Purpose

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.

Condition

Eligibility

Eligible Ages
Between 2 Years and 17 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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:
  6. idiopathic PAH
  7. heritable PAH
  8. PAH associated with congenital heart disease (CHD)
  9. Drug or toxin induced PAH
  10. PAH associated with HIV
  11. PAH associated with connective tissue diseases (PAH-aCTD)
  12. WHO Functional class I to III.
  13. 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

  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.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Macitentan
Macitentan is administered once daily via oral route
  • Drug: Macitentan
    Dispersible tablet; Oral use
    Other names:
    • ACT-064992
Other
Standard-of-care
Standard-of-care as per site's clinical practice which may comprise treatment with PAH non-specific treatment and/or up to two PAH-specific medications excluding macitentan and i.v./s.c. prostanoids.
  • Other: Standard-of-care
    Standard-of-care as per site's clinical practice which may comprise treatment with PAH non-specific treatment and/or up to two PAH-specific medications excluding macitentan and i.v./s.c. prostanoids.

Recruiting Locations

Montefiore Medical Center
Bronx, New York 10467
Contact:
Principal Investigator
718-920-2183
nsutton@montefiore.org

More Details

NCT ID
NCT02932410
Status
Recruiting
Sponsor
Actelion

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.