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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04175600
Registration number
NCT04175600
Ethics application status
Date submitted
8/11/2019
Date registered
25/11/2019
Titles & IDs
Public title
A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension
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Scientific title
A Randomized, Multicenter, Double-Blind, Placebo-Controlled, Parallel-Group Study With Open-Label Extension Period to Assess the Efficacy and Safety of Selexipag as Add-On Treatment to Standard of Care in Children Aged >=2 to <18 Years With Pulmonary Arterial Hypertension
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Secondary ID [1]
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2019-002817-21
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Secondary ID [2]
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CR108716
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Universal Trial Number (UTN)
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Trial acronym
SALTO
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hypertension, Pulmonary
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Condition category
Condition code
Respiratory
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Other respiratory disorders / diseases
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Cardiovascular
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0
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Hypertension
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Selexipag
Treatment: Drugs - Placebo
Experimental: Selexipag - Participants will receive selexipag based on the body weight on Day 1 and will continue thereafter with twice daily dosing. Selexipag will be uptitrated during the first 12 weeks until the participants reaches the individual maximum tolerated dose (iMTD) or until a maximum dose corresponding to their baseline body-weight category is achieved. Uptitration is followed by a maintenance period after Week 12 until end of treatment (EOT), at the maximum tolerated dose.
Placebo comparator: Placebo - Participants will receive matching placebo based on the body weight on Day 1 and will continue thereafter with twice daily dosing.
Treatment: Drugs: Selexipag
Selexipag tablet will be administered orally.
Treatment: Drugs: Placebo
Matching placebo tablets will be administered orally.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Time to Disease Progression
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Assessment method [1]
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Time to disease progression is the time from randomization up to 7 days after study treatment discontinuation. Disease progression is defined as the first occurrence of either of the following components: Death (all causes), Atrial septostomy or Potts' anastomosis, or registration on lung transplant list, Hospitalization due to worsening pulmonary arterial hypertension (PAH), Clinical worsening of PAH.
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Timepoint [1]
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From randomization up to 7 days after study treatment discontinuation (up to 5 years)
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Secondary outcome [1]
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Percentage of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious AEs
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Assessment method [1]
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An adverse event (AE) is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. TEAEs are AEs with onset during the intervention period or that are a consequence of a pre-existing condition that has worsened since baseline. A SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
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Timepoint [1]
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Up to 5 years
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Secondary outcome [2]
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Percentage of Participants with AEs Leading to Premature Discontinuation of Study Treatment
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Assessment method [2]
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Percentage of participants with AEs leading to premature discontinuation of study treatment will be reported.
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Timepoint [2]
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Up to 5 years
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Secondary outcome [3]
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Change from Baseline in Systolic and Diastolic Arterial Blood Pressure
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Assessment method [3]
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Change from baseline in systolic and diastolic arterial blood pressure to all assessed time points will be reported.
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Timepoint [3]
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Baseline up to end of treatment (EOT) (up to 8 years)
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Secondary outcome [4]
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Change from Baseline in Pulse Rate
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Assessment method [4]
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Change from baseline in pulse rate to all assessed time points will be reported.
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Timepoint [4]
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Baseline up to EOT (up to 8 years)
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Secondary outcome [5]
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Change from Baseline in Body Weight
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Assessment method [5]
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Change from baseline in body weight to all assessed time points will be reported.
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Timepoint [5]
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Baseline up to EOT (up to 8 years)
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Secondary outcome [6]
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Change from Baseline in Height
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Assessment method [6]
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Change from baseline in height to all assessed time points will be reported.
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Timepoint [6]
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Baseline up to EOT (up to 8 years)
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Secondary outcome [7]
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Sexual Maturation (Tanner Stage) Change from Baseline to all Assessed Time Points
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Assessment method [7]
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The sexual maturation change as per Tanner stage will be assessed from baseline to all assessed time points. Tanner stage I is defined as no pubic hair at all (prepubertal Dominic state); stage II is defined as a small amount of long, downy hair with slight pigmentation at the base of the penis and scrotum (males) or on the labia majora (females); stage III is defined as when the hair becomes more coarse and curly, and begins to extend laterally; stage IV is defined as adult-like hair quality, extending across pubis but sparing medial thighs; and stage V is defined as when the: hair extends to medial surface of the thighs.
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Timepoint [7]
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Up to 3 days after study treatment discontinuation (up to EOT) (multiple timepoints up to 8 years)
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Secondary outcome [8]
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Percentage of Participants with Treatment-emergent Electrocardiogram Abnormalities
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Assessment method [8]
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Percentage of participants with treatment-emergent electrocardiogram abnormalities will be reported.
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Timepoint [8]
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Baseline up to EOT (up to 8 years)
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Secondary outcome [9]
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Percentage of Participants with Treatment-emergent Marked Laboratory Abnormalities
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Assessment method [9]
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Percentage of participants with treatment-emergent marked laboratory (serum chemistry \[including pregnancy testing and thyroid markers\] and hematology) abnormalities will be reported.
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Timepoint [9]
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Baseline up to EOT (up to 8 years)
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Secondary outcome [10]
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Treatment-emergent Change from Baseline in Thyroid Stimulating Hormone
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Assessment method [10]
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Treatment-emergent change from baseline in thyroid stimulating hormone over time will be reported.
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Timepoint [10]
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Baseline up to EOT (up to 8 years)
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Secondary outcome [11]
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Time to First Clinical Event Committee (CEC)-confirmed Hospitalization or Death for PAH
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Assessment method [11]
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Time to first CEC-confirmed hospitalization or death for PAH is the time (days) from randomization to first occurrence of CEC-confirmed hospitalization for PAH or death due to PAH up to 7 days after study intervention discontinuation.
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Timepoint [11]
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Until 7 days after study treatment discontinuation (Up to 8 years)
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Secondary outcome [12]
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Trough Plasma Concentration at Steady-state (Ctrough,ss) of Selexipag and its Metabolite ACT-333679
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Assessment method [12]
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Ctrough,ss is defined as the plasma concentration just prior to the morning dose, with the last study intervention administration one day prior to the pharmacokinetic sampling and will be reported for Selexipag and its metabolite ACT-333679.
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Timepoint [12]
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Weeks 16, 24 and every 12 weeks thereafter (up to 8 years)
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Secondary outcome [13]
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Change from Baseline at Week 24 in Log2 N-terminal Pro-brain Natriuretic Peptide (NT-proBNP)
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Assessment method [13]
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The change from baseline at week 24 in log2 NT-proBNP will be reported.
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Timepoint [13]
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Baseline up to Week 24
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Eligibility
Key inclusion criteria
* Participants between greater than or equal to (>=) 2 and less than (<) 18 years of age weighing >=9 kilogram (kg) at randomization
* Pulmonary arterial hypertension (PAH) diagnosis confirmed by documented historical right heart catheterization (RHC) performed at any time before participant's screening
* PAH (World Health Organization [WHO] Group 1), including participants with Down syndrome, of the following etiologies: Idiopathic PAH (IPAH); Heritable PAH (HPAH); PAH associated with congenital heart disease (PAH-associated with congenital heart disease [aCHD]) (PAH with coincidental CHD [that is, a small atrial septal defect, ventricular septal defect, or patent ductus arteriosus that does not itself account for the development of elevated PVR] and if approved by the BCAC) and Post-operative PAH (persisting / recurring/ developing >=6 months after repair of CHD); Drug or toxin-induced; PAH associated with Human immunodeficiency virus (HIV)
* WHO functional class (FC) II and III
* Participants treated with at least 1 PAH-specific treatment, example, an Endothelin receptor antagonist (ERA) and/or a Phosphodiesterase type-5 (PDE-5) inhibitor/soluble guanylate cyclase stimulator, provided that the treatment dose(s) has been stable for at least 3 months prior to first dose of study intervention
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Minimum age
2
Years
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Maximum age
17
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease, and/or pulmonary capillary hemangiomatosis
* PAH associated with Eisenmenger syndrome
* Previous exposure to Uptravi (selexipag)
* Known concomitant life-threatening disease with a life expectancy <12 months
* Pregnant, planning to become pregnant, or lactating
* Known allergies, hypersensitivity, or intolerance to selexipag or its excipients
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
16/01/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
10/01/2028
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Actual
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Sample size
Target
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Accrual to date
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Final
138
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Queensland CHILDREN'S HOSPITAL - South Brisbane
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Recruitment postcode(s) [1]
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4101 - South Brisbane
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Recruitment outside Australia
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Arizona
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California
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Colorado
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United States of America
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District of Columbia
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United States of America
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Florida
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United States of America
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Indiana
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Michigan
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Ohio
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Utah
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Virginia
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Belarus
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Minsk
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Belgium
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Brussels
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Belgium
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Gent
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Belgium
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Leuven
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Brazil
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Blumenau
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Brazil
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Brasilia
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Brazil
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Curitiba
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Brazil
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Fortaleza
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Brazil
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Porto Alegre
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Brazil
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Sao Paulo
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Sofia
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Alberta
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Canada
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Ontario
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Beijing
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China
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Guangzhou
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China
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Qingdao
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China
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Shanghai
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China
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Shenyang
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Colombia
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Bogota
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Colombia
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Cali
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Colombia
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Piedecuesta
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Colombia
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Soledad
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Finland
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Helsinki
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Lille Cedex
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France
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France
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Montpellier Cedex 5
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France
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Paris
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France
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Pessac
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France
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Toulouse Cedex 9
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Germany
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Freiburg
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Germany
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Heidelberg
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Germany
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Leipzig
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Germany
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München
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Dublin
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Israel
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Haifa
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Israel
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Ramat Gan
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Italy
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Bologna
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Italy
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Milano
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Italy
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Padova
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Italy
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Roma
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Italy
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S. Donato Milanese
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Italy
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Torino
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Korea, Republic of
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Seoul
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Korea, Republic of
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Yangsan-si
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Lithuania
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Vilnius
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Malaysia
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Kuala Lumpur
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Mexico
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Guadalajara
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Mexico
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Mexico
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Mexico
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Monterrey
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Poland
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Gdansk
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Poland
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Kraków
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Poznan
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Warszawa
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Wroclaw
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Zabrze
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Portugal
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Lisboa
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Portugal
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Porto
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Russian Federation
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Kazan
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Russian Federation
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Kemerovo
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Russian Federation
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Moscow
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Russian Federation
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Samara
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Serbia
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Belgrade
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Spain
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A Coruña
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Spain
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Barcelona
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Spain
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Esplugues de Llobregat
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Spain
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Madrid
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Spain
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Sevilla
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Sweden
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Gothenburg
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Sweden
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Lund
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Switzerland
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Lausanne
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Taiwan
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Kaohsiung
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Taiwan
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Tainan
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Taiwan
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Taipei
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Thailand
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Chiang Mai
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Thailand
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Songkhla
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Turkey
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Adana
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Turkey
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Ankara
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Turkey
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Istanbul
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Turkey
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Izmir
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Ukraine
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Dnipro
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Ukraine
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Kyiv
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Ukraine
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Zaporizhzhya
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Vietnam
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Hanoi
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Vietnam
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Ho Chi Minh
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Actelion
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of this study is to evaluate whether the addition of selexipag to standard of care treatment delays disease progression in children with Pulmonary Arterial Hypertension (PAH) in comparison to placebo.
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Trial website
https://clinicaltrials.gov/study/NCT04175600
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Actelion Clinical Trial
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Address
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Actelion
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Phone
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Fax
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Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
The data sharing policy of the Janssen Pharmaceutical Companies of Johnson \& Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale open Data Access (YODA) Project site at yoda.yale.edu
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://www.janssen.com/clinical-trials/transparency
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT04175600