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

May 7, 2026 updated by: Actelion

A Prospective, Multicenter, Open Label, Single Arm, Phase 2 Study to Investigate the Safety, Tolerability and Pharmacokinetics of Selexipag in Children With Pulmonary Arterial Hypertension

The purpose of this study to confirm the selexipag starting dose(s), selected based on pharmacokinetic (PK) extrapolation from adults, that leads to similar exposure as adults doses in children from greater than or equal to (>=) 2 to less than (˂) 18 years of age with Pulmonary Arterial Hypertension (PAH), by investigating the PK of selexipag and its active metabolite ACT-333679 in this population.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

The selection of the starting dose for pediatric participants is based on the 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 participants at a starting dose of 200 micrograms (mcg). As in adults, selexipag will be up-titrated to the individual maximum tolerated dose (iMTD) during the first 12 weeks. Approximately 60 participants will be enrolled in 3 different age cohorts to obtain at least 45 participants 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 participants from Cohort 1 at Week 12, a first interim analysis will be conducted to establish the dose-exposure relationship using a population PK model. The PK data from any participants in Cohort 2 who have completed their PK assessments at this time will be included in this first interim analysis. 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).

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
63

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belarus
      • Minsk, Belarus, 220013
        • State Institution Republican Scientific And Practical Center For Pediatric Surgery
      • Minsk, Belarus, 220118
        • Health Institution 4Th City Children'S Clinical Hospital
  • Belgium
      • Ghent, Belgium, 9000
        • UZ Gent
  • Canada
    • Quebec
      • Montreal, Quebec, Canada, H3T 1C4
        • Centre Hospitalier Sainte Justine
  • China
      • Beijing, China, 100029
        • Beijing Anzhen Hospital
      • Shanghai, China, 200127
        • Shanghai Childrens Medical Center
  • France
      • Montpellier, France, 34295
        • CHU Arnaud de Villeneuve
      • Paris, France, 75015
        • Hôpital Necker - Enfants Malades
      • Toulouse, France, 31059
        • Chu Hopital Des Enfants
  • Germany
      • Freiburg im Breisgau, Germany, 70106
        • Universitätsklinikum Freiburg Zentrum
  • Hungary
      • Budapest, Hungary, 1096
        • Gottsegen Gyorgy Orszagos Kardiologiai Intezet, Felnott kardiologiai osztaly
  • Israel
      • Petach Tikvah, Israel
        • Schneider Children's Medical Center
      • Ramat Gan, Israel, 52621
        • Sheba Medical Center
  • Malaysia
      • Kota Samarahan, Malaysia, 94300
        • Sarawak Heart Center
      • Kuala Lumpur, Malaysia, 50400
        • Institut Jantung Negara (National Heart Institute)
  • Poland
      • Wroclaw, Poland, 51 124
        • Wojewodzki Szpital Specjalistyczny we Wroclawiu
  • Russia
      • Kazan', Russia, 420059
        • Kazan State Medical University
      • Kemerovo, Russia, 650002
        • Scientific and Research Institution of Cardiovascular Diseases Complex Problems
      • Moscow, Russia, 125412
        • Moscow Scientific Research Institute For Pediatrics And Childrens Surgery Of Rosmedtechnologies
      • Saint Petersburg, Russia, 194100
        • Saint Petersburg State Pediatric Medical University
      • Saint Petersburg, Russia, 197341
        • Almazov National Medical Research Center Of The Ministry Of Health Of The Russian Federation
      • Samara, Russia, 443070
        • Samara Regional Clinical Cardiological Dispensary
  • Serbia
      • Belgrade, Serbia, 11000
        • Univerzitetska Dečja Klinika
      • Belgrade, Serbia, 11070
        • Institut Za Zdravstvenu Zastitu Majke I Deteta Srbije Dr Vukan Cupic
  • Taiwan
      • Tainan, Taiwan, 704
        • National Cheng Kung University Hospital
      • Taipei, Taiwan, 100
        • National Taiwan University Hospital
  • Ukraine
      • Dnipro, Ukraine, 49070
        • Municipal Enterprise Of The Dnipropetrovsk Regional Council
      • Kiev, Ukraine, 04050
        • State Institution Of The Ministry Of Health Of Ukraine
      • Lviv, Ukraine, 79010
        • Lviv Regional Clinical Hospital
      • Zaporizhzhya, Ukraine, 69063
        • Municipal Institution Of The Zaporizhzhya Regional Council
  • United Kingdom
      • London, United Kingdom, WC1N 3JH
        • Great Ormond Street Hospital
  • United States
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children'S Hospital Cardiac Care Center University Of Colorado
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospital
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

2 years to 17 years (Child)

Accepts Healthy Volunteers

No

Description

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 greater than or equal to (>=) 2 and less than (<) 18 years of age with weight >= 9 kilograms (kg)
  • Pulmonary arterial hypertension (PAH) diagnosis confirmed by documented historical right heart catheterization (RHC) performed at any time before participant'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
  • Participants 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 participants 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:

  • Participants with PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease (PVOD) and/or pulmonary capillary hemangiomatosis
  • Participants with PAH associated with Eisenmenger syndrome
  • Participants with moderate to large left-to-right shunts
  • Participants 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 Participants with Fontan-palliation
  • Participants with pulmonary hypertension due to lung disease
  • Previous treatment with Uptravi (selexipag) within 2 weeks prior to enrollment
  • Participants having received prostacyclin (epoprostenol) or prostacyclin analogs (that is, 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 percentage (%) 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
  • Participants with severe renal insufficiency
  • Known hypersensitivity to the investigational treatment or to any of the excipients of the drug formulations

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: open label selexipag
The first dose of selexipag (Uptravi) will be administered in the evening of Day 1 and will be based on the body weight. Thereafter selexipag will be administered twice daily (morning and evening). Selexipag will be up-titrated during the first 12 weeks, with weekly increments equal to the starting dose until the participants reach their individual maximum tolerated dose (iMTD) or until a maximum dose corresponding to their baseline weight category is achieved (which will be 8-fold of the corresponding starting dose). Up-titration is followed by a stable maintenance treatment period from Week 12 to Week 16, at the maximum tolerated dose. Thereafter, participants will be treated with selexipag as long as the treatment is beneficial to the participants, as per investigator's decision.
Drug: selexipag (Uptravi)
Film-coated tablets for oral administration
Other Names:
  • JNJ-67896049
  • ACT-293987

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Area Under the Plasma Concentration-time Curve Over a Dose Interval at Steady State of Selexipag and Its Metabolite ACT-333679 Combined (AUCτ, ss, Combined)
Time Frame: Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
AUCτ, ss, combined was defined as the area under the plasma concentration-time curve over one dosing interval at steady state. AUCτ,ss,combined was calculated as 1/38 AUCτ,ss,selexipag plus 37/38 AUCτ,ss,ACT-333679.
Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Area Under the Plasma Concentration-time Curve Over a Dose Interval of Selexipag at Steady State (AUCτ,ss)
Time Frame: Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Area Under the Plasma Concentration-Time Curve Over a Dose Interval of ACT-333679 at Steady State (AUCτ,ss)
Time Frame: Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Maximum Observed Plasma Concentration of Selexipag at Steady State (Cmax,ss)
Time Frame: Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Maximum Observed Plasma Concentration of ACT-333679 at Steady State (Cmax,ss)
Time Frame: Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Time to Reach the Maximum Observed Plasma Concentration of Selexipag at Steady State (Tmax,ss)
Time Frame: Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Time to Reach the Maximum Observed Plasma Concentration of ACT-333679 at Steady State (Tmax,ss)
Time Frame: Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Trough Concentration of Selexipag at Steady State (Ctrough,ss)
Time Frame: Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Trough Concentration of ACT-333679 at Steady State (Ctrough,ss)
Time Frame: Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Week 1,Week 12: pre-dose, 1, 2, 4, 6, 8 and 12 h post-morning dose. Week 2, 4 and 6: pre-dose (Up to Week 12)
Number of Participants With Treatment-emergent Adverse Events (TEAEs) (End of Treatment [EOT] + 3 Days)
Time Frame: EOT+3 days (Up to Week 17)
EOT+3 days (Up to Week 17)
Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs) (EOT + 3 Days)
Time Frame: EOT+3 days (Up to Week 17)
EOT+3 days (Up to Week 17)
Number of Participants With Adverse Events (AEs) Leading to Permanent Discontinuation of Study Drug
Time Frame: Up to 7 years
Up to 7 years
Number of Participants With Treatment-emergent Deaths (EOT + 3 Days)
Time Frame: EOT+3 days (Up to Week 17)
EOT+3 days (Up to Week 17)
Number of Participants With Treatment-emergent Marked Laboratory Abnormalities (EOT + 3 Days)
Time Frame: EOT+3 days (Up to Week 17)
EOT+3 days (Up to Week 17)
Change From Baseline in Hematology Parameters (EOT + 3 Days)
Time Frame: EOT+3 days (Up to Week 17)
EOT+3 days (Up to Week 17)
Change From Baseline in Chemistry Parameters (EOT + 3 Days)
Time Frame: EOT+3 days (Up to Week 17)
EOT+3 days (Up to Week 17)
Number of Participants With Treatment-emergent Electrocardiogram (ECG) Abnormalities (EOT + 3 Days)
Time Frame: EOT+3 days (Up to Week 17)
EOT+3 days (Up to Week 17)
Change From Baseline in Thyroid Stimulating Hormone (TSH) up to EOT + 3 Days
Time Frame: EOT+3 days (Up to Week 17)
EOT+3 days (Up to Week 17)
Change From Baseline in Blood Pressure
Time Frame: Up to 7 years
Up to 7 years
Change From Baseline in Heart Rate
Time Frame: Up to 7 years
Up to 7 years
Change From Baseline Over Time in Height up to EOT+3 Days
Time Frame: EOT+3 days (Up to Week 17)
EOT+3 days (Up to Week 17)
Change From Baseline Over Time in Body Mass Index (BMI) up to EOT + 3 Days
Time Frame: EOT+ 3 days (Up to Week 17)
EOT+ 3 days (Up to Week 17)
Change From Baseline in Sexual Maturation (Tanner Stage) up to End of Treatment (EOT + 3 Days)
Time Frame: EOT+ 3 days (Up to Week 17)
EOT+ 3 days (Up to Week 17)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Actelion

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Catherine Boisson, Actelion

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 23, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 28, 2022

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 3, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 3, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 10, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 8, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 7, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AC-065A203 (Other Identifier: Janssen Research & Development, LLC)
  • 2022-503042-42-00 (Registry Identifier: EUCT number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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