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

Recruiting
2 years of age
Both
Phase 3

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Overview

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.

Eligibility

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

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])

Study details

Hypertension, Pulmonary

NCT05179876

Actelion

12 January 2025

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FAQs

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A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

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Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

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The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

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Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
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