A Clinical Study of to Confirm the Doses of Selexipag in Children With Pulmonary Arterial Hypertension

Study Purpose

Selexipag is available for the treatment of pulmonary arterial hypertension (PAH) in adults in various countries. The efficacy of selexipag to delay disease progression was shown in a previous pivotal study conducted in 1156 adult patients with PAH. Given the similarities in the functional changes of PAH in children and adults, it is expected that children suffering from PAH could benefit from treatment with selexipag. The aim of the present study is to confirm the doses of selexipag to be used in pediatric patients with PAH older than 2 years. To fulfill this aim, blood levels of selexipag (pharmacokinetic assessments) as well as the safety, and tolerability of selexipag in children with PAH will be assessed.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
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 2 Years - 18 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - Signed and dated informed consent by the parent(s) or Legally authorized representative(s) AND assent from developmentally capable children.
  • - Males or females between ≥ 2 and < 18 years of age with weight ≥ 9 kg.
  • - PAH diagnosis confirmed by documented historical right heart catheterization (RHC) performed at any time before subject's enrollment - PAH with one of the following etiologies: - idiopathic (iPAH), - heritable (hPAH), - associated with congenital heart disease (CHD): PAH with co-incidental CHD; post-operative PAH (persisting/ recurring/ developing ≥ 6 months after repair of CHD) - Drug or toxin-induced - PAH associated with HIV - PAH associated with connective tissue disease - Word Health Organization functional class (WHO FC) II to III.
  • - Subjects treated with an endothelin receptor antagonist (ERA) and/or a phosphodiesterase type 5 (PDE-5) inhibitor provided that the treatment dose(s) has been stable for at least 3 months prior to enrollment, or patients who are not candidates for these therapies.
  • - Females of childbearing potential must have a negative pregnancy test at Screening and at Enrollment, and must agree to undertake monthly pregnancy tests, and to use a reliable method of contraception (if sexually active) from screening up to study drug discontinuation plus 30 days (EOS).
Key

Exclusion Criteria:

  • - Subjects with PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease (PVOD) and/or pulmonary capillary hemangiomatosis.
  • - Subjects with PAH associated with Eisenmenger syndrome.
  • - Subjects with moderate to large left-to-right shunts.
  • - Subjects with cyanotic congenital cardiac lesions such as transposition of the great arteries, truncus arteriosus, univentricular heart or pulmonary atresia with ventricular septal defect, as well as subjects with Fontan-palliation.
  • - Subjects with pulmonary hypertension due to lung disease.
  • - Previous treatment with Uptravi (selexipag) within 2 weeks prior to enrollment.
  • - Subjects having received prostacyclin (epoprostenol) or prostacyclin analogs (i.e., treprostinil, iloprost, beraprost) within 2 months prior to enrollment or are scheduled to receive any of these compounds during the trial.
  • - Treatment with another investigational drug within 4 weeks prior to enrollment.
  • - History, or current suspicion of intussusception or ileus or gastrointestinal obstruction as per investigator's judgment.
  • - Uncontrolled thyroid disease as per investigator judgment.
  • - Hemoglobin or hematocrit < 75% of the lower limit of normal range.
  • - Known severe or moderate hepatic impairment.
  • - Clinical signs of hypotension that in the investigator's judgment would preclude initiation of a PAH-specific therapy.
  • - Subjects with severe renal insufficiency.
  • - Known hypersensitivity to the investigational treatment or to any of the excipients of the drug formulations.
  • - History or clinical evidence of any disease and/or existence of any surgical or medical condition which might interfere with the absorption, distribution, metabolism or excretion of the study treatment(s) (e.g., cholecystectomy).
  • - Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol.

Trial Details

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.

NCT03492177
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 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Actelion
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Sofija Cerovic
Principal Investigator Affiliation Actelion
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Recruiting
Countries Belarus, France, Germany, Hungary, Israel, Malaysia, Poland, Russian Federation, Serbia, Taiwan, Ukraine, United Kingdom, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Pulmonary Arterial Hypertension
Additional Details

The selection of the starting dose for pediatric patients is based on the pharmacokinetic (PK) extrapolation from adults, taking into account the children body weight category, in order to lead to an exposure similar to that in adult PAH patients at a starting dose of 200 microgram (μg). As in adults, selexipag will be up-titrated to the individual maximum tolerated dose (iMTD) during the first 12 weeks. Approximately 55 subjects will be enrolled in 3 different age cohorts to obtain at least 40 subjects with evaluable PK profiles: Cohort 1: ≥ 12 to < 18 years of age, Cohort 2: ≥ 6 to < 12 years of age, Cohort 3: ≥ 2 to < 6 years of age. In each age cohort the starting dose will depend on the body weight. Enrollment will start with both Cohort 1 and Cohort 2. After completion of PK assessments in at least 15 subjects from Cohort 1 at Week 12, a first interim analysis will be conducted to establish the dose-exposure relationship using a population PK model. Results of this model-based analysis will be used to confirm or adjust the selexipag doses initially selected. Enrollment of Cohort 3 (children ≥ 2 to < 6 years of age) will start once the appropriate doses have been confirmed in a second interim analysis of PK data from Cohorts 1 and 2, and if there is no safety concern based on review by an Independent Data Monitoring Committee (IDMC).

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.

SEATTLE CHILDREN'S HOSP (Seattle), Seattle, Washington

Status

Recruiting

Address

SEATTLE CHILDREN'S HOSP (Seattle)

Seattle, Washington, 98145-5005

Site Contact

Principal Investigator

delphine.yung@seattlechildrens.org

206-987-2000

Vanderbilt University Medical Center, Nashville, Tennessee

Status

Recruiting

Address

Vanderbilt University Medical Center

Nashville, Tennessee, 37232

UNIV OF IOWA CHILDREN'S HOSP (Iowa City), Iowa City, Iowa

Status

Recruiting

Address

UNIV OF IOWA CHILDREN'S HOSP (Iowa City)

Iowa City, Iowa, 52248

Site Contact

Principal Investigator

abhay-divekar@uiowa.edu

319-356-8831

Denver, Colorado

Status

Recruiting

Address

Children'S Hospital Cardiac Care Center University Of Colorado

Denver, Colorado, 80218

Site Contact

Principal Investigator

dunbar.ivy@childrenscolorado.org

720-777-6820

International Sites

London, United Kingdom

Status

Recruiting

Address

Great Ormond Street Hospital PAEDIATRIC ITU (London)

London, , WC1N 3JH

Site Contact

Principal Investigator

Christine.Pierce@GOSH.nhs.uk

+44(0)2078138213

Zaporizhzhya, Ukraine

Status

Recruiting

Address

Municipal Institution Of The Zaporizhzhya Regional Council

Zaporizhzhya, , 69063

Site Contact

Principal Investigator

yureznichenko17@gmail.com

+30965562713

Lviv, Ukraine

Status

Recruiting

Address

LVIV REGIONAL CLINICAL HOSPITAL, CHILDRENS CARDIAC SURGERY (Lviv)

Lviv, , 79010

Site Contact

Principal Investigator

mklknk@gmail.com

+38 067 398 78 16

Kiev, Ukraine

Status

Recruiting

Address

State Institution Of The Ministry Of Health Of Ukraine

Kiev, , 1135

Site Contact

Principal Investigator

lillit@ukr.net

+380442065041

Dnipro, Ukraine

Status

Recruiting

Address

Municipal Enterprise Of The Dnipropetrovsk Regional Council

Dnipro, , 49070

Site Contact

Principal Investigator

o_shashko@ukr.net

+38 056 726 56 84

Taipei, Taiwan

Status

Recruiting

Address

NATIONAL TAIWAN UNIV HOSP-DEPT OF PAED (Taipei)

Taipei, , 100

National Cheng Kung University Hospital, Tainan, Taiwan

Status

Recruiting

Address

National Cheng Kung University Hospital

Tainan, , 704

Belgrade, Serbia

Status

Recruiting

Address

Institut Za Zdravstvenu Zaštitu Majke I Deteta Srbije "Dr Vukan Čupić"

Belgrade, , 11070

Site Contact

Principal Investigator

info@imd.org.rs

+381 11 3108 108 / 307

Univerzitetska Dečja Klinika, Belgrade, Serbia

Status

Recruiting

Address

Univerzitetska Dečja Klinika

Belgrade, , 11000

Site Contact

Principal Investigator

klinika@udk.bg.ac.yu

+381 11 2060 651

Tyumen Regional Hospital #1, Tyumen, Russian Federation

Status

Not yet recruiting

Address

Tyumen Regional Hospital #1

Tyumen, , 625023

Federal State Budgetary Institution, St.Petersburg, Russian Federation

Status

Recruiting

Address

Federal State Budgetary Institution

St.Petersburg, , 197341

St.Petersburg, Russian Federation

Status

Recruiting

Address

Saint Petersburg State Pediatric Medical University

St.Petersburg, , 194100

Samara, Russian Federation

Status

Recruiting

Address

Samara Regional Clinical Cardiological Dispensary

Samara, , 443070

Site Contact

Principal Investigator

duplyakov@yahoo.com

+78463737082

Moscow, Russian Federation

Status

Recruiting

Address

Moscow Scientific Research Institute For Pediatrics And Childrens Surgery Of Rosmedtechnologies

Moscow, , 125412

Site Contact

Principal Investigator

lirina2006@mail.ru

+7 495 483 71 01

Kemerovo, Russian Federation

Status

Recruiting

Address

Federal State Budget Scientific Institution

Kemerovo, , 650002

Site Contact

Principal Investigator

olb61@mail.ru

73842521474

Kazan, Russian Federation

Status

Recruiting

Address

KAZAN STATE MEDICAL UNIVERSITY PEDIATRIC SURGERY (Kazan)

Kazan, , 420059

Site Contact

Principal Investigator

nurmeev@gmail.com

8(843)2678444

Wrocław, Poland

Status

Recruiting

Address

Wojewódzki Szpital Specjalistyczny We Wrocławiu

Wrocław, , 51-124

Szpital Kliniczny im. Karola Jonschera, Poznan, Poland

Status

Recruiting

Address

Szpital Kliniczny im. Karola Jonschera

Poznan, , 60-572

Uniwersyteckie Centrum Kliniczne, Gdańsk, Poland

Status

Recruiting

Address

Uniwersyteckie Centrum Kliniczne

Gdańsk, , 80-952

Sarawak General Hospital, Kuching, Malaysia

Status

Recruiting

Address

Sarawak General Hospital

Kuching, , 93586

Site Contact

Principal Investigator

martinwnl@gmail.com

+60 82-668 111

INSTITUT JANTUNG NEGARA (Kuala Lumpur), Kuala Lumpur, Malaysia

Status

Recruiting

Address

INSTITUT JANTUNG NEGARA (Kuala Lumpur)

Kuala Lumpur, , 50400

Site Contact

Principal Investigator

geetha@ijn.com.my

+60 3 2617 8200

Ramat Gan, Israel

Status

Recruiting

Address

SHEBA MC_PEDIATRIC CARDIOLOGY DEPARTMENT (Ramat Gan)

Ramat Gan, , 52621

Site Contact

Principal Investigator

uriel.katz@sheba.health.gov.il

03-5303030

Petach Tikvah, Israel

Status

Recruiting

Address

SCHNEIDER CHILD MED CTR INST PEDIATRIC CARD (Petach Tikvah)

Petach Tikvah, , 49202

Site Contact

Principal Investigator

ebirk@clalit.org.il

+972 3 9253 715

Budapest, Hungary

Status

Recruiting

Address

Gottsegen Gyorgy National Card Inst - Pediatric Card

Budapest, , 1096

Site Contact

Principal Investigator

ablonczyl@kardio.hu

+36 1 215 1220

Universitätsklinikum Münster, Münster, Germany

Status

Recruiting

Address

Universitätsklinikum Münster

Münster, , 48149

Hamburg, Germany

Status

Recruiting

Address

Universitäres Herzzentrum Hamburg, Klinik für Kinderkardiologie

Hamburg, , 20246

Giessen, Germany

Status

Recruiting

Address

Justus-Liebig-Universität Gießen, Kinderherzzentrum

Giessen, , 35392

UNI FREIBURG, PED CARDIOLOGY (Freiburg), Freiburg, Germany

Status

Recruiting

Address

UNI FREIBURG, PED CARDIOLOGY (Freiburg)

Freiburg, , 70106

Site Contact

Principal Investigator

brigitte.stiller@uniklinik-freiburg.de

+49 (0) 761 27043230

Toulouse Cedex 9, France

Status

Recruiting

Address

HOSP DES ENFANTS_CARDIOLOGY DEPT (Toulouse Cedex 9)

Toulouse Cedex 9, , 31059

Site Contact

Principal Investigator

dulac.y@chu-toulouse.fr

+ 33 5 61 77 22 33

Paris, France

Status

Recruiting

Address

HOSP NECKER _CARDIOLOGY PEDIATRY DEPT (Paris)

Paris, , 75015

Site Contact

Principal Investigator

damien.bonnet@nck.aphp.fr

+33 1 44 49 43 44

Montpellier, France

Status

Recruiting

Address

HOSP ARNAUD DE VILLENEUVE - PAED DEPT (Montpellier)

Montpellier, , 34295

Site Contact

Principal Investigator

p-amedro@chu-montpellier.fr

+ 33 4 67 33 67 33

Minsk, Belarus

Status

Recruiting

Address

MINSK 4TH CITY CHILDREN'S CLINICAL HOSPITAL (Minsk)

Minsk, , 220118

Site Contact

Principal Investigator

chizhevskaya@mail.ru

+375 17 340-37-72

SI RSPC FOR PEDIATRIC SURGERY (Minsk), Minsk, Belarus

Status

Recruiting

Address

SI RSPC FOR PEDIATRIC SURGERY (Minsk)

Minsk, , 220013

Site Contact

Principal Investigator

director@dhc.by

+375 17 290-21-81

For more information, please contact PHA at Research@PHAssociation.org and refer to the terms of service below.

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