A Study Providing Treatment Access in Participants With Pulmonary Hypertension Completing a Parent Study and Having no Other Option

Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2+
SponsorActelion

About this trial

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 \[NCT03422328, NCT03904693,NCT04565990, NCT02932410, NCT03492177, and NCT04175600\]), 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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Known allergies, hypersensitivity, or intolerance to macitentan or its excipients

Trial design

Design model

Parallel

Treatments tested in this trial

  • Macitentan

    Drug

    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.

  • Selexipag

    Drug

    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.

  • Macitentan/Tadalafil FDC

    Drug

    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.

Treatment groups

280 Participants
are divided into 3 treatment groups
Group A: MacitentanExperimental treatment 1 intervention
Group B: SelexipagExperimental treatment 1 intervention
Group C: Macitentan/Tadalafil FDCExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Frequency of Treatment Emergent Adverse Events (TEAEs)

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 a treatment-emergent AE is any AE temporally associated with the use of study treatment (from start of treatment in the PLATYPUS protocol until 30 days after study treatment discontinuation) whether or not considered by the investigator as related to study treatment.

Time frame
Baseline until End of Study (EOS) (up to 84 months)
2

Frequency of TEAEs Leading to Discontinuation

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 a treatment-emergent AE is any AE temporally associated with the use of study treatment (from start of treatment in the PLATYPUS protocol until 30 days after study treatment discontinuation) whether or not considered by the investigator as related to study treatment.

Time frame
Baseline until EOS (up to 84 months)
3

Frequency of Serious Adverse Events (SAEs)

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.

Time frame
Baseline until EOS (up to 84 months)
4

Frequency of Deaths

Frequency of deaths will be reported.

Time frame
Baseline until EOS (up to 84 months)

Secondary outcomes

Other outcomes

Sponsors and contacts

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