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Selexipag

Clinical trials are investigating Selexipag in children and in long-term follow-up studies for pulmonary arterial hypertension. These studies look at dose confirmation, disease progression, and safety in young patients with this serious lung blood vessel condition. They aim to see whether Selexipag can help as part of standard care.

Table of contents

Overview of the Selexipag trials

These clinical trials are studying Selexipag in children and young people with pulmonary arterial hypertension (PAH), which is a type of high blood pressure in the lung arteries.[1][2] The research is focused on whether Selexipag can be used safely and effectively as part of treatment for this condition.[1][2] The available studies include one Phase 2 trial and two Phase 3 trials, all with an authorised status.[1][2][3]

The studies are not general drug information studies; they are designed to answer specific research questions about dose, benefit, and safety in the trial populations.[1][2][3] The target group is mainly children aged 2 to less than 18 years with PAH, and one study also follows participants from earlier pulmonary hypertension parent studies.[1][2][3]

Phase 2 dose confirmation study

The Phase 2 study, NCT 2022-503042-42-00, is designed to confirm the Selexipag starting dose in children with PAH.[1] Researchers chose the starting dose based on pharmacokinetic extrapolation from adults, which means they used adult body-handling data to help estimate a suitable dose for children.[1]

The main goal is to find whether the selected dose gives similar drug exposure in children as adult doses do in adults.[1] This is done by measuring Selexipag and its active metabolite, ACT-333679, during the 12-week up-titration period, which is the time when treatment is gradually increased.[1]

This study is interventional, meaning participants receive the study treatment as part of the research.[1] It plans to enroll 66 participants and is authorised.[1]

Phase 3 efficacy and safety study

The Phase 3 study, NCT 2022-501012-34-00, is a randomized, multicenter, double-blind, placebo-controlled, parallel-group study with an open-label extension period.[2] In simple terms, this means participants are assigned by chance to study groups, the study is run at more than one center, neither participants nor investigators know who gets the active treatment during the blinded part, and some participants may later continue in a follow-up period where the treatment is known.[2]

The study checks whether adding Selexipag to standard of care can delay disease progression compared with placebo in children aged 2 to less than 18 years with PAH.[2] Disease progression means the illness gets worse over time.[2]

The primary outcome is time to disease progression from randomization up to 7 days after treatment stops.[2] This endpoint helps show whether the treatment can keep the condition stable for longer.[2] The study plans to enroll 213 participants and is authorised.[2]

Phase 3 long-term follow-up study

The third study, NCT 2023-506791-27-00, is a prospective, open-label, platform study for long-term follow-up of participants using study interventions in pulmonary hypertension parent studies.[3] A prospective study follows participants forward in time, and open-label means everyone knows which treatment is being used.[3]

This study is focused on long-term safety of the study interventions in treated participants.[3] It measures treatment-emergent adverse events, adverse events that lead to stopping treatment, serious adverse events, and deaths from baseline until end of study.[3]

The study is authorised, is Phase 3, and plans to enroll 155 participants.[3] Its role is to extend safety observation after earlier parent studies in pulmonary hypertension.[3]

Who can participate

The main eligibility group in the pediatric studies is children aged 2 to less than 18 years with pulmonary arterial hypertension.[1][2] This age range is stated in both the dose study and the efficacy study.[1][2]

The long-term follow-up study is for participants from pulmonary hypertension parent studies, so it is not limited to a new diagnosis group in the data provided.[3] The source data do not give more detailed inclusion or exclusion rules, so only the age and condition information can be confirmed here.[1][2][3]

Main endpoints and what they mean

The Phase 2 study uses a pharmacokinetic endpoint, specifically model-based exposure of Selexipag and ACT-333679 corrected for potency during the 12-week up-titration period.[1] This means researchers are checking how much of the drug and active metabolite reach the body and how that compares with the expected strength of each substance.[1]

The Phase 3 efficacy study uses time to disease progression as the main endpoint.[2] That endpoint is important because it shows how long the treatment may help keep the disease from getting worse.[2]

The Phase 3 long-term follow-up study measures safety endpoints, including treatment-emergent adverse events, serious adverse events, and deaths.[3] These measures help researchers understand the longer-term risk profile in treated participants.[3]

Trial summary

Across the three authorised studies, Selexipag is being studied in pediatric pulmonary arterial hypertension, with one trial focused on dose confirmation, one on added benefit over placebo, and one on long-term safety follow-up.[1][2][3] Together, these trials aim to build evidence for how Selexipag may be used in children and young people with this serious condition.[1][2][3]

Trial ID Phase Condition studied Status Enrollment
2022-503042-42-00 Phase 2 Pulmonary Arterial Hypertension Authorised 66
2022-501012-34-00 Phase 3 Pulmonary Arterial Hypertension Authorised 213
2023-506791-27-00 Phase 3 pulmonary arterial hypertension Authorised 155

FAQ

  • What is being studied in Selexipag clinical trials? The trials are studying Selexipag in children with pulmonary arterial hypertension. They look at dose confirmation, delay of disease progression, and long-term safety.
  • Who can take part in these trials? The main study groups are children aged 2 to less than 18 years with pulmonary arterial hypertension. One long-term study follows participants from parent pulmonary hypertension studies.
  • What trial phases are included? The trials include Phase 2 and Phase 3 studies. Phase 2 focuses on confirming doses, and Phase 3 focuses on efficacy and safety.
  • What condition is being studied? All listed trials focus on pulmonary arterial hypertension, a type of high blood pressure in the lung arteries. This condition can make it harder for the heart to pump blood through the lungs.
  • What outcomes are the trials measuring? The trials measure drug exposure in the body, time to disease progression, and safety outcomes such as side effects, serious adverse events, and deaths. These outcomes help show whether the treatment is working and how well it is tolerated.

Ongoing Clinical Trials on Selexipag

  • Long-term Safety Study of Tadalafil and Macitentan for Patients with Pulmonary Arterial Hypertension or Chronic Thromboembolic Pulmonary Hypertension

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Bulgaria Hungary Poland

  • Study on Selexipag and Bosentan for Children with Pulmonary Arterial Hypertension

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Bulgaria Finland France Germany Hungary +7

  • Study to Determine Safe Doses of Selexipag for Children with Pulmonary Arterial Hypertension

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Hungary

Glossary

  • Pulmonary arterial hypertension (PAH): A serious condition where the blood pressure in the arteries of the lungs is too high. This can strain the heart and reduce blood flow through the lungs.
  • Phase 2: An early clinical trial phase that often checks dose, how the body handles the study drug, and early signs of benefit.
  • Phase 3: A later trial phase that compares treatment with placebo or standard care in larger groups to better measure benefit and safety.
  • Interventional study: A study where participants receive a study treatment or control so researchers can compare outcomes.
  • Placebo: An inactive treatment that looks like the study drug. It is used to compare results fairly.
  • Standard of care: The usual treatment that patients normally receive for a condition.
  • Pharmacokinetic (PK) endpoint: A measurement of how the body absorbs, moves, and clears a study drug. It helps researchers understand drug exposure.
  • Exposure (AUCτ,ss): A measure of how much of the drug is in the body over time at steady state, meaning after repeated dosing when levels are more stable.
  • Disease progression: Worsening of the illness over time. In these trials, researchers check how long it takes before the condition gets worse.
  • Serious adverse event (SAE): A harmful medical problem during a study that is severe, may be life-threatening, or may need hospital care.
  • Open-label extension: A follow-up study period where participants know which treatment they are receiving. It is often used to collect longer-term safety data.

References

  1. https://clinicaltrials.gov/study/2022-503042-42-00
  2. https://clinicaltrials.gov/study/2022-501012-34-00
  3. https://clinicaltrials.gov/study/2023-506791-27-00