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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
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
1/10/2027
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
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
District of Columbia
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Indiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
United States of America
State/province [11] 0 0
Utah
Country [12] 0 0
United States of America
State/province [12] 0 0
Virginia
Country [13] 0 0
Belarus
State/province [13] 0 0
Minsk
Country [14] 0 0
Belgium
State/province [14] 0 0
Brussels
Country [15] 0 0
Belgium
State/province [15] 0 0
Gent
Country [16] 0 0
Belgium
State/province [16] 0 0
Leuven
Country [17] 0 0
Brazil
State/province [17] 0 0
Blumenau
Country [18] 0 0
Brazil
State/province [18] 0 0
Brasilia
Country [19] 0 0
Brazil
State/province [19] 0 0
Curitiba
Country [20] 0 0
Brazil
State/province [20] 0 0
Fortaleza
Country [21] 0 0
Brazil
State/province [21] 0 0
Porto Alegre
Country [22] 0 0
Brazil
State/province [22] 0 0
Sao Paulo
Country [23] 0 0
Bulgaria
State/province [23] 0 0
Sofia
Country [24] 0 0
Canada
State/province [24] 0 0
Alberta
Country [25] 0 0
Canada
State/province [25] 0 0
Ontario
Country [26] 0 0
China
State/province [26] 0 0
Beijing
Country [27] 0 0
China
State/province [27] 0 0
Guangzhou
Country [28] 0 0
China
State/province [28] 0 0
Qingdao
Country [29] 0 0
China
State/province [29] 0 0
Shanghai
Country [30] 0 0
China
State/province [30] 0 0
Shenyang
Country [31] 0 0
Colombia
State/province [31] 0 0
Bogota
Country [32] 0 0
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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France
State/province [36] 0 0
Lille Cedex
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France
State/province [37] 0 0
Marseille Cedex 5
Country [38] 0 0
France
State/province [38] 0 0
Montpellier Cedex 5
Country [39] 0 0
France
State/province [39] 0 0
Paris
Country [40] 0 0
France
State/province [40] 0 0
Pessac
Country [41] 0 0
France
State/province [41] 0 0
Toulouse Cedex 9
Country [42] 0 0
Germany
State/province [42] 0 0
Freiburg
Country [43] 0 0
Germany
State/province [43] 0 0
Heidelberg
Country [44] 0 0
Germany
State/province [44] 0 0
Leipzig
Country [45] 0 0
Germany
State/province [45] 0 0
München
Country [46] 0 0
Hungary
State/province [46] 0 0
Budapest
Country [47] 0 0
Ireland
State/province [47] 0 0
Dublin
Country [48] 0 0
Israel
State/province [48] 0 0
Haifa
Country [49] 0 0
Israel
State/province [49] 0 0
Ramat Gan
Country [50] 0 0
Italy
State/province [50] 0 0
Bologna
Country [51] 0 0
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
State/province [54] 0 0
S. Donato Milanese
Country [55] 0 0
Italy
State/province [55] 0 0
Torino
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Korea, Republic of
State/province [56] 0 0
Seoul
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Korea, Republic of
State/province [57] 0 0
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
State/province [60] 0 0
Guadalajara
Country [61] 0 0
Mexico
State/province [61] 0 0
Mexico
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Mexico
State/province [62] 0 0
Monterrey
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Poland
State/province [63] 0 0
Gdansk
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Poland
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Kraków
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Poland
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Poznan
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Poland
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Warszawa
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Poland
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Wroclaw
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Poland
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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
State/province [84] 0 0
Kaohsiung
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Taiwan
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Tainan
Country [86] 0 0
Taiwan
State/province [86] 0 0
Taipei
Country [87] 0 0
Thailand
State/province [87] 0 0
Chiang Mai
Country [88] 0 0
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
Country [94] 0 0
Ukraine
State/province [94] 0 0
Kyiv
Country [95] 0 0
Ukraine
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Zaporizhzhya
Country [96] 0 0
Vietnam
State/province [96] 0 0
Hanoi
Country [97] 0 0
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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.janssen.com/clinical-trials/transparency


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