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

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ClinicalTrials.gov Identifier: NCT03153137
Recruitment Status : Recruiting
First Posted : May 15, 2017
Last Update Posted : April 17, 2019
Sponsor:
Information provided by (Responsible Party):
Actelion

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

Condition or disease Intervention/treatment Phase
Congenital Heart Disease Drug: Macitentan 10 mg Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 134 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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.
Actual Study Start Date : December 7, 2017
Estimated Primary Completion Date : March 28, 2020
Estimated Study Completion Date : March 28, 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Macitentan

Arm Intervention/treatment
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




Primary Outcome Measures :
  1. Change in peak VO2 (oxygen uptake) [ Time Frame: 16 weeks, i.e. from Baseline (Randomization/Visit 2) to Week 16 (Visit 4) ]
    Change in peak VO2 (oxygen uptake)


Secondary Outcome Measures :
  1. Change in peak VO2 [ Time Frame: 52 weeks, i.e. from Baseline (Visit 2) over 52 weeks ]
    Change in peak VO2

  2. Physical activity measured by accelerometer [ Time Frame: 16 weeks, i.e. from Baseline (Visit 2) to Week 16 (Visit 4) ]
    Change in mean count per minute of daily physical activity measured by accelerometer

  3. Treatment-emergent AEs and SAEs [ Time Frame: at least 52 weeks, i.e. up to 30 days after study treatment discontinuation ]
    Treatment-emergent AEs and SAEs

  4. Trough (pre dose) plasma concentrations [ Time Frame: at Week 8 (Visit 3) and Week 16 (Visit 4): 2 days OR at EOT in the case of premature study drug discontinuation: 1 day ]
    Trough (pre dose) plasma concentrations



Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03153137


Contacts
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Contact: Study Contact 844-434-4210 JNJ.CT@sylogent.com

  Show 27 Study Locations
Sponsors and Collaborators
Actelion

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Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT03153137     History of Changes
Other Study ID Numbers: AC-055H301
First Posted: May 15, 2017    Key Record Dates
Last Update Posted: April 17, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Heart Diseases
Cardiovascular Diseases
Macitentan
Endothelin A Receptor Antagonists
Endothelin Receptor Antagonists
Molecular Mechanisms of Pharmacological Action
Endothelin B Receptor Antagonists