A Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension (PAH)

Description

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.

Study Start Date

December, 05 2017

Estimated Completion Date

July 2022

Interventions

  • Drug: Macitentan
  • Other: Standard-of-care

Study ID

Actelion -- AC-055-312

Status

Recruiting

Trial ID

NCT02932410

Study Type

Interventional

Trial Phase

Phase 3

Enrollment Quota

300

Sponsor

Actelion

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) 6. WHO Functional class I to III. 7. 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.

Gender

All

Ages

2 Years to 17 Years

Accepts Healthy Volunteers

No

Study Locations and Contact Information (55)

Study Location Distance Name Phone Email
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Principal Investigator 617-355-7866 mary.mullen@cardio.chboston.org
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Principal Investigator 617-355-7866 mary.mullen@cardio.chboston.org
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Principal Investigator 617-355-7866 mary.mullen@cardio.chboston.org
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Principal Investigator 617-355-7866 mary.mullen@cardio.chboston.org
Montefiore Medical Center - The Bronx, New York 176.6 miles Principal Investigator 718-920-2183 nsutton@montefiore.org
Morgan Stanley Childrens Hospital of New York Presbyterian CHONY PIN - New York, New York 181.2 miles Principal Investigator 212-305-4436 usk1@cumc.columbia.edu
Morgan Stanley Childrens Hospital of New York Presbyterian CHONY PIN - New York, New York 181.2 miles Principal Investigator 212-305-4436 usk1@cumc.columbia.edu
Morgan Stanley Childrens Hospital of New York Presbyterian CHONY PIN - New York, New York 181.2 miles Principal Investigator 212-305-4436 usk1@cumc.columbia.edu
Morgan Stanley Childrens Hospital of New York Presbyterian CHONY PIN - New York, New York 181.2 miles Principal Investigator 212-305-4436 usk1@cumc.columbia.edu
Childrens National Medical Center - Washington, District of Columbia 391.7 miles Principal Investigator 202-476-5000 jberger@cnmc.org
Childrens National Medical Center - Washington, District of Columbia 391.7 miles Principal Investigator 202-476-5000 jberger@cnmc.org
Childrens National Medical Center - Washington, District of Columbia 391.7 miles Principal Investigator 202-476-5000 jberger@cnmc.org
Childrens National Medical Center - Washington, District of Columbia 391.7 miles Principal Investigator 202-476-5000 jberger@cnmc.org
University Hospitals Cleveland Medical Center Rainbow Babies and Childrens Hospital - Cleveland, Ohio 544.0 miles Principal Investigator 216-844-8180 amy.dimarino@uhhospitals.org
University Hospitals Cleveland Medical Center Rainbow Babies and Childrens Hospital - Cleveland, Ohio 544.0 miles Principal Investigator 216-844-8180 amy.dimarino@uhhospitals.org
University Hospitals Cleveland Medical Center Rainbow Babies and Childrens Hospital - Cleveland, Ohio 544.0 miles Principal Investigator 216-844-8180 amy.dimarino@uhhospitals.org
University Hospitals Cleveland Medical Center Rainbow Babies and Childrens Hospital - Cleveland, Ohio 544.0 miles Principal Investigator 216-844-8180 amy.dimarino@uhhospitals.org
East Carolina University - Greenville, North Carolina 577.7 miles Principal Investigator 252-744-2161 sangc@ecu.edu
Duke University Medical Center - Durham, North Carolina 608.4 miles Principal Investigator 919-668-8305 kevin.hill@duke.edu
Detroit Medical Center - Detroit, Michigan 611.5 miles Principal Investigator 313-745-5956 rross@dmc.org
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles Principal Investigator 614-722-9479 Curt.daniels@nationwidechildrens.org
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles Principal Investigator 614-722-9479 Curt.daniels@nationwidechildrens.org
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles Principal Investigator 614-722-9479 Curt.daniels@nationwidechildrens.org
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles Principal Investigator 614-722-9479 Curt.daniels@nationwidechildrens.org
Cincinnati Childrens Hospital Medical Center - Cincinnati, Ohio 735.6 miles Principal Investigator 513-636-7072 russel.hirsch@cchmc.org
Cincinnati Childrens Hospital Medical Center - Cincinnati, Ohio 735.6 miles Principal Investigator 513-636-7072 russel.hirsch@cchmc.org
Cincinnati Childrens Hospital Medical Center - Cincinnati, Ohio 735.6 miles Principal Investigator 513-636-7072 russel.hirsch@cchmc.org
Cincinnati Childrens Hospital Medical Center - Cincinnati, Ohio 735.6 miles Principal Investigator 513-636-7072 russel.hirsch@cchmc.org
Riley Hospital For Children - Indianapolis, Indiana 805.0 miles Principal Investigator 317-274-7208 gsmontgo@iu.edu
Childrens Hospital of Winsconsin - Milwaukee, Wisconsin 862.1 miles Principal Investigator 414-266-2380 ekirkpatrick@chw.org
Childrens Hospital of Winsconsin - Milwaukee, Wisconsin 862.1 miles Principal Investigator 414-266-2380 ekirkpatrick@chw.org
Childrens Hospital of Winsconsin - Milwaukee, Wisconsin 862.1 miles Principal Investigator 414-266-2380 ekirkpatrick@chw.org
Childrens Hospital of Winsconsin - Milwaukee, Wisconsin 862.1 miles Principal Investigator 414-266-2380 ekirkpatrick@chw.org
Mayo Clinic - Rochester, Minnesota 1,083.1 miles Principal Investigator 507-284-0676 johnson.jonathan@mayo.edu
Texas Childrens Hospital - Houston, Texas 1,609.9 miles Principal Investigator 601-984-5208 feruiz@texaschildrens.org
Texas Childrens Hospital - Houston, Texas 1,609.9 miles Principal Investigator 601-984-5208 feruiz@texaschildrens.org
Texas Childrens Hospital - Houston, Texas 1,609.9 miles Principal Investigator 601-984-5208 feruiz@texaschildrens.org
Texas Childrens Hospital - Houston, Texas 1,609.9 miles Principal Investigator 601-984-5208 feruiz@texaschildrens.org
Colorado Childrens Hospital - Aurora, Colorado 1,759.8 miles Principal Investigator 720-777-6820 Dunbar.ivy@childrenscolorado.org
Colorado Childrens Hospital - Aurora, Colorado 1,759.8 miles Principal Investigator 720-777-6820 Dunbar.ivy@childrenscolorado.org
Colorado Childrens Hospital - Aurora, Colorado 1,759.8 miles Principal Investigator 720-777-6820 Dunbar.ivy@childrenscolorado.org
Colorado Childrens Hospital - Aurora, Colorado 1,759.8 miles Principal Investigator 720-777-6820 Dunbar.ivy@childrenscolorado.org
Primary Childrens Medical Center - Salt Lake City, Utah 2,093.2 miles Principal Investigator 801-662-5400 ron.day@hsc.utah.edu
Primary Childrens Medical Center - Salt Lake City, Utah 2,093.2 miles Principal Investigator 801-662-5400 ron.day@hsc.utah.edu
Primary Childrens Medical Center - Salt Lake City, Utah 2,093.2 miles Principal Investigator 801-662-5400 ron.day@hsc.utah.edu
Primary Childrens Medical Center - Salt Lake City, Utah 2,093.2 miles Principal Investigator 801-662-5400 ron.day@hsc.utah.edu
Phoenix Childrens Hospital - Phoenix, Arizona 2,294.8 miles Principal Investigator 602-933-1000 rrao@phoenixchildrens.com
Childrens Heart Center - Las Vegas, Nevada 2,374.9 miles Principal Investigator 702-732-1290 arothman@childrensheartcenter.com
Childrens Heart Center - Las Vegas, Nevada 2,374.9 miles Principal Investigator 702-732-1290 arothman@childrensheartcenter.com
Childrens Heart Center - Las Vegas, Nevada 2,374.9 miles Principal Investigator 702-732-1290 arothman@childrensheartcenter.com
Childrens Heart Center - Las Vegas, Nevada 2,374.9 miles Principal Investigator 702-732-1290 arothman@childrensheartcenter.com
UCLA Childrens Heart Center - Los Angeles, California 2,605.2 miles Principal Investigator 310-825-4321 jalejos@mednet.ucla.edu
UCLA Childrens Heart Center - Los Angeles, California 2,605.2 miles Principal Investigator 310-825-4321 jalejos@mednet.ucla.edu
UCLA Childrens Heart Center - Los Angeles, California 2,605.2 miles Principal Investigator 310-825-4321 jalejos@mednet.ucla.edu
UCLA Childrens Heart Center - Los Angeles, California 2,605.2 miles Principal Investigator 310-825-4321 jalejos@mednet.ucla.edu

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