Clinical Trials List
Protocol NumberAC-065A310
NCT Number(ClinicalTrials.gov Identfier)NCT04175600
2020-04-01 - 2028-02-29
Phase III
Not yet recruiting1
Recruiting1
Terminated1
ICD-10I27.0
Primary pulmonary hypertension
ICD-9416.0
Primary pulmonary hypertension
A Randomized, Multicenter, Double-Blind, Placebo-Controlled, Parallel-Group, Event-Driven, Group-Sequential 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
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Trial Applicant
Johnson & Johnson
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Sponsor
Janssen Research & Development, LLC
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- MING-TAI LIN Division of Pediatrics
- 吳美環 Division of Pediatrics
- Hsao-Hsun Hsu Division of Thoracic Surgery
- Jou Kou Wang Division of Pediatrics
- 盧俊維 Division of Pediatrics
- 陳俊安 Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
The Actual Total Number of Participants Enrolled
0 Stop recruiting
Condition/Disease
Pulmonary Arterial Hypertension
Objectives
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.
Test Drug
Uptravi
Active Ingredient
Selexipag
Selexipag
Selexipag
Dosage Form
tablet
Dosage
50 mcg/tablet
200 mcg/tablet
200 mcg/tablet
Endpoints
Primary Outcome Measures :
Time to Disease Progression [ Time Frame: From randomization up to 7 days after study treatment discontinuation (up to 5 years) ]
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.
Secondary Outcome Measures :
Percentage of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious AEs [ Time Frame: Up to 5 years ]
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.
Percentage of Participants with AEs Leading to Premature Discontinuation of Study Treatment [ Time Frame: Up to 5 years ]
Percentage of participants with AEs leading to premature discontinuation of study treatment will be reported.
Change from Baseline in Systolic and Diastolic Arterial Blood Pressure [ Time Frame: Baseline up to end of treatment (EOT) (up to 8.3 years) ]
Change from baseline in systolic and diastolic arterial blood pressure to all assessed time points will be reported.
Change from Baseline in Pulse Rate [ Time Frame: Baseline up to EOT (up to 8.3 years) ]
Change from baseline in pulse rate to all assessed time points will be reported.
Change from Baseline in Body Weight [ Time Frame: Baseline up to EOT (up to 8.3 years) ]
Change from baseline in body weight to all assessed time points will be reported.
Change from Baseline in Height [ Time Frame: Baseline up to EOT (up to 8.3 years) ]
Change from baseline in height to all assessed time points will be reported.
Sexual Maturation (Tanner Stage) Change from Baseline to all Assessed Time Points [ Time Frame: Up to 3 days after study treatment discontinuation (up to EOT) (multiple timepoints up to 8.3 years) ]
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.
Percentage of Participants with Treatment-emergent Electrocardiogram Abnormalities [ Time Frame: Baseline up to EOT (up to 8.3 years) ]
Percentage of participants with treatment-emergent electrocardiogram abnormalities will be reported.
Percentage of Participants with Treatment-emergent Marked Laboratory Abnormalities [ Time Frame: Baseline up to EOT (up to 8.3 years) ]
Percentage of participants with treatment-emergent marked laboratory (serum chemistry [including pregnancy testing and thyroid markers] and hematology) abnormalities will be reported.
Treatment-emergent Change from Baseline in Thyroid Stimulating Hormone [ Time Frame: Baseline up to EOT (up to 8.3 years) ]
Treatment-emergent change from baseline in thyroid stimulating hormone over time will be reported.
Time to First Clinical Event Committee (CEC)-confirmed Hospitalization and Death for PAH [ Time Frame: Until 7 days after study treatment discontinuation (Up to 8.3 years) ]
Time to first CEC-confirmed hospitalization and 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.
Trough Plasma Concentration at Steady-state (Ctrough,ss) of Selexipag and its Metabolite ACT-333679 [ Time Frame: Weeks 16, 24 and every 12 weeks thereafter (up to 8.3 years) ]
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.
Time to Disease Progression [ Time Frame: From randomization up to 7 days after study treatment discontinuation (up to 5 years) ]
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.
Secondary Outcome Measures :
Percentage of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious AEs [ Time Frame: Up to 5 years ]
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.
Percentage of Participants with AEs Leading to Premature Discontinuation of Study Treatment [ Time Frame: Up to 5 years ]
Percentage of participants with AEs leading to premature discontinuation of study treatment will be reported.
Change from Baseline in Systolic and Diastolic Arterial Blood Pressure [ Time Frame: Baseline up to end of treatment (EOT) (up to 8.3 years) ]
Change from baseline in systolic and diastolic arterial blood pressure to all assessed time points will be reported.
Change from Baseline in Pulse Rate [ Time Frame: Baseline up to EOT (up to 8.3 years) ]
Change from baseline in pulse rate to all assessed time points will be reported.
Change from Baseline in Body Weight [ Time Frame: Baseline up to EOT (up to 8.3 years) ]
Change from baseline in body weight to all assessed time points will be reported.
Change from Baseline in Height [ Time Frame: Baseline up to EOT (up to 8.3 years) ]
Change from baseline in height to all assessed time points will be reported.
Sexual Maturation (Tanner Stage) Change from Baseline to all Assessed Time Points [ Time Frame: Up to 3 days after study treatment discontinuation (up to EOT) (multiple timepoints up to 8.3 years) ]
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.
Percentage of Participants with Treatment-emergent Electrocardiogram Abnormalities [ Time Frame: Baseline up to EOT (up to 8.3 years) ]
Percentage of participants with treatment-emergent electrocardiogram abnormalities will be reported.
Percentage of Participants with Treatment-emergent Marked Laboratory Abnormalities [ Time Frame: Baseline up to EOT (up to 8.3 years) ]
Percentage of participants with treatment-emergent marked laboratory (serum chemistry [including pregnancy testing and thyroid markers] and hematology) abnormalities will be reported.
Treatment-emergent Change from Baseline in Thyroid Stimulating Hormone [ Time Frame: Baseline up to EOT (up to 8.3 years) ]
Treatment-emergent change from baseline in thyroid stimulating hormone over time will be reported.
Time to First Clinical Event Committee (CEC)-confirmed Hospitalization and Death for PAH [ Time Frame: Until 7 days after study treatment discontinuation (Up to 8.3 years) ]
Time to first CEC-confirmed hospitalization and 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.
Trough Plasma Concentration at Steady-state (Ctrough,ss) of Selexipag and its Metabolite ACT-333679 [ Time Frame: Weeks 16, 24 and every 12 weeks thereafter (up to 8.3 years) ]
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.
Inclution Criteria
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 screening
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 screening
Exclusion Criteria
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
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
The Estimated Number of Participants
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Taiwan
6 participants
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Global
125 participants
