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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
Date last updated
9/10/2024

Titles & IDs
Public title
A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension
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
Secondary ID [1] 0 0
2019-002817-21
Secondary ID [2] 0 0
CR108716
Universal Trial Number (UTN)
Trial acronym
SALTO
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypertension, Pulmonary 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Cardiovascular 0 0 0 0
Hypertension

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time to Disease Progression
Timepoint [1] 0 0
From randomization up to 7 days after study treatment discontinuation (up to 5 years)
Secondary outcome [1] 0 0
Percentage of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious AEs
Timepoint [1] 0 0
Up to 5 years
Secondary outcome [2] 0 0
Percentage of Participants with AEs Leading to Premature Discontinuation of Study Treatment
Timepoint [2] 0 0
Up to 5 years
Secondary outcome [3] 0 0
Change from Baseline in Systolic and Diastolic Arterial Blood Pressure
Timepoint [3] 0 0
Baseline up to end of treatment (EOT) (up to 8 years)
Secondary outcome [4] 0 0
Change from Baseline in Pulse Rate
Timepoint [4] 0 0
Baseline up to EOT (up to 8 years)
Secondary outcome [5] 0 0
Change from Baseline in Body Weight
Timepoint [5] 0 0
Baseline up to EOT (up to 8 years)
Secondary outcome [6] 0 0
Change from Baseline in Height
Timepoint [6] 0 0
Baseline up to EOT (up to 8 years)
Secondary outcome [7] 0 0
Sexual Maturation (Tanner Stage) Change from Baseline to all Assessed Time Points
Timepoint [7] 0 0
Up to 3 days after study treatment discontinuation (up to EOT) (multiple timepoints up to 8 years)
Secondary outcome [8] 0 0
Percentage of Participants with Treatment-emergent Electrocardiogram Abnormalities
Timepoint [8] 0 0
Baseline up to EOT (up to 8 years)
Secondary outcome [9] 0 0
Percentage of Participants with Treatment-emergent Marked Laboratory Abnormalities
Timepoint [9] 0 0
Baseline up to EOT (up to 8 years)
Secondary outcome [10] 0 0
Treatment-emergent Change from Baseline in Thyroid Stimulating Hormone
Timepoint [10] 0 0
Baseline up to EOT (up to 8 years)
Secondary outcome [11] 0 0
Time to First Clinical Event Committee (CEC)-confirmed Hospitalization or Death for PAH
Timepoint [11] 0 0
Until 7 days after study treatment discontinuation (Up to 8 years)
Secondary outcome [12] 0 0
Trough Plasma Concentration at Steady-state (Ctrough,ss) of Selexipag and its Metabolite ACT-333679
Timepoint [12] 0 0
Weeks 16, 24 and every 12 weeks thereafter (up to 8 years)
Secondary outcome [13] 0 0
Change from Baseline at Week 24 in Log2 N-terminal Pro-brain Natriuretic Peptide (NT-proBNP)
Timepoint [13] 0 0
Baseline up to Week 24

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
Minimum age
2 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/01/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
10/01/2028
Actual
Sample size
Target
Accrual to date
Final
138
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Queensland CHILDREN'S HOSPITAL - South Brisbane
Recruitment postcode(s) [1] 0 0
4101 - South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
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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
State/province [5] 0 0
Florida
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United States of America
State/province [6] 0 0
Indiana
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United States of America
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Michigan
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United States of America
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Ohio
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United States of America
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Pennsylvania
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Texas
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Utah
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United States of America
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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
State/province [21] 0 0
Porto Alegre
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Brazil
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Sao Paulo
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Bulgaria
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Sofia
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Canada
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Alberta
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Canada
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Ontario
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China
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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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France
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Paris
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France
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Pessac
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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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Hungary
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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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Bologna
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Padova
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Roma
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S. Donato Milanese
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Torino
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Seoul
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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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Guadalajara
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Mexico
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Mexico
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Mexico
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Gdansk
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Kraków
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Warszawa
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Wroclaw
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Porto
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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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A Coruña
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Barcelona
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Madrid
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Sevilla
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Sweden
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Lund
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Lausanne
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Kaohsiung
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Tainan
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Taipei
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Thailand
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Chiang Mai
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Songkhla
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Turkey
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Adana
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Ankara
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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
State/province [97] 0 0
Ho Chi Minh

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Actelion
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT04175600
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Actelion Clinical Trial
Address 0 0
Actelion
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04175600