Study on Selexipag and Bosentan for Children with Pulmonary Arterial Hypertension

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What is this study about?

This clinical trial is focused on studying Pulmonary Arterial Hypertension (PAH), a condition where the blood pressure in the arteries of the lungs is higher than normal. The study is testing a medication called Selexipag, which is being used as an additional treatment to the standard care for children aged 2 to under 18 years who have PAH. The purpose of the study is to see if adding Selexipag can help delay the progression of the disease compared to a placebo.

Participants in the study will receive either Selexipag or a placebo, in addition to their usual treatment for PAH. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication and who is receiving the placebo. This helps ensure that the results are not biased. The study will also include an open-label extension period, where all participants may receive Selexipag after the initial phase. The treatment will be given in the form of film-coated tablets, taken orally.

The study will last for a period of up to 96 weeks, during which the participants’ health and response to the treatment will be closely monitored. The main goal is to determine how long it takes for the disease to progress in participants taking Selexipag compared to those taking a placebo. This information will help researchers understand the effectiveness and safety of Selexipag as an add-on treatment for children with PAH.

1 joining the study

Upon joining the study, the parent or legal guardian must sign an informed consent form indicating understanding of the study’s purpose and procedures. If the child is capable of understanding, typically at age 7 or older, their assent is also required.

2 randomization

Participants are randomly assigned to receive either the study medication, selexipag, or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

3 medication administration

Participants will take the study medication, selexipag, in the form of a film-coated tablet. The medication is taken orally. The dosage and frequency will be determined by the study protocol and communicated to the participant by the study team.

4 treatment period

The treatment period involves regular administration of the study medication as per the prescribed dosage and frequency. Participants will continue their standard care treatment alongside the study medication.

5 monitoring and assessments

Throughout the study, participants will undergo regular monitoring and assessments to evaluate the efficacy and safety of the treatment. This includes physical examinations, laboratory tests, and other necessary evaluations as determined by the study protocol.

6 open-label extension

After the initial study period, participants may enter an open-label extension phase where all participants receive selexipag. This phase allows further assessment of the medication’s long-term effects.

7 end of study

The study concludes with a final assessment to determine the overall impact of the treatment. Participants will be informed about the study’s findings and any further steps if necessary.

Who Can Join the Study?

Parent(s) or legal guardians must sign a form to show they understand the study and agree for their child to participate. Children who can understand the study (usually 7 years and older) must also agree to participate.
The child must be between the ages of 2 and 18 years old and weigh at least 9 kg (about 20 pounds).
The child must have a confirmed diagnosis of Pulmonary Arterial Hypertension (PAH), which is a type of high blood pressure affecting the lungs and heart. This diagnosis must be confirmed by a past medical test called Right Heart Catheterization (RHC).
The child must have PAH that falls under specific categories, including:
- Idiopathic PAH (IPAH): PAH with no known cause.
- Heritable PAH (HPAH): PAH that runs in families.
- PAH associated with congenital heart disease (PAH-aCHD): PAH related to heart defects present at birth.
- PAH caused by drugs or toxins.
- PAH associated with HIV.
The child must be in World Health Organization Functional Class (WHO FC) II or III, which describes the severity of their symptoms.
The child must be receiving at least one specific treatment for PAH, such as an endothelin receptor antagonist (ERA) or a PDE-5 inhibitor, and the treatment dose must have been stable for at least 3 months before starting the study.
If the child is a female who can become pregnant, she must have a negative pregnancy test before starting the study and agree to regular pregnancy tests during the study. She must also agree to use effective birth control if she is sexually active.

Who Cannot Join the Study?

Patients who have a different condition than Pulmonary Arterial Hypertension cannot participate.
Patients who are not within the specified age range for the study cannot participate.
Patients who are not able to follow the study procedures or take the study medication cannot participate.
Patients who have other serious health conditions that might interfere with the study cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who are currently participating in another clinical trial cannot participate.
Patients who have had a recent surgery or are planning to have surgery during the study cannot participate.
Patients who have allergies to the study medication or its ingredients cannot participate.
Patients who have a history of drug or alcohol abuse cannot participate.
Patients who have been diagnosed with a mental health condition that might interfere with the study cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Centro Hospitalar Universitario Lisboa Central E.P.E.	Lisbon	Portugal
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany
Centro Hospitalar Universitario Sao Joao E.P.E.	Porto	Portugal
Katholieke Universiteit te Leuven	Leuven	Belgium
Université Libre de Bruxelles – Hôpital Erasme	Brussels	Belgium

Other Sites

Site Name	City	Country
Slaskie Centrum Chorob Serca W Zabrzu	Zabrze	Poland
Region Skane Skanes Universitetssjukhus	Lund	Sweden
Instytut Pomnik Centrum Zdrowia Dziecka	Warsaw	Poland
Vilniaus universiteto ligonine Santaros klinikos VšĮ	Vilnius	Lithuania
MHAT National Heart Hospital EAD	Sofia	Bulgaria
Virgen del Rocío University Hospital	Sevilla	Spain
Centre Hospitalier Universitaire De Toulouse	Toulouse	France
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Assistance Publique Hopitaux De Paris	Paris	France
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Universitair Ziekenhuis Gent	Gent	Belgium
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen	Gothenburg	Sweden
Herzzentrum Leipzig GmbH	Leipzig	Germany
Gottsegen National Cardiovascular Center	Budapest	Hungary
Wojewodzki Szpital Specjalistyczny We Wroclawiu	Wroclaw	Poland
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Children’s Health Ireland	Dublin	Ireland
Hopital Jeanne De Flandre	Lille	France
HUS	Helsinki	Finland
Avudklsspa Ptvtlkyj Huiaorrj Dj Mkjfecgvx	Marseille	France
Knqnovad dgn Uatqcgwbnrrh Mhlvorkq Aax	Munich	Germany
Aathxgv Uadjt Swpwkkdrb Lesxtk De Bzvijch	Bologna	Italy
Uxwglddkjiupmf Chzgzyd Kcsxiamtv	Gdansk	Poland
Ufegyvumkz Mvgihax Cmeazj Hiaasofbhxjmmjwbh	Hamburg	Germany
Fvsxliwlf Pucz Lo Ixftedmmunizn Bcntvcctk Duz Hczzokcz Unrvhzhjwhazu Lf Ptv	Madrid	Spain
Sapupvf Kdmmmcodh Iax Kxgglx Jcztdshff Uojyadrkgwnd Mcrjquldoj Iqv Kdsgpm Mtmenbhnrowesgu W Pusrjzwx	Poznan	Poland
Hlclxkbd Vqqr dudmqjis	Barcelona	Spain
Hujizdwt Uvfvfqqchkioq df A Cjjxnp	A Coruna Galicia	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	27.02.2020
Bulgaria	Not recruiting	27.02.2020
Finland	Not recruiting	27.02.2020
France	Not recruiting	27.02.2020
Germany	Not recruiting	27.02.2020
Hungary	Not recruiting	27.02.2020
Ireland	Not recruiting	27.02.2020
Italy	Not recruiting	27.02.2020
Lithuania	Not recruiting	27.02.2020
Poland	Not recruiting	27.02.2020
Portugal	Not recruiting	27.02.2020
Spain	Not recruiting	27.02.2020
Sweden	Not recruiting	27.02.2020

Trial locations

Investigated drugs:

Selexipag is a medication used in this clinical trial to help treat children with a condition called Pulmonary Arterial Hypertension (PAH). PAH is a type of high blood pressure that affects the arteries in the lungs and the right side of the heart. Selexipag works by relaxing and widening the blood vessels in the lungs, which can help lower the blood pressure in these vessels and improve the ability to exercise. In this trial, Selexipag is being tested to see if it can help slow down the progression of the disease when added to the standard treatments that children with PAH are already receiving.

Investigated diseases:

Pulmonary arterial hypertension

Pulmonary Arterial Hypertension – Pulmonary Arterial Hypertension is a condition characterized by high blood pressure in the arteries that supply the lungs. This disease causes the small arteries in the lungs to become narrow or blocked, leading to increased resistance to blood flow. As the condition progresses, the heart must work harder to pump blood through the lungs, which can lead to heart strain and enlargement. Over time, this increased workload can weaken the heart muscle and affect its ability to function properly. Symptoms often include shortness of breath, fatigue, dizziness, and swelling in the ankles or legs. The progression of the disease can vary, with symptoms gradually worsening over time.

Trial ID:

2022-501012-34-00

Protocol code:

AC-065A310

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Selexipag and Bosentan for Children with Pulmonary Arterial Hypertension

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?