Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05179876
Registration number
NCT05179876
Ethics application status
Date submitted
17/12/2021
Date registered
5/01/2022
Date last updated
8/11/2023
Titles & IDs
Public title
A Study Providing Treatment Access in Participants With Pulmonary Hypertension Completing a Parent Study and Having no Other Option
Query!
Scientific title
A Prospective, Open-label, Platform Study for Long-term Follow-up of Participants Using Study Intervention in Pulmonary Hypertension Parent Studies
Query!
Secondary ID [1]
0
0
2021-002297-11
Query!
Secondary ID [2]
0
0
CR109121
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
PLATYPUS
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Hypertension, Pulmonary
0
0
Query!
Condition category
Condition code
Respiratory
0
0
0
0
Query!
Other respiratory disorders / diseases
Query!
Cardiovascular
0
0
0
0
Query!
Hypertension
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - Macitentan
Treatment: Drugs - Selexipag
Treatment: Drugs - Macitentan/Tadalafil FDC
Experimental: Macitentan - Participants who have completed a parent study, benefit from their study intervention maintenance and have no adequate alternative local treatment option will be enrolled in this study and will continue to receive study drug macitentan orally during the course of the study. For adult participants study visits will be scheduled every 6 months and for pediatric participants study visits will be scheduled every 3 months. The study includes on-site visits to collect efficacy and safety information until participant discontinuation/withdrawal, or the respective study intervention is made commercially available in the country/territory or an equivalent approved therapy becomes available, or the sponsor decides to terminate the study prematurely.
Experimental: Selexipag - Participants who have completed a parent study, benefit from their study intervention maintenance and have no adequate alternative local treatment option will be enrolled in this study and will continue to receive study drug selexipag orally during the course of the study. Study visits are scheduled every 6 months to collect efficacy and safety information until participant discontinuation/withdrawal, or the respective study intervention is made commercially available in the country/territory or an equivalent approved therapy becomes available, or the sponsor decides to terminate the study prematurely.
Experimental: Macitentan/Tadalafil FDC - Participants who have completed a parent study, benefit from their study intervention maintenance and have no adequate alternative local treatment option will be enrolled in this study and will continue to receive drug Macitentan and Tadalafil fixed dose combination (FDC) orally during the course of the study. Study visits are scheduled every 6 months to collect efficacy and safety information until participant discontinuation/withdrawal, or the respective study intervention is made commercially available in the country/territory or an equivalent approved therapy becomes available, or the sponsor decides to terminate the study prematurely.
Treatment: Drugs: Macitentan
Adult participants will receive oral dose of macitentan 10 milligrams (mg) tablet once daily. Children greater than or equal to (>=) 2 year to less than (<) 18 years will be given an oral macitentan dose tailored to their body weight, ensuring an equivalent level of systemic exposure as in adults.
Treatment: Drugs: Selexipag
Participant will receive oral dose of selexipag tablet twice daily at the dose strength corresponding to their maintenance dose at the end of their parent study. Available strengths: 200, 400, 600, 800, 1000, 1200, 1400 and 1600 micrograms.
Treatment: Drugs: Macitentan/Tadalafil FDC
Participants will receive oral FDC of macitentan 10 mg and tadalafil 40 mg once daily during the course of the study as already received in the parent studies.
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Frequency of Treatment Emergent Adverse Events (TEAEs)
Query!
Assessment method [1]
0
0
An adverse event (AE) is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention. TEAEs are defined as AEs with onset or worsening on or after date of first dose of study treatment.
Query!
Timepoint [1]
0
0
Baseline until End of Study (EOS) (up to 57 months)
Query!
Primary outcome [2]
0
0
Frequency of TEAEs Leading to Discontinuation
Query!
Assessment method [2]
0
0
Frequency of TEAEs leading to discontinuation of study intervention will be reported. An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention. TEAEs are defined as AEs with onset or worsening on or after date of first dose of study treatment.
Query!
Timepoint [2]
0
0
Baseline until EOS (up to 57 months)
Query!
Primary outcome [3]
0
0
Frequency of Serious Adverse Events (SAEs)
Query!
Assessment method [3]
0
0
SAE is any untoward medical occurrence that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a suspected transmission of any infectious agent via a medicinal product and is medically important.
Query!
Timepoint [3]
0
0
Baseline until EOS (up to 57 months)
Query!
Primary outcome [4]
0
0
Frequency of Deaths
Query!
Assessment method [4]
0
0
Frequency of deaths will be reported.
Query!
Timepoint [4]
0
0
Baseline until EOS (up to 57 months)
Query!
Eligibility
Key inclusion criteria
- Participant must sign an informed consent form (ICF) (or their legally acceptable
representative must sign) indicating that participant understands the purpose of, and
procedures required for, the study and is willing to participate in the study
- Participant treated with oral macitentan or selexipag or fixed dose combination (FDC)
of macitentan 10 milligrams (mg) and tadalafil 40 mg at the end of a sponsor parent
study and: a) the indication of the parent study is included in the
intervention-specific appendices (ISA) (pulmonary arterial hypertension [PAH] or
chronic thromboembolic pulmonary hypertension [CTEPH] for adults, PAH for pediatric
participants); b) participant has completed the parent study; c) no alternative means
of access to study intervention (or equivalent approved therapy) have been identified;
d) participant may continue to benefit from treatment with the study intervention; e)
Participant is at least 18 years old for selexipag or macitentan/tadalafil FDC, and at
least 2 years old for macitentan
- A female participant of childbearing potential must: a) have a negative urine or serum
pregnancy test prior to first intake of study intervention; b) agree to perform
monthly urine pregnancy test up to the end of the safety follow-up period; c) agree to
follow contraceptive methods until 30 days after the last intake of the study
intervention
Query!
Minimum age
2
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
General:
- Participants prematurely discontinued from the study intervention in their parent
study
- Female participant being pregnant, or breastfeeding, or planning to become pregnant
while enrolled in this study
- Planned or current treatment with another investigational treatment
Macitentan-specific:
- Known allergies, hypersensitivity, or intolerance to macitentan or its excipients
- Hemoglobin less than (<) 80 grams per liter (g/L)
- Serum aspartate (AST) and/or alanine aminotransferases (ALT) greater than (>) 3* upper
limit of normal (ULN)
- Known and documented severe hepatic impairment that is, Child-Pugh Class C. For
participants with hepatic impairment, Child-Pugh Class (Child-Pugh score) should be
fully assessed and documented in the source documents at screening
Selexipag-specific:
- Known allergies, hypersensitivity, or intolerance to selexipag or its excipients
- Suspected or known pulmonary veno-occlusive disease (PVOD)
- Uncontrolled thyroid disease
- Severe coronary heart disease or unstable angina, myocardial infarction within the
last 6 months, decompensated cardiac failure (if not under close medical supervision),
severe arrhythmia, cerebrovascular events (for example, transient ischemic attack,
stroke) within the last 3 months, or congenital or acquired valvular defects with
clinically relevant myocardial function disorders not related to pulmonary
hypertension (PH)
- Known and documented severe hepatic impairment that is, Child-Pugh Class C. For
participants with hepatic impairment, Child-Pugh Class (Child-Pugh score) should be
fully assessed and documented in the source documents at screening
Macitentan/tadalafil FDC-specific:
- Known allergies, hypersensitivity, or intolerance to macitentan or tadalafil or their
excipients
- Hemoglobin <80 g/L
- Serum aspartate (AST) and/or alanine aminotransferases (ALT) >3* ULN range
- Known and documented severe hepatic impairment that is, Child-Pugh Class C. For
participants with hepatic impairment, Child-Pugh Class should be fully assessed and
documented in the source documents at screening
- Severe renal impairment (estimated glomerular filtration rate [eGF]/creatinine
clearance <30 milliliter per minute [mL/min])
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Non-randomised trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
4/05/2022
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
31/01/2027
Query!
Actual
Query!
Sample size
Target
230
Query!
Accrual to date
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment outside Australia
Country [1]
0
0
Belarus
Query!
State/province [1]
0
0
Minsk
Query!
Country [2]
0
0
Belgium
Query!
State/province [2]
0
0
Leuven
Query!
Country [3]
0
0
Korea, Republic of
Query!
State/province [3]
0
0
Daejeon
Query!
Country [4]
0
0
Korea, Republic of
Query!
State/province [4]
0
0
Incheon
Query!
Country [5]
0
0
Korea, Republic of
Query!
State/province [5]
0
0
Seoul
Query!
Country [6]
0
0
Poland
Query!
State/province [6]
0
0
Bydgoszcz
Query!
Country [7]
0
0
Poland
Query!
State/province [7]
0
0
Lodz
Query!
Country [8]
0
0
Poland
Query!
State/province [8]
0
0
Lublin
Query!
Country [9]
0
0
Poland
Query!
State/province [9]
0
0
Szczecin
Query!
Country [10]
0
0
Poland
Query!
State/province [10]
0
0
Wroclaw
Query!
Country [11]
0
0
South Africa
Query!
State/province [11]
0
0
Durban
Query!
Country [12]
0
0
Taiwan
Query!
State/province [12]
0
0
Kaohsiung
Query!
Country [13]
0
0
Taiwan
Query!
State/province [13]
0
0
Tainan
Query!
Country [14]
0
0
Taiwan
Query!
State/province [14]
0
0
Taipei
Query!
Country [15]
0
0
Ukraine
Query!
State/province [15]
0
0
Dnipro
Query!
Country [16]
0
0
Ukraine
Query!
State/province [16]
0
0
Kyiv
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Actelion
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
The purpose of the study is to enable participants with pulmonary hypertension (PH) currently
treated with study intervention(s) in a clinical study (parent studies [NCT03422328,
NCT03904693 and NCT04565990]), to continue to benefit from the intervention after closure of
the parent study in case they have no alternative means of access to the study intervention.
This study will allow assessment of the long-term safety of each study intervention.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT05179876
Query!
Trial related presentations / publications
Query!
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed
Query!
Contacts
Principal investigator
Name
0
0
Actelion Clinical Trial
Query!
Address
0
0
Actelion
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Study Contact
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
844-434-4210
Query!
Fax
0
0
Query!
Email
0
0
[email protected]
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05179876
Download to PDF