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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03153137
Ethics application status
Date submitted
12/05/2017
Date registered
15/05/2017

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
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

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 From Baseline in Peak Oxygen Uptake/Consumption (VO2) Up to Week 16
Timepoint [1] 0 0
Baseline up to Week 16
Secondary outcome [1] 0 0
Change From Baseline in Peak VO2 Up to Week 52
Timepoint [1] 0 0
Baseline up to Week 52
Secondary outcome [2] 0 0
Change From Baseline in Mean Count Per Minute of Daily Physical Activity Measured by Accelerometer (PA-Ac) Up to Week 16
Timepoint [2] 0 0
Baseline up to Week 16
Secondary outcome [3] 0 0
Number of Participants With Treatment-emergent Serious Adverse Events (SAEs)
Timepoint [3] 0 0
Up to 56 weeks
Secondary outcome [4] 0 0
Number of Participants With Treatment-emergent Adverse Events (AEs)
Timepoint [4] 0 0
Up to 56 weeks
Secondary outcome [5] 0 0
Number of Participants With AEs Leading to Premature Discontinuation of Study Treatment
Timepoint [5] 0 0
Up to 56 weeks
Secondary outcome [6] 0 0
Change From Baseline in Systolic and Diastolic Arterial Blood Pressure (BP)
Timepoint [6] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [7] 0 0
Change From Baseline in Pulse Rate
Timepoint [7] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [8] 0 0
Change From Baseline in Oxygen Saturation (SpO2)
Timepoint [8] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [9] 0 0
Change From Baseline in Body Weight
Timepoint [9] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [10] 0 0
Number of Participants With Treatment-emergent Markedly Abnormal Laboratory Values
Timepoint [10] 0 0
Up to 56 weeks
Secondary outcome [11] 0 0
Change From Baseline in Hemoglobin
Timepoint [11] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [12] 0 0
Change From Baseline in Hematocrit
Timepoint [12] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [13] 0 0
Change From Baseline in Erythrocytes and Reticulocytes
Timepoint [13] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [14] 0 0
Change From Baseline in Leucocytes, Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils and Platelets
Timepoint [14] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [15] 0 0
Change From Baseline in Prothrombin Time
Timepoint [15] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [16] 0 0
Change From Baseline in Prothrombin International Normalized Ratio
Timepoint [16] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [17] 0 0
Change From Baseline in Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST) and Alkaline Phosphatase (AP)
Timepoint [17] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [18] 0 0
Change From Baseline in Bilirubin and Direct Bilirubin
Timepoint [18] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [19] 0 0
Change From Baseline in Gamma Glutamyl Transferase
Timepoint [19] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [20] 0 0
Change From Baseline in Creatinine
Timepoint [20] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [21] 0 0
Change From Baseline in Urea Nitrogen
Timepoint [21] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [22] 0 0
Change From Baseline in Urate
Timepoint [22] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [23] 0 0
Change From Baseline in Glucose, Cholesterol, Triglycerides, Sodium, Potassium, Chloride and Calcium
Timepoint [23] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [24] 0 0
Change From Baseline in Albumin and Protein
Timepoint [24] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [25] 0 0
Change From Baseline in Alpha Fetoprotein
Timepoint [25] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52
Secondary outcome [26] 0 0
Change From Baseline in Cystatin C
Timepoint [26] 0 0
Baseline, Week 8, Week 16, Week 32 and Week 52

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
Sex
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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/08/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/07/2021
Sample size
Target
Accrual to date
Final
142
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
Queensland CHILDREN'S HOSPITAL - South Brisbane
Recruitment hospital [6] 0 0
Westmead Hospital - 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
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Ohio
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
United States of America
State/province [6] 0 0
Washington
Country [7] 0 0
Canada
State/province [7] 0 0
Quebec
Country [8] 0 0
China
State/province [8] 0 0
Beijing
Country [9] 0 0
China
State/province [9] 0 0
Shanghai
Country [10] 0 0
China
State/province [10] 0 0
Wuhan
Country [11] 0 0
Czechia
State/province [11] 0 0
Praha 5
Country [12] 0 0
Denmark
State/province [12] 0 0
Copenhagen
Country [13] 0 0
France
State/province [13] 0 0
Montpellier Cedex 5
Country [14] 0 0
France
State/province [14] 0 0
Paris
Country [15] 0 0
France
State/province [15] 0 0
Pessac
Country [16] 0 0
Germany
State/province [16] 0 0
Berlin
Country [17] 0 0
Germany
State/province [17] 0 0
München
Country [18] 0 0
New Zealand
State/province [18] 0 0
Auckland
Country [19] 0 0
Poland
State/province [19] 0 0
Gdansk
Country [20] 0 0
Poland
State/province [20] 0 0
Krakow
Country [21] 0 0
Poland
State/province [21] 0 0
Kraków
Country [22] 0 0
Poland
State/province [22] 0 0
Wroclaw
Country [23] 0 0
Taiwan
State/province [23] 0 0
Taipei
Country [24] 0 0
United Kingdom
State/province [24] 0 0
Birmingham

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03153137