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An Upcoming Clinical Study to Measure the Safety and Impact of a Drug Called Macitentan in Teenage and Adult Fontan Patients. (RUBATO OL)

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: NCT03775421
Recruitment Status : Recruiting
First Posted : December 14, 2018
Last Update Posted : October 28, 2020
Sponsor:
Collaborators:
Covance
Henry Ford Health System
Almac Clinical Technologies
ActiGraph LLC
Medidata Solutions
Information provided by (Responsible Party):
Actelion

Tracking Information
First Submitted Date  ICMJE November 21, 2018
First Posted Date  ICMJE December 14, 2018
Last Update Posted Date October 28, 2020
Actual Study Start Date  ICMJE April 11, 2019
Estimated Primary Completion Date November 7, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 11, 2018)
  • Frequency of treatment-emergent adverse events (AEs), serious AEs and AEs leading to death [ Time Frame: From Enrollment to End-of-Study visit (Week 104 + 30 days safety follow-up) ]
    Treatment-emergent AEs and SAEs are captured up to 30 days after OL treatment discontinuation (End-of-Treatment visit). A treatment-emergent AE is any AE temporally associated with the use of study treatment (from OL treatment initiation until 30 days after OL treatment discontinuation) whether or not considered by the investigator as related to study treatment.
  • Number of AEs leading to premature discontinuation of study treatment [ Time Frame: From Enrollment to End-of-Treatment visit (Week 104) ]
    Number of subjects with an AE assessed by the investigator as leading to discontinuation of study treatment.
  • Frequency of treatment-emergent marked laboratory abnormalities up to 30 days after study treatment discontinuation [ Time Frame: From Enrollment to End-of-Study visit (Week 104 + 30 days safety follow-up) ]
    Laboratory abnormalities are laboratory values below of above the normal range. The definitions of marked abnormal values are based mainly on the Common Terminology Criteria for Adverse Events (CTCAE).
  • Incident rate of changes in laboratory parameters over time [ Time Frame: From Enrollment to End-of-Study visit (Week 104 + 30 days safety follow-up) ]
    Laboratory parameters (hematology, clinical chemistry) are assessed at every visit from baseline (enrollment) to End-of-Study visit.
  • Change in pulse rate (PR) over time [ Time Frame: From Enrollment to End-of-Treatment visit (Week 104) ]
    PR will be assessed at all scheduled visits from baseline (enrollment) to End-of-Treatment visit.
  • Change in peripheral oxygen saturation (SpO2) over time [ Time Frame: From Enrollment to End-of-Treatment visit (Week 104) ]
    SpO2 will be assessed at all scheduled visits from baseline (enrollment) to End-of-Treatment visit.
  • Change in blood pressure (BP) over time [ Time Frame: From Enrollment to End-of-Treatment visit (Week 104) ]
    Systolic and diastolic blood pressure will be assessed at all scheduled visits from baseline (enrollment) to End-of-Treatment visit.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures
 (submitted: December 11, 2018)
  • Change from baseline in peak oxygen uptake (VO2) [ Time Frame: From Enrollment to End-of-Treatment visit (Week 104) ]
    Peak VO2 is measured from baseline to each scheduled time point to assess the effect of macitentan on exercise capacity.
  • Change from baseline in mean count per minute of daily physical activity measured by accelerometer (PA-Ac) [ Time Frame: From Enrollment to End-of-Treatment visit (Week 104) ]
    The daily physical activity (counts/min) of the subject is assessed via accelerometer during daytime. The mean count per minute of daily PA-Ac will be measured from baseline to each scheduled time point.
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE An Upcoming Clinical Study to Measure the Safety and Impact of a Drug Called Macitentan in Teenage and Adult Fontan Patients.
Official Title  ICMJE Prospective, Multi-center, Single-arm, Open-label Long-term Study Assessing the Safety, Tolerability, and Effectiveness of Macitentan in Fontan-palliated Adult and Adolescent Subjects
Brief Summary The aim of this open-label (OL) trial is to study the long-term use of macitentan for up to 2 years in Fontan-palliated adult and adolescent patients beyond the 52 weeks of treatment in the parent RUBATO double-blind (DB) study (AC-055H301, NCT03153137). This OL trial studies the long-term effect of macitentan in Fontan-palliated patients as it is not known if the effect of macitentan is sustained beyond 52 weeks (end of the parent RUBATO DB study). In addition, the trial also studies the long-term safety of macitentan as this is also unknown. Furthermore, the opportunity will be given to patients who were on placebo in the parent RUBATO DB study to receive macitentan 10 mg and benefit from a potentially active treatment.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
The study is designed as an open-label (OL), single-arm, multicenter long-term trial in which all adolescent (≥ 12 years) and adult male and female subjects who had previously completed in the parent RUBATO DB study (AC-055H301, NCT03153137) will enroll. The primary objective of the study is to assess the long-term safety and tolerability of macitentan. All efficacy endpoints including the ones listed below are considered as exploratory in nature.
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Congenital Heart Disease With Fontan Circulation
Intervention  ICMJE Drug: macitentan 10 mg
macitentan 10 mg, film-coated tablet, oral use
Study Arms  ICMJE Experimental: Open-label treatment period
oral administration of 10 mg macitentan once daily
Intervention: Drug: macitentan 10 mg
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: December 11, 2018)
134
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 31, 2024
Estimated Primary Completion Date November 7, 2022   (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.
  • Subjects who have completed Week 52 of the parent AC-055H301/RUBATO DB study (NCT03153137)
  • Women of childbearing potential must:

    1. have a negative serum pregnancy test prior to first intake of OL study drug, and,
    2. agree to perform monthly pregnancy tests up to the end of the safety follow up (S-FU) period, and,
    3. use reliable methods of contraception from enrollment up to at least 30 days after study treatment discontinuation.

Exclusion Criteria:

  • Clinical worsening leading to medical interventions including reoperation of Fontan circulation (Fontan take-down) during the enrollment period
  • Systolic blood pressure < 90 mmHg (< 85 mmHg for subjects < 18 years old and < 150 cm of height) at rest
  • Criteria related to macitentan use
  • 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,   Canada,   China,   Czechia,   Denmark,   France,   New Zealand,   Poland,   Taiwan,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03775421
Other Study ID Numbers  ICMJE AC-055H302
2018-002821-45 ( 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: Yes
Plan Description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials\transparency. As noted on this site, requests for access to the study data can be submitted through Yale open Access (YODA) Project site at yoda.yale.edu
URL: https://www.janssen.com/clinical-trials/transparency
Responsible Party Actelion
Study Sponsor  ICMJE Actelion
Collaborators  ICMJE
  • Covance
  • Henry Ford Health System
  • Almac Clinical Technologies
  • ActiGraph LLC
  • Medidata Solutions
Investigators  ICMJE
Study Director: Thierry Francis Briand, MD Actelion
PRS Account Actelion
Verification Date October 2020

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