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Completed

NCT Number: NCT03153137

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

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

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Key information

Conditions

Congenital Heart Disease Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Heart Defects, Congenital Heart Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Royal Adelaide Hospital, Adelaide, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Macitentan 10 mg

Drug

film-coated tablet; oral use

Placebo

Drug

film-coated tablet; oral use

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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).

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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.

  9. 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.

  10. 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".

  11. Change From Baseline in Hemoglobin

    Time frame: Baseline, Week 8, Week 16, Week 32 and Week 52

    Change from baseline in hemoglobin was reported.

  12. Change From Baseline in Hematocrit

    Time frame: Baseline, Week 8, Week 16, Week 32 and Week 52

    Change from baseline in hematocrit was reported.

  13. 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.

  14. 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.

  15. 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.

  16. 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.

  17. 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.

  18. 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.

  19. 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.

  20. Change From Baseline in Creatinine

    Time frame: Baseline, Week 8, Week 16, Week 32 and Week 52

    Change from baseline in creatinine was reported.

  21. 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.

  22. Change From Baseline in Urate

    Time frame: Baseline, Week 8, Week 16, Week 32 and Week 52

    Change from baseline in urate was reported.

  23. 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.

  24. 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.

  25. 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.

  26. 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.

Sponsors and collaborators

Lead sponsor

Actelion

Industry

Registry information

Official study title

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

Acronym: RUBATO

Important dates

Study start
2017
Primary completion
2021
Study completion
2021
First posted
May 15, 2017
Registry last updated
Mar 30, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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