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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 : August 5, 2020
Sponsor:
Collaborators:
Covance
Henry Ford Health System
Almac Clinical Technologies
ActiGraph LLC
Medidata Solutions
Information provided by (Responsible Party):
Actelion

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.

Condition or disease Intervention/treatment Phase
Congenital Heart Disease With Fontan Circulation Drug: macitentan 10 mg Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 134 participants
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
Official Title: 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
Actual Study Start Date : April 11, 2019
Estimated Primary Completion Date : November 7, 2022
Estimated Study Completion Date : March 31, 2024

Resource links provided by the National Library of Medicine

Drug Information available for: Macitentan

Arm Intervention/treatment
Experimental: Open-label treatment period
oral administration of 10 mg macitentan once daily
Drug: macitentan 10 mg
macitentan 10 mg, film-coated tablet, oral use




Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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).

  4. 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.

  5. 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.

  6. 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.

  7. 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.


Other Outcome Measures:
  1. 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.

  2. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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.
  • 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

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): NCT03775421


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

Locations
Show Show 23 study locations
Sponsors and Collaborators
Actelion
Covance
Henry Ford Health System
Almac Clinical Technologies
ActiGraph LLC
Medidata Solutions
Investigators
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Study Director: Thierry Francis Briand, MD Actelion
Additional Information:
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Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT03775421    
Other Study ID Numbers: AC-055H302
2018-002821-45 ( EudraCT Number )
First Posted: December 14, 2018    Key Record Dates
Last Update Posted: August 5, 2020
Last Verified: August 2020
Individual Participant Data (IPD) Sharing Statement:
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Actelion:
Congenital Heart Failure
macitentan
Univentricular Heart
Cavopulmonary Anastomosis
Fontan circulation
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