Selexipag for Pulmonary Hypertension

Kaohsiung Veterans General Hospital, Kaohsiung, Taiwan
Pulmonary HypertensionSelexipag - Drug
2 - 17
All Sexes
What conditions do you have?

Study Summary

This trial is testing if a new drug can help kids with Pulmonary Arterial Hypertension by delaying disease progression.

Eligible Conditions
  • Pulmonary Hypertension

Treatment Effectiveness

Phase-Based Effectiveness

2 of 3
Phase 3
This is further along than 85% of similar trials

Study Objectives

1 Primary · 14 Secondary · Reporting Duration: Weeks 16, 24 and every 12 weeks thereafter (up to 8 years)

Year 8
Change from Baseline in Body Weight
Change from Baseline in Height
Change from Baseline in Pulse Rate
Percentage of Participants with Treatment-emergent Electrocardiogram Abnormalities
Percentage of Participants with Treatment-emergent Marked Laboratory Abnormalities
Week 24
Change from Baseline at Week 24 in Log2 N-terminal Pro-brain Natriuretic Peptide (NT-proBNP)
Year 8
Change from Baseline in Systolic and Diastolic Arterial Blood Pressure
Year 5
Time to Disease Progression
Year 8
Time to First Clinical Event Committee (CEC)-confirmed Hospitalization and Death for PAH
Time to First Clinical Event Committee (CEC)-confirmed Hospitalization or Death for PAH
Year 8
Sexual Maturation (Tanner Stage) Change from Baseline to all Assessed Time Points
Up to 5 years
Percentage of Participants with AEs Leading to Premature Discontinuation of Study Treatment
Percentage of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious AEs
Year 8
Trough Plasma Concentration at Steady-state (Ctrough,ss) of Selexipag and its Metabolite ACT-333679

Trial Safety

Phase-Based Safety

3 of 3
This is further along than 85% of similar trials

Side Effects for

38%Pain in Jaw
21%Pain in Extremity
11%Upper Respiratory Tract Infection
9%Nasal Congestion
8%Decreased Appetite
8%Back Pain
6%Oedema Peripheral
4%Oropharyngeal Pain
4%Non-Cardiac Chest Pain
4%Abdominal Pain Upper
4%Lower Respiratory Tract Infection
2%Respiratory Tract Infection
2%Right Ventricular Failure
2%Abdominal Distension
2%Atrial Flutter
This histogram enumerates side effects from a completed 2020 Phase 4 trial (NCT03078907) in the Selexipag ARM group. Side effects include: Headache with 77%, Diarrhoea with 53%, Nausea with 42%, Pain in Jaw with 38%, Vomiting with 25%.

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.

Trial Design

2 Treatment Groups

1 of 2
1 of 2

Experimental Treatment

Non-Treatment Group

125 Total Participants · 2 Treatment Groups

Primary Treatment: Selexipag · Has Placebo Group · Phase 3

Experimental Group · 1 Intervention: Selexipag · Intervention Types: Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug
First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: weeks 16, 24 and every 12 weeks thereafter (up to 8 years)

Who is running the clinical trial?

ActelionLead Sponsor
190 Previous Clinical Trials
35,717 Total Patients Enrolled
Actelion Clinical TrialStudy DirectorActelion
12 Previous Clinical Trials
1,001 Total Patients Enrolled

Eligibility Criteria

Age 2 - 17 · All Participants · 0 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:

Frequently Asked Questions

Would this experimental therapy be appropriate for patients above the age of 50?

"This particular clinical trial is for patients aged 2 to 17. Out of the 61 clinical trials conducted for people under 18, this is one of them. On the other hand, there are 710 clinical trials for people over 65." - Anonymous Online Contributor

Unverified Answer

What sets this experiment apart from others like it?

"Selexipag has had a relatively short journey in clinical research, with the first study being published in 2018. Actelion was the primary sponsor for this 63-person Phase 1 trial. Due to the success of this initial testing Selexipag received approval for Phase 2 trials. Currently, 5 separate studies are ongoing and recruiting patients from 45 different cities across 43 countries." - Anonymous Online Contributor

Unverified Answer

In how many different medical clinics is this research project being carried out today?

"In addition to other medical facilities, this trial is taking place at Children's National Medical Center in Washington D.C., Texas Children's Hospital in Houston, Texas, and Congenital Heart Center of the University of Florida in Gainesville, Florida." - Anonymous Online Contributor

Unverified Answer

Are patients being accepted for enrollment in this trial at this time?

"That is accurate. The information available on reveals that this study, which was first publicized on 16th January 2020, is presently looking for participants. There are 237 spots open at 16 different hospitals or clinics." - Anonymous Online Contributor

Unverified Answer

How can I sign up for the research project?

"This clinical trial is looking for 237 participants, between the ages of 2 and 17 who currently have hypertension, pulmonary. It is required that participants also meet the following 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 PA" - Anonymous Online Contributor

Unverified Answer

What other research has been done on Selexipag in the past?

"Originally, selexipag was researched in 2018 at UZ Gent. Out of the 18302 completed clinical trials, 5 are ongoing as of today. Most of these live studies are situated in Washington D.C." - Anonymous Online Contributor

Unverified Answer

Are there any harmful side effects associated with Selexipag?

"Selexipag has received a score of 3 for safety. This is due to the fact that it is a Phase 3 trial, which suggests that not only does some data support efficacy, but multiple rounds also support safety." - Anonymous Online Contributor

Unverified Answer

How many people have been chosen to participate in this experiment?

"In order to carry out this research, the study sponsor, Actelion, needs 237 patients that fulfill the inclusion criteria. The trial will take place in various locations including Children's National Medical Center (Washington D.C.) and Texas Children's Hospital (Houston)." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.