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Study on the Safety and Effects of Riociguat for Children Aged 6 to 17 with Pulmonary Arterial Hypertension

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What is this study about?

This clinical trial is focused on studying a condition known as pulmonary arterial hypertension (PAH), which is a type of high blood pressure that affects the arteries in the lungs and the right side of the heart. The study is testing a treatment called Riociguat Bayer, which is available in two forms: film-coated tablets and granules for oral suspension. The purpose of the study is to evaluate the safety, tolerability, and how the body processes the medication in children aged 6 to less than 18 years who have PAH.

Participants in the study will receive the medication Riociguat Bayer and may also receive a placebo. The study will involve an open-label approach, meaning that both the participants and the researchers will know which treatment is being administered. The study will include a period of individual dose titration, which means adjusting the dose to find the most effective and safe amount for each participant. The study will last for a maximum of 24 weeks, during which time participants will be monitored for any side effects and changes in their condition.

Throughout the study, various assessments will be conducted, including checking vital signs, performing x-rays, and analyzing blood samples to understand how the medication is working in the body. Participants will also be asked to complete questionnaires about their quality of life and the taste and texture of the pediatric formulation. The study aims to gather important information that could help improve the treatment of pulmonary arterial hypertension in children.

1 joining the study

Upon joining the study, the patient will receive detailed information about the trial, including its purpose, procedures, and potential risks and benefits. Written informed consent must be provided, and if applicable, child assent is required.

2 initial assessment

The patient will undergo an initial assessment to confirm eligibility. This includes a review of medical history, current medications, and a physical examination. A right heart catheterization (RHC) may be performed if not done previously to confirm the diagnosis of pulmonary arterial hypertension (PAH).

3 medication administration

The patient will begin taking Riociguat Bayer, which is administered orally. The dosage will be individually adjusted based on the patient’s response and tolerance. The medication is available in film-coated tablets or granules for oral suspension.

4 regular follow-up visits

The patient will attend regular follow-up visits to monitor safety and effectiveness. These visits will include recording vital signs, conducting left-hand x-rays, and performing pharmacokinetic and pharmacodynamic analyses.

5 evaluation of treatment effects

The effects of the treatment will be evaluated through various tests, including the 6-minute walking distance test, assessment of WHO functional class, and echocardiographic parameters. Quality of life will be assessed using questionnaires.

6 completion of the study

Upon completion of the study, the patient will undergo a final assessment to evaluate the overall impact of the treatment. This includes a review of any adverse events and changes in health status.

Who Can Join the Study?

  • Children aged 6 to under 18 years.
  • Diagnosed with Pulmonary Arterial Hypertension (PAH), which can be:
    • Idiopathic PAH (IPAH): PAH with no known cause.
    • Hereditable PAH (HPAH): PAH that runs in families.
    • PAH associated with other conditions (APAH):
      • Connective tissue disease.
      • Congenital heart disease with shunt closure more than 6 months ago (no open shunts, confirmed by a test called right heart catheterization (RHC) at least 4 months after surgery).
  • Diagnosis of PAH confirmed by right heart catheterization (RHC) at any time before joining the study. For patients with closed shunts, RHC should be done at least 4 months after surgery.
  • PAH confirmed by RHC with:
    • Mean pulmonary artery pressure (PAPmean) of at least 25 mmHg at rest.
    • Pulmonary capillary wedge pressure (PCWP) or left ventricular end-diastolic pressure (LVEDP) of 15 mmHg or less.
    • Pulmonary vascular resistance (PVR) greater than 240 dyn·sec·cm-5 (or at least 3.0 wood units·m2).
  • Patients must be on standard PAH medications, which can include Endothelin Receptor Antagonists (ERA) and/or Prostacyclin Analogues (PCA), for at least 12 weeks before the start of the study. Two groups of patients will be included:
    • Prevalent: Patients already on PAH medication who need additional treatment.
    • Incident: Patients who have not been treated before, starting on PAH medication, and then adding a drug called riociguat once stable on standard care.
  • Must be in WHO functional class I-III, which describes the severity of PAH symptoms.
  • Adolescent females who can have children must have a negative pregnancy test and agree to receive sexual counseling and use effective birth control methods, such as:
    • Progestogen-only hormonal contraception (implant).
    • Intrauterine device (IUD).
    • Intrauterine hormone-releasing system (IUS).
    • Combination of methods like condoms with hormonal contraception.

    This agreement is required from the signing of the consent form until 4 weeks after the last study drug is taken.

  • Young men must agree to use adequate contraception when sexually active.
  • Written informed consent must be provided, and if applicable, child assent must be given.

Who Cannot Join the Study?

  • Patients with any other serious health conditions that could interfere with the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial recently.
  • Patients who have allergies to the study medication or its ingredients.
  • Patients who are unable to follow the study procedures or instructions.
  • Patients with a history of drug or alcohol abuse.
  • Patients with certain heart conditions that are not stable.
  • Patients who are taking medications that might interfere with the study drug.
  • Patients with severe liver or kidney disease.
  • Patients with a history of certain types of cancer.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Universitaetsklinikum Heidelberg AöR Heidelberg Germany

Other Sites

Site Name City Country Status
Azienda Ospedaliera di Padova Padua Italy
Deutsches Herzzentrum Berlin Berlin Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Germany Germany
Not recruiting
29.10.2015
Italy Italy
Not recruiting
29.10.2015

Trial locations

Investigated drugs:

Riociguat is a medication used in this clinical trial to help treat pulmonary arterial hypertension (PAH) in children aged 6 to less than 18 years. Pulmonary arterial hypertension is a condition where there is high blood pressure in the arteries that go from the heart to the lungs. This medication works by relaxing and widening the blood vessels in the lungs, which can help lower the blood pressure in these vessels and make it easier for the heart to pump blood through them. The trial aims to find out how safe and tolerable this medication is for children, as well as how the body processes it.

Investigated diseases:

Pulmonary arterial hypertension (PAH) – 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 pump blood efficiently. Symptoms often include shortness of breath, fatigue, dizziness, and swelling in the ankles or legs. The progression of PAH can vary, but it generally involves a gradual worsening of symptoms and increased difficulty in performing physical activities.

Trial ID:
2023-503536-40-00
Protocol code:
BAY63-2521/15681
NCT ID:
NCT02562235
Trial Phase:
Therapeutic confirmatory (Phase III)

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