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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03153137




Registration number
NCT03153137
Ethics application status
Date submitted
12/05/2017
Date registered
15/05/2017
Date last updated
13/05/2020

Titles & IDs
Public title
Clinical Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Subjects
Scientific 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
Secondary ID [1] 0 0
2016-003320-23
Secondary ID [2] 0 0
AC-055H301
Universal Trial Number (UTN)
Trial acronym
RUBATO
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Congenital Heart Disease 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Coronary heart disease
Cardiovascular 0 0 0 0
Other cardiovascular diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Macitentan 10 mg
Treatment: Drugs - Placebo

Experimental: Macitentan - Macitentan 10 mg per day; film-coated tablet; oral use

Placebo Comparator: Placebo - film-coated tablet; oral use


Treatment: Drugs: Macitentan 10 mg
film-coated tablet; oral use

Treatment: Drugs: Placebo
film-coated tablet; oral use

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in peak VO2 (oxygen uptake) - Change in peak VO2 (oxygen uptake)
Timepoint [1] 0 0
16 weeks, i.e. from Baseline (Randomization/Visit 2) to Week 16 (Visit 4)
Secondary outcome [1] 0 0
Change in peak VO2 - Change in peak VO2
Timepoint [1] 0 0
52 weeks, i.e. from Baseline (Visit 2) over 52 weeks
Secondary outcome [2] 0 0
Physical activity measured by accelerometer - Change in mean count per minute of daily physical activity measured by accelerometer
Timepoint [2] 0 0
16 weeks, i.e. from Baseline (Visit 2) to Week 16 (Visit 4)
Secondary outcome [3] 0 0
Treatment-emergent AEs and SAEs - Treatment-emergent AEs and SAEs
Timepoint [3] 0 0
at least 52 weeks, i.e. up to 30 days after study treatment discontinuation
Secondary outcome [4] 0 0
Trough (pre dose) plasma concentrations - Trough (pre dose) plasma concentrations
Timepoint [4] 0 0
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

Eligibility
Key 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
Minimum age
12 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [3] 0 0
The Prince Charles Hospital, Adult Congenital Heart Disease Unit - Chermside
Recruitment hospital [4] 0 0
Royal Children's Hospital - Parkville
Recruitment hospital [5] 0 0
Royal Melbourne Hospital - Parkville
Recruitment hospital [6] 0 0
Queensland CHILDREN'S HOSPITAL - South Brisbane
Recruitment hospital [7] 0 0
Westmead Hospital - Westmead
Recruitment hospital [8] 0 0
Westmead Private Cardiology - Westmead
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
2050 - Camperdown
Recruitment postcode(s) [3] 0 0
4032 - Chermside
Recruitment postcode(s) [4] 0 0
3052 - Parkville
Recruitment postcode(s) [5] 0 0
4101 - South Brisbane
Recruitment postcode(s) [6] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
District of Columbia
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Iowa
Country [8] 0 0
United States of America
State/province [8] 0 0
Maryland
Country [9] 0 0
United States of America
State/province [9] 0 0
Massachusetts
Country [10] 0 0
United States of America
State/province [10] 0 0
Nebraska
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United States of America
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New York
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United States of America
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Ohio
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United States of America
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Pennsylvania
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United States of America
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Texas
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United States of America
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Utah
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United States of America
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Washington
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Brazil
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Curitiba
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Brazil
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Fortaleza
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Brazil
State/province [19] 0 0
Porto Alegre
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Brazil
State/province [20] 0 0
Sao Paulo
Country [21] 0 0
Brazil
State/province [21] 0 0
São Paulo
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Canada
State/province [22] 0 0
Quebec
Country [23] 0 0
China
State/province [23] 0 0
Beijing
Country [24] 0 0
China
State/province [24] 0 0
Guangzhou
Country [25] 0 0
China
State/province [25] 0 0
Shanghai
Country [26] 0 0
China
State/province [26] 0 0
Wuhan
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Czechia
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Praha 5
Country [28] 0 0
Denmark
State/province [28] 0 0
Copenhagen
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France
State/province [29] 0 0
Montpellier Cedex 5
Country [30] 0 0
France
State/province [30] 0 0
Paris
Country [31] 0 0
France
State/province [31] 0 0
Pessac
Country [32] 0 0
Germany
State/province [32] 0 0
Berlin
Country [33] 0 0
Germany
State/province [33] 0 0
München
Country [34] 0 0
Ireland
State/province [34] 0 0
Dublin
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New Zealand
State/province [35] 0 0
Auckland
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Poland
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Gdansk
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Poland
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Krakow
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Poland
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Kraków
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Poland
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Wroclaw
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Taiwan
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Kaohsiung
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Taiwan
State/province [41] 0 0
Taipei
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United Kingdom
State/province [42] 0 0
Birmingham

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Actelion
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT03153137
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Study Contact
Address 0 0
Country 0 0
Phone 0 0
844-434-4210
Fax 0 0
Email 0 0
JNJ.CT@sylogent.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03153137