with AEs leading to premature discontinuation of study treatment was reported. AEs leading to premature discontinuation of study treatment were those with action taken with study drug reported as 'permanently discontinued' by the investigator.
Up to 56 weeks
Change From Baseline in Systolic and Diastolic Arterial Blood Pressure (BP)
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in systolic and diastolic arterial BP at Week 8, Week 16, Week 32 and Week 52 was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Pulse Rate
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in pulse rate at Week 8, Week 16, Week 32 and Week 52 was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Oxygen Saturation (SpO2)
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in SpO2 was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Body Weight
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in body weight was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Number of Participants With Treatment-emergent Markedly Abnormal Laboratory Values
Time Frame: Up to 56 weeks
Number of participants with treatment-emergent markedly laboratory abnormal laboratory values were reported. Abnormal values for platelets (LL < 75); Lymphocytes (HH > 4.0); Neutrophils (LL < 1.5); Prothrombin International Normalized Ratio: HH (greater than and equal to [>=] 1.5 upper limit of normal [ULN]), Ratio: HH >= 2.5 ULN); Bilirubin (HH >= 2 ULN); Alkaline Phosphatase (HH > 2.5 ULN); Glomerular Filtration Rate (LL < 60); Glucose (HH > 8.9); Triglycerides (HH > 3.42). Here "HH" refers to values above the normal range, where H stands for "high" and "LL" refers to values below the normal range where L stands for "low".
Up to 56 weeks
Change From Baseline in Hemoglobin
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in hemoglobin was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Hematocrit
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in hematocrit was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Erythrocytes and Reticulocytes
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in erythrocytes and reticulocytes at Week 8, Week 16, Week 32 and Week 52 was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Leucocytes, Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils and Platelets
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in leucocytes, neutrophils, lymphocytes, monocytes, eosinophils, basophils and platelets at Week 8, Week 16, Week 32 and Week 52 was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Prothrombin Time
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in prothrombin time was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Prothrombin International Normalized Ratio
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in prothrombin international normalized ratio was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST) and Alkaline Phosphatase (AP)
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in ALT, AST and AP were reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Bilirubin and Direct Bilirubin
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in bilirubin and direct bilirubin was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Gamma Glutamyl Transferase
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in gamma glutamyl transferase was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Creatinine
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in creatinine was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Urea Nitrogen
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in urea nitrogen was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Urate
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in urate was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Glucose, Cholesterol, Triglycerides, Sodium, Potassium, Chloride and Calcium
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in glucose, cholesterol, triglycerides, sodium, potassium, chloride and calcium was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Albumin and Protein
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in albumin and protein was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Alpha Fetoprotein
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in alpha fetoprotein was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52
Change From Baseline in Cystatin C
Time Frame: Baseline, Week 8, Week 16, Week 32 and Week 52
Change from baseline in cystatin C was reported.
Baseline, Week 8, Week 16, Week 32 and Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Actelion

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 14, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 30, 2021

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 26, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 12, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 12, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 15, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 30, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 28, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AC-055H301
  • 2016-003320-23 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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Locations

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