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Summary

for people ages 2–17 (full criteria)
at San Francisco, California and other locations
study started
estimated completion:

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 Macitentan

Eligibility

For people ages 2–17

Key Inclusion Criteria:

  1. 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.
  2. Males or females between ≥ 2 years and < 18 years of age.
  3. Subjects with body weight ≥ 10 kg at randomization.
  4. Pulmonary arterial hypertension (PAH) diagnosis, confirmed by historical right heart catheterization (mPAP ≥ 25 mmHg, and PAWP ≤ 15 mmHg, and PVRi > 3 WUxm2).
  5. PAH belonging to the Nice 2013 Updated Classification Group 1 (including subjects 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)
  • 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).

Key Exclusion Criteria:

  1. Subjects 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.
  2. Subjects with PAH associated with Eisenmenger syndrome, or with moderate to large left-to-right shunts.
  3. Subjects receiving a combination of > 2 PAH-specific treatments at randomization.
  4. Treatment with i.v. or s.c. prostanoids within 4 weeks before randomization, unless given for vasoreactivity testing.
  5. Hemoglobin or hematocrit <75% of the lower limit of normal range
  6. Serum AST and/or ALT > 3 times the upper limit of normal range'
  7. Pregnancy (including family planning) or breastfeeding.
  8. Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol.

Locations

  • UCLA Children's Heart Center not yet accepting patients
    Los Angeles, California, 90095, United States
  • Children's Heart Center accepting new patients
    Las Vegas, Nevada, 89109, United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Actelion
ID
NCT02932410
Phase
Phase 3
Study Type
Interventional
Last Updated
September 1, 2017
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