A Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension (PAH)
Study Purpose
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).
Recruitment Criteria
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Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms |
No |
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Study Type
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies. |
Interventional |
| Eligible Ages | 1 Month - 17 Years |
| Gender | All |
Key
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)
Key
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.
Trial Details
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Trial ID:
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries. |
NCT02932410 |
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Phase
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use. |
Phase 3 |
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Lead Sponsor
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data. |
Actelion |
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Principal Investigator
The person who is responsible for the scientific and technical direction of the entire clinical study. |
N/A |
| Principal Investigator Affiliation | N/A |
|
Agency Class
Category of organization(s) involved as sponsor (and collaborator) supporting the trial. |
Industry |
| Overall Status | Recruiting |
| Countries | Australia, Austria, Brazil, Canada, China, Colombia, Finland, France, Hungary, Israel, Korea, Republic of, Malaysia, Mexico, Philippines, Poland, Portugal, Russian Federation, South Africa, Spain, Thailand, Ukraine, United States, Vietnam |
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Conditions
The disease, disorder, syndrome, illness, or injury that is being studied. |
Pulmonary Arterial Hypertension |
Arms
Experimental: Macitentan
Macitentan is administered once daily via oral route. Children less than (<) 2 years old (y.o.) will be assigned as a cohort to the macitentan group without randomization. The dose will be adjusted to the participant's age (for those < 2 y.o.) or to the participant's body weight (for those greater than or equal to (>=) 2 y.o.). single-arm extension period (SAEP) will start at end of core period (EOCP) visit and ends at end of study (EOS) visit.
Other: Standard-of-care
Standard-of-care as per site's clinical practice which may comprise treatment with pulmonary arterial hypertension (PAH) non-specific treatment and/or up to two PAH-specific medications excluding macitentan and intravenous/subcutaneous (IV/SC) prostanoids.
Interventions
Drug: - Macitentan
Dispersible tablet; Oral use
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 IV/SC prostanoids.
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.
Status
Completed
Address
Phoenix Childrens Hospital
Phoenix, Arizona, 85016
Status
Recruiting
Address
UCLA Children's Heart Center
Los Angeles, California, 90095
Status
Completed
Address
UCSF Medical Center
San Francisco, California, 94143-2202
Status
Recruiting
Address
Childrens Hospital Colorado
Aurora, Colorado, 80045
Status
Recruiting
Address
Children's National Medical Center
Washington, District of Columbia, 20010
Status
Recruiting
Address
Riley Hospital For Children
Indianapolis, Indiana, 46202
Status
Recruiting
Address
Boston Children's Hospital
Boston, Massachusetts, 02115
Status
Completed
Address
Detroit Medical Center
Detroit, Michigan, 48201
Status
Completed
Address
Mayo Clinic - PPDS
Rochester, Minnesota, 55905
Status
Recruiting
Address
Children's Heart Center
Las Vegas, Nevada, 89109
Status
Recruiting
Address
Montefiore Medical Center
Bronx, New York, 10467
Status
Recruiting
Address
Columbia University Medical Center - PIN
New York, New York, 10032
Status
Recruiting
Address
Duke University Medical Center
Durham, North Carolina, 27710
Status
Completed
Address
East Carolina University
Greenville, North Carolina, 27834
Status
Recruiting
Address
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Status
Completed
Address
University Hospitals Cleveland Medical Center, Rainbow Babies and Childrens Hospital
Cleveland, Ohio, 44106
Status
Completed
Address
Nationwide Children's Hospital
Columbus, Ohio, 43205
Status
Withdrawn
Address
Texas Children's Hospital
Houston, Texas, 77030
Status
Completed
Address
Primary Children's Medical Center
Salt Lake City, Utah, 84113
Status
Recruiting
Address
University of Virginia Health System
Charlottesville, Virginia, 19803
Status
Completed
Address
Childrens Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
International Sites
Status
Recruiting
Address
Royal Children's Hospital Melbourne - PIN
Parkville, , 3052
Status
Recruiting
Address
Lady Cilento Children's Hospital
South Brisbane, , 4101
Status
Completed
Address
Children's Hospital at Westmead
Westmead, , 2145
Status
Recruiting
Address
Medizinische Universität Graz
Graz, , 8036
Status
Completed
Address
Landes Frauen Und Kinderklinik Linz
Linz, , 4020
Status
Recruiting
Address
Medizinische Universitat Wien
Linz, , 4020
Status
Recruiting
Address
Irmandade Da Santa Casa de Misericórdia de São Paulo
São Paulo, , 01221-020
Status
Recruiting
Address
Sainte Justine Hospital
Montreal, , H3T 1C5
Status
Completed
Address
Stollery Children's Hospital
Toronto, , T6G 2B7
Status
Recruiting
Address
Beijing Anzhen Hospital of The Capital University of Medical Sciences
Beijing, , 100029
Status
Withdrawn
Address
Beijing Children's Hospital,Capital Medical University
Beijing, , 100037
Status
Withdrawn
Address
Fuwai Hospital
Beijing, , 100037
Status
Withdrawn
Address
The Children's Hospital, Zhejiang University school of Medicine
Hangzhou, , 310003
Status
Recruiting
Address
Qingdao Women and Children's Hospital
Qingdao, , 26600
Status
Recruiting
Address
Childrens Hospital of Shanghai
Shanghai, , 200062
Status
Recruiting
Address
Shanghai Children's Medical Center
Shanghai, , 200127
Status
Withdrawn
Address
General Hospital of Shenyang Military Region
Shenyang, , 110015
Status
Withdrawn
Address
Wuhan Asia Heart Hospital
Wuhan, , 430015
Status
Recruiting
Address
Fundacion Santa Fe de Bogota
Bogota, , 220246
Status
Recruiting
Address
Centro Medico Imbanaco de Cali SA
Cali, , 760042
Status
Completed
Address
HUS Uusi lastensairaala
Helsinki, , 00290
Status
Recruiting
Address
Hôpital de la Timone Enfants
Marseille, , 13385
Status
Recruiting
Address
Groupe Hospitalier Necker Enfants Malades
Paris cedex 15, , 75743
Status
Recruiting
Address
Hôpital Haut-Lévêque - Hôpital cardiologique
Pessac, , 33604
Status
Recruiting
Address
Hôpital Des Enfants
Toulouse Cedex 9, , 31059
Status
Recruiting
Address
Gottsegen Gyorgy Orszagos Kardiologiai Intezet
Budapest, , 1096
Status
Recruiting
Address
Rambam Medical Center - PPDS
Haifa, , 31096
Status
Recruiting
Address
Schneider Children's Medical Center of Israel - PIN
Petah-Tikva, , 49100
Status
Completed
Address
Chaim Sheba Medical Center
Ramat Gan, , 52621
Status
Recruiting
Address
Seoul National University Hospital
Seoul, , 03080
Status
Recruiting
Address
Severance Hospital Yonsei University Health System - PPDS
Seoul, , 03722
Status
Recruiting
Address
Institut Jantung Negara
Kuala Lumpur, , 50400
Status
Recruiting
Address
University Malaya Medical Centre
Kuala Lumpur, , 59100
Status
Recruiting
Address
Instituto Nacional de Pediatría
Ciudad De México, , 04530
Status
Recruiting
Address
Instituto Nacional de Cardiologia Dr. Ignacio Chavez
Ciudad De México, , 14080
Status
Recruiting
Address
CICUM San Miguel
Guadalajara, , 44160
Status
Recruiting
Address
Operadora de Hospitales Angeles SA de CV Hospital Angeles Lomas
Mexico, , 52763
Status
Recruiting
Address
Unidad de Investigación Clínica En Medicina SC
Monterrey, , 64718
Status
Completed
Address
Makati Medical Center
Makati City, , 1229
Status
Recruiting
Address
Philippine Heart Center
Quezon City, , 0850
Status
Recruiting
Address
Szpital Kliniczny im. Karola Jonschera Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
Poznań, , 60-572
Status
Recruiting
Address
Instytut Pomnik - Centrum Zdrowia Dziecka
Warszawa, , 04-730
Status
Recruiting
Address
Wojewodzki Szpital Specjalistyczny we Wroclawiu Osrodek Badawczo-Rozwojowy
Wroclaw, , 51-124
Status
Recruiting
Address
Centro Hospitalar de Lisboa Ocidental, EPE - Hospital de Santa Cruz
Carnaxide, , 2790-134
Status
Recruiting
Address
Centro Hospitalar E Universitário de Coimbra EPE
Coimbra, , 3000-075
Status
Recruiting
Address
Hospital Santa Marta
Lisboa, , 1169-024
Status
Recruiting
Address
Centro Hospitalar de Sao Joao EPE
Porto, , 4200-319
Status
Recruiting
Address
Research Institute of Complex Cardiovascular Pathology
Kemerovo, , 650002
Status
Completed
Address
GBUZ Children's Hospital named after Bashlyaeva Z.A. Moscow
Moscow, , 125408
Status
Recruiting
Address
Russian National Research Medical University n.a. N.I.Pirogov
Moscow, , 125412
Status
Recruiting
Address
Novosibirsk Research Institue of Blood Circulation Pathology n.a. E.N. Meshalkin
Novosibirsk, , 630055
Status
Completed
Address
Saint Petersburg State Pediatric Medical Academy
St. Petersburg, , 194100
Status
Recruiting
Address
Clinical Hospital №1
Tyumen, , 625023
Status
Completed
Address
Bashkiria State Medical University
Ufa, , 450000
Status
Completed
Address
University of The Free State
Bloemfontein, , 9300
Status
Completed
Address
Inkosi Albert Luthuli Central Hospital
Durban, , 4001
Status
Completed
Address
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, , 08035
Status
Completed
Address
C.H. Regional Reina Sofia
Cordoba, , 14001
Status
Completed
Address
Hospital General Universitario Gregorio Marañon
Madrid, , 28009
Status
Recruiting
Address
Hospital Universitario Ramon y Cajal
Madrid, , 28034
Status
Completed
Address
Hospital Universitario 12 de Octubre
Madrid, , 28041
Status
Completed
Address
Hospital Universitario La Paz
Madrid, , 28046
Status
Recruiting
Address
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, , 46026
Status
Withdrawn
Address
Ramathibodi Hospital Mahidol University
Bangkok, , 10400
Status
Withdrawn
Address
Siriraj Hospital Mahidol University
Bangkok, , 10700
Status
Recruiting
Address
Maharaj Nakorn Chiang Mai Chiang Mai University
Chiang Mai, , 50200
Status
Recruiting
Address
MI Dnipropetrovsk Specialized Clin. Med. Center of Mother and Child n.a. prof. M.F. Rudnev of DRC
Dnipro, , 49000
Status
Recruiting
Address
Municipal Institution of Health Care Regional Children's Clinical Hospital
Kharkiv, , 61093
Status
Recruiting
Address
MI Scientific Practical Medical Center for Children Cardiology and Cardiosurgery of MOH of Ukraine
Kyiv, , 04050
Status
Recruiting
Address
Hanoi Heart Hospital
Hanoi, , 100000
Status
Recruiting
Address
Hanoi Medical University Hospital
Hanoi, , 100000
Status
Recruiting
Address
Children's Hospital 1
Ho Chi Minh City, , 700000
Status
Recruiting
Address
Tam Duc Hospital
Ho Chi Minh, , 700000
