A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension (SALTO)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04175600|
Recruitment Status : Recruiting
First Posted : November 25, 2019
Last Update Posted : April 14, 2021
|Condition or disease||Intervention/treatment||Phase|
|Hypertension, Pulmonary||Drug: Selexipag Drug: Placebo||Phase 3|
Expanded Access : Actelion has indicated that access to an investigational treatment associated with this study is available outside the clinical trial.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||237 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||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 <18 Years With Pulmonary Arterial Hypertension|
|Actual Study Start Date :||January 16, 2020|
|Estimated Primary Completion Date :||December 9, 2024|
|Estimated Study Completion Date :||January 10, 2028|
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.
Selexipag tablet will be administered orally.
Other Name: JNJ-67896049
Placebo Comparator: Placebo
Participants will receive matching placebo based on the body weight on Day 1 and will continue thereafter with twice daily dosing.
Matching placebo tablets will be administered orally.
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04175600
|Contact: Study Contact||844-434-4210||JNJ.CT@sylogent.com|
|Study Director:||Actelion Clinical Trial||Actelion|