A Multicenter, Open-label, Randomized, Study With Single-arm Extension Period to Assess the Pharmacokinetics, Safety and Efficacy of Macitentan Versus Standard of Care in Children With Pulmonary Arterial Hypertension (TOMORROW)

STATUS

Recruiting
End date

Feb 29, 2024
participants needed

300
sponsor

Actelion

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Updated on 20 October 2022

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hypertension

heart disease

connective tissue disease

macitentan

Summary

This is a prospective, multicenter, open-label, randomized, controlled, parallel Phase 3 study with an open-label single-arm extension period to evaluate pharmacokinetics (PK), safety and efficacy of macitentan in children with pulmonary arterial hypertension (PAH).

Details

Condition	Pulmonary Arterial Hypertension
Treatment	Macitentan, Standard-of-Care
Clinical Study Identifier	NCT02932410
Sponsor	Actelion
Last Modified on	20 October 2022

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Eligibility		Yes	No	Not Sure
Inclusion Criteria
	Signed informed consent by the parent(s) or legally designated representative and assent from developmentally capable children prior to initiation of any study-mandated procedure
	Males or females between greater than or equal to (>=) 1 month and less than (<) 18 years of age
	Participants with body weight >= 3.5 kilograms (kg) at randomization
	Pulmonary arterial hypertension (PAH) diagnosis confirmed by historical RHC (mPAP greater than or equal to [>=] 25 millimeters of mercury [mmHg], and Pulmonary artery wedge pressure [PAWP] less than or equal to [<=] 15 mmHg, and Pulmonary vascular resistance index [PVRi] greater than [>] 3 WU × m2), where in the absence of pulmonary vein obstruction and/or significant lung disease PAWP can be replaced by Left atrium pressure [LAP] or Left ventricular end diastolic pressure [LVEDP] (in absence of mitral stenosis) assessed by heart catheterization
	PAH belonging to the Nice 2013 Updated Classification Group 1 (including participants with Down Syndrome) and of following etiologies: idiopathic PAH; heritable PAH; PAH associated with congenital heart disease (CHD); Drug or toxin induced PAH; PAH associated with HIV; PAH associated with connective tissue diseases (PAH-aCTD); and World health organization (WHO) Functional class I to III
	Females of childbearing potential must have a negative pregnancy test at Screening and at Baseline, and must agree to undertake monthly pregnancy tests, and to use a reliable method of contraception (if sexually active) up to the end of study (EOS)
Exclusion Criteria
	Participants with PAH due to portal hypertension, schistosomiasis, or with pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis, and persistent pulmonary hypertension of the newborn
	Participants with PAH associated with Eisenmenger syndrome, or with moderate to large left-to-right shunts
	Participants receiving a combination of > 2 PAH-specific treatments at randomization
	Treatment with intravenous (IV) or subcutaneous (SC) prostanoids within 4 weeks before randomization, unless given for vasoreactivity testing
	Hemoglobin or hematocrit <75 percent (%) of the lower limit of normal range
	Serum Aspartate aminotransferase (AST) and/or Alanine aminotransferase (ALT) greater than (>) 3 times the upper limit of normal range
	Pregnancy (including family planning) or breastfeeding
	Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol
	Severe hepatic impairment, for example Child-Pugh Class C
	Clinical signs of hypotension which in the investigator's judgment would preclude initiation of a PAH-specific therapy
	Severe renal insufficiency (estimated creatinine clearance <30 mL/min or serum creatinine >221 micro-moles per liter [micro-mol/L])
	Participants with known diagnosis of bronchopulmonary dysplasia

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A Multicenter, Open-label, Randomized, Study With Single-arm Extension Period to Assess the Pharmacokinetics, Safety and Efficacy of Macitentan Versus Standard of Care in Children With Pulmonary Arterial Hypertension (TOMORROW)