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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05179876




Registration number
NCT05179876
Ethics application status
Date submitted
17/12/2021
Date registered
5/01/2022

Titles & IDs
Public title
A Study Providing Treatment Access in Participants With Pulmonary Hypertension Completing a Parent Study and Having no Other Option
Scientific title
A Prospective, Open-label, Platform Study for Long-term Follow-up of Participants Using Study Intervention in Pulmonary Hypertension Parent Studies
Secondary ID [1] 0 0
NOPRODPAPUH3001
Secondary ID [2] 0 0
CR109121
Universal Trial Number (UTN)
Trial acronym
PLATYPUS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypertension, Pulmonary 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Cardiovascular 0 0 0 0
Hypertension

Intervention/exposure
Study type
Interventional
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
Adult 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 (µg). Children with body weight category of \>=50 kg will use the tablets at the required dose strength as described for adults. Children with a body weight \< 50 kg will receive tablets for pediatric use (dose strengths: 100 and 150 mcg), twice daily to enable continuation of individually maximum tolerated dose of selexipag according to their body weight category.

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

Outcomes
Primary outcome [1] 0 0
Frequency of Treatment Emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
Baseline until End of Study (EOS) (up to 84 months)
Primary outcome [2] 0 0
Frequency of TEAEs Leading to Discontinuation
Timepoint [2] 0 0
Baseline until EOS (up to 84 months)
Primary outcome [3] 0 0
Frequency of Serious Adverse Events (SAEs)
Timepoint [3] 0 0
Baseline until EOS (up to 84 months)
Primary outcome [4] 0 0
Frequency of Deaths
Timepoint [4] 0 0
Baseline until EOS (up to 84 months)

Eligibility
Key inclusion criteria
* Participant must sign an informed consent form (ICF) (or their legally designated 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]; 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 macitentan/tadalafil FDC, and at least 2 years old for macitentan or selexipag
* 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) If heterosexually active, agree to follow contraceptive methods until 30 days after the last intake of the study intervention. For pediatric female participants: It is the responsibility of the investigator to ensure appropriate counselling, including consultation with a specialist (if needed), to the participant and/or parent(s)/ legally designated representative (LDR)(s) on the acceptable method of contraception
Minimum age
2 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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
* Children only: (a) Current suspicion of intussusception or ileus or gastrointestinal obstruction, per the investigator's judgment; (b) hemoglobin or hematocrit <75 percent (%) of the lower limit of normal range

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])

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



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
4/05/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
28/09/2029
Actual
Sample size
Target
280
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
Belarus
State/province [1] 0 0
Minsk
Country [2] 0 0
Belgium
State/province [2] 0 0
Leuven
Country [3] 0 0
Bulgaria
State/province [3] 0 0
Sofia
Country [4] 0 0
China
State/province [4] 0 0
Beijing
Country [5] 0 0
China
State/province [5] 0 0
Changsha
Country [6] 0 0
China
State/province [6] 0 0
Nanjing
Country [7] 0 0
China
State/province [7] 0 0
Xi'An
Country [8] 0 0
Korea, Republic of
State/province [8] 0 0
Daejeon
Country [9] 0 0
Korea, Republic of
State/province [9] 0 0
Incheon
Country [10] 0 0
Korea, Republic of
State/province [10] 0 0
Seoul
Country [11] 0 0
Poland
State/province [11] 0 0
Bialystok
Country [12] 0 0
Poland
State/province [12] 0 0
Bydgoszcz
Country [13] 0 0
Poland
State/province [13] 0 0
Katowice
Country [14] 0 0
Poland
State/province [14] 0 0
Lodz
Country [15] 0 0
Poland
State/province [15] 0 0
Lublin
Country [16] 0 0
Poland
State/province [16] 0 0
Szczecin
Country [17] 0 0
Poland
State/province [17] 0 0
Wroclaw
Country [18] 0 0
Russian Federation
State/province [18] 0 0
Kemerovo
Country [19] 0 0
Russian Federation
State/province [19] 0 0
St Petersburg
Country [20] 0 0
Russian Federation
State/province [20] 0 0
Tomsk
Country [21] 0 0
South Africa
State/province [21] 0 0
Durban
Country [22] 0 0
South Africa
State/province [22] 0 0
Lenasia
Country [23] 0 0
Taiwan
State/province [23] 0 0
Kaohsiung
Country [24] 0 0
Taiwan
State/province [24] 0 0
Tainan
Country [25] 0 0
Taiwan
State/province [25] 0 0
Taipei
Country [26] 0 0
Taiwan
State/province [26] 0 0
Taoyuan
Country [27] 0 0
Ukraine
State/province [27] 0 0
Dnipro
Country [28] 0 0
Ukraine
State/province [28] 0 0
Kyiv

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
Actelion Pharmaceuticals Ltd Clinical Trial
Address 0 0
Actelion
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
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05179876