Summary

Eligibility
for people ages 2 years and up (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Jeffrey Fineman
Photo of Jeffrey Fineman
Jeffrey Fineman

Description

Summary

This is a prospective, multicenter, open-label, randomized, controlled, parallel group, group-sequential, event-driven Phase 3 study to evaluate efficacy, safety and pharmacokinetics (PK) of macitentan in children.

Official Title

A Multicenter, Open-label, Randomized, Event-driven Study to Assess Efficacy, Safety and Pharmacokinetics of Macitentan Versus Standard of Care in Children With Pulmonary Arterial Hypertension

Keywords

Pulmonary Arterial Hypertension Familial Primary Pulmonary Hypertension Hypertension Macitentan

Eligibility

You can join if…

Open to people ages 2 years and up

  • 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 (>=) 2 years and less than (<) 18 years of age
  • Participants with body weight >= 10 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)

You CAN'T join if...

  • 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])

Locations

  • UCSF Medical Center accepting new patients
    San Francisco California 94143-2202 United States
  • UCLA Children's Heart Center accepting new patients
    Los Angeles California 90095 United States

Lead Scientist at UCSF

  • Jeffrey Fineman
    Professor, Pediatrics. Authored (or co-authored) 194 research publications.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Actelion
ID
NCT02932410
Phase
Phase 3
Study Type
Interventional
Last Updated