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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03153137
Recruitment Status : Recruiting
First Posted : May 15, 2017
Last Update Posted : March 18, 2020
Sponsor:
Information provided by (Responsible Party):
Actelion

Tracking Information
First Submitted Date  ICMJE May 12, 2017
First Posted Date  ICMJE May 15, 2017
Last Update Posted Date March 18, 2020
Actual Study Start Date  ICMJE August 14, 2017
Estimated Primary Completion Date June 11, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 12, 2017)
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)
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 12, 2017)
  • Change in peak VO2 [ Time Frame: 52 weeks, i.e. from Baseline (Visit 2) over 52 weeks ]
    Change in peak VO2
  • 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
  • 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
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Clinical Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Subjects
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Congenital Heart Disease
Intervention  ICMJE
  • Drug: Macitentan 10 mg
    film-coated tablet; oral use
  • Drug: Placebo
    film-coated tablet; oral use
Study Arms  ICMJE
  • Experimental: Macitentan
    Macitentan 10 mg per day; film-coated tablet; oral use
    Intervention: Drug: Macitentan 10 mg
  • Placebo Comparator: Placebo
    film-coated tablet; oral use
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 12, 2017)
134
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 31, 2021
Estimated Primary Completion Date June 11, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Study Contact 844-434-4210 JNJ.CT@sylogent.com
Listed Location Countries  ICMJE Australia,   Brazil,   Canada,   China,   Czechia,   Denmark,   France,   Germany,   Ireland,   New Zealand,   Poland,   Taiwan,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03153137
Other Study ID Numbers  ICMJE AC-055H301
2016-003320-23 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Actelion
Study Sponsor  ICMJE Actelion
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Actelion
Verification Date March 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP