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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03775421




Registration number
NCT03775421
Ethics application status
Date submitted
21/11/2018
Date registered
14/12/2018

Titles & IDs
Public title
An Upcoming Clinical Study to Measure the Safety and Impact of a Drug Called Macitentan in Teenage and Adult Fontan Patients.
Scientific 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
Secondary ID [1] 0 0
2018-002821-45
Secondary ID [2] 0 0
AC-055H302
Universal Trial Number (UTN)
Trial acronym
RUBATO OL
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Congenital Heart Disease With Fontan Circulation 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

Experimental: Open-label treatment period - oral administration of 10 mg macitentan once daily


Treatment: Drugs: macitentan 10 mg
macitentan 10 mg, film-coated tablet, oral use
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
Up to 133 weeks
Primary outcome [2] 0 0
Number of Participants With Treatment-emergent Serious AEs (TESAEs)
Timepoint [2] 0 0
Up to 133 weeks
Primary outcome [3] 0 0
Number of Participants With TEAEs Leading to Death
Timepoint [3] 0 0
Up to 133 weeks
Primary outcome [4] 0 0
Number of Participants With TEAEs Leading to Premature Discontinuation of Study Treatment
Timepoint [4] 0 0
Up to 133 weeks
Primary outcome [5] 0 0
Number of Participants With Treatment-emergent Marked Laboratory Abnormalities up to 30 Days After Study Treatment Discontinuation
Timepoint [5] 0 0
Up to 133 weeks
Primary outcome [6] 0 0
Change From Baseline in Hemoglobin Over Time
Timepoint [6] 0 0
Baseline up to Week 130
Primary outcome [7] 0 0
Change From Baseline in Hematocrit Over Time
Timepoint [7] 0 0
Baseline up to Week 130
Primary outcome [8] 0 0
Change From Baseline in Leukocytes, Neutrophils, Lymphocytes, and Platelets Over Time
Timepoint [8] 0 0
Baseline up to Week 130
Primary outcome [9] 0 0
Change From Baseline in Systolic and Diastolic Arterial Blood Pressure (BP) Over Time
Timepoint [9] 0 0
Baseline up to Week 130
Primary outcome [10] 0 0
Change From Baseline in Pulse Rate Over Time
Timepoint [10] 0 0
Baseline up to Week 130
Primary outcome [11] 0 0
Change From Baseline in Peripheral Oxygen Saturation (SpO2) Over Time
Timepoint [11] 0 0
Baseline up to Week 130
Primary outcome [12] 0 0
Change From Baseline in Body Weight Over Time
Timepoint [12] 0 0
Baseline up to Week 130
Primary outcome [13] 0 0
Change From Baseline in Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Alkaline Phosphatase (AP), and Gamma Glutamyl Transferase (GGT) Over Time
Timepoint [13] 0 0
Baseline up to Week 130
Primary outcome [14] 0 0
Change From Baseline in Bilirubin, Direct Bilirubin, and Creatinine Over Time
Timepoint [14] 0 0
Baseline up to Week 130
Primary outcome [15] 0 0
Change From Baseline in Glomerular Filtration Rate (GFR) Over Time
Timepoint [15] 0 0
Baseline up to Week 130
Primary outcome [16] 0 0
Change From Baseline in Prothrombin Time Over Time
Timepoint [16] 0 0
Baseline up to Week 130
Primary outcome [17] 0 0
Change From Baseline in Prothrombin International Normalized Ratio Over Time
Timepoint [17] 0 0
Baseline up to Week 130
Secondary outcome [1] 0 0
Change From Baseline in Peak Oxygen Uptake/Consumption (VO2)
Timepoint [1] 0 0
Baseline, Week 52, and Week 104
Secondary outcome [2] 0 0
Change From Baseline in Mean Count Per Minute of Daily Physical Activity Measured by Accelerometer (PA-Ac)
Timepoint [2] 0 0
Baseline, Week 26, Week 52, Week 78, and Week 104

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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
11/04/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
18/01/2022
Sample size
Target
Accrual to date
Final
112
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 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 outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Massachusetts
Country [2] 0 0
United States of America
State/province [2] 0 0
Washington
Country [3] 0 0
Canada
State/province [3] 0 0
Quebec
Country [4] 0 0
China
State/province [4] 0 0
Beijing
Country [5] 0 0
China
State/province [5] 0 0
Shanghai
Country [6] 0 0
Czechia
State/province [6] 0 0
Praha 5
Country [7] 0 0
Denmark
State/province [7] 0 0
Copenhagen
Country [8] 0 0
France
State/province [8] 0 0
Paris
Country [9] 0 0
France
State/province [9] 0 0
Pessac
Country [10] 0 0
New Zealand
State/province [10] 0 0
Auckland
Country [11] 0 0
Poland
State/province [11] 0 0
Gdansk
Country [12] 0 0
Poland
State/province [12] 0 0
Krakow
Country [13] 0 0
Poland
State/province [13] 0 0
Wroclaw
Country [14] 0 0
Taiwan
State/province [14] 0 0
Taipei
Country [15] 0 0
United Kingdom
State/province [15] 0 0
Birmingham

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Actelion
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Covance
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Henry Ford Health System
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Almac Clinical Technologies
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
ActiGraph LLC
Address [4] 0 0
Country [4] 0 0
Other collaborator category [5] 0 0
Commercial sector/industry
Name [5] 0 0
Medidata Solutions
Address [5] 0 0
Country [5] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Thierry Francis Briand, MD
Address 0 0
Actelion
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)?
Yes
What data in particular will be shared?
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
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.janssen.com/clinical-trials/transparency


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/NCT03775421