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Study on Valsartan for Slowing Aortic Root Dilatation in Children and Young Adults with Marfan Syndrome and Related Conditions

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of the medication valsartan in children and young adults who have certain genetic conditions that affect the heart's main artery, known as the aorta. These conditions include Marfan syndrome and related diseases such as Loeys-Dietz syndrome, Vascular type of Ehlers-Danlos syndrome, and others that can cause the aorta to become enlarged or develop aneurysms. The purpose of the study is to evaluate whether valsartan can help slow down the enlargement of the aorta in these patients.

Participants in the study will receive either valsartan or a placebo, which is a substance with no active medication. Valsartan is a medication commonly used to treat high blood pressure and heart failure. It is being tested here to see if it can help manage the size of the aorta in people with these genetic conditions. The study will last for a period of up to 36 months, during which participants will take the medication orally in the form of tablets.

Throughout the study, the size of the aorta will be monitored using medical imaging techniques like transthoracic echocardiography, which is a type of ultrasound that looks at the heart and aorta. The goal is to see if there is a difference in the rate of aortic enlargement between those taking valsartan and those taking the placebo. This research aims to provide valuable information on whether valsartan can be an effective treatment option for managing aortic enlargement in these genetic conditions.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the trial

    Upon joining the trial, you will be asked to provide a signed informed consent. This is a document that confirms your agreement to participate in the study after understanding all the details and potential risks involved.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to confirm your eligibility. This includes verifying your age, which should be between 1 and 39 years, and confirming a diagnosis of a condition related to Marfan syndrome with aortic root dilatation.

  3. Step 3

    Medication administration

    You will be randomly assigned to receive either the medication valsartan or a placebo. Valsartan is administered in the form of a tablet, taken orally. The dosage will be either 40 mg or 80 mg, depending on the specific product assigned to you.

    The medication is to be taken daily for the duration of the trial, which is expected to last until May 31, 2027.

  4. Step 4

    Regular monitoring

    Throughout the trial, regular monitoring will be conducted to assess the effect of the medication on your aortic root diameter. This will involve tests such as transthoracic echocardiography, which uses sound waves to create images of your heart, and possibly angio-CT scans, which provide detailed images of your blood vessels.

    The primary goal is to measure the annual change in the size of your aortic root.

  5. Step 5

    Completion of the trial

    At the end of the trial, a final assessment will be conducted to evaluate the overall impact of the treatment on your condition. This will include a review of all the data collected during the study period.

Who can join the trial?

13 criteria

  • Age between 1 and 39 years.
  • Diagnosis of a syndrome classified under the HTAD group, which includes:
    • Marfan Syndrome
    • Diseases related to Marfan syndrome, such as:
      • Loeys-Dietz syndrome
      • Vascular type of Ehlers-Danlos syndrome
      • Arterial Tortuosity syndrome
      • Shprintzen-Goldberg syndrome
      • Neonatal form of Marfan syndrome
      • Aneurysms-osteoarthritis syndrome
      • Multi-system smooth muscle dysfunction syndrome
      • Familial thoracic aortic aneurysms and aortic dissections
      • Bicuspid aortic valve syndrome - familial BAV
      • Aortic root dilatation with a z-score of 2 or more. (This means the aorta, a large blood vessel, is larger than normal for the person's age and size.)
      • Signed informed consent to participate in the study. (This means the patient or their guardian agrees to join the study after understanding all the details.)

Who cannot join the trial?

5 criteria

  • Patients with any condition other than Marfan Syndrome or related diseases cannot participate.
  • Patients who are not within the age range of children and young adults are excluded.
  • Patients who are not diagnosed with heritable thoracic aortic diseases related to Marfan Syndrome are excluded.
  • Patients who are not able to follow the study procedures or take the study medication as required are excluded.
  • Patients who have any other medical condition that might interfere with the study or pose a risk to the patient are excluded.
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Investigated drugs

Valsartan is a medication that is used to help manage certain heart conditions. In this clinical trial, it is being tested to see if it can slow down the enlargement of the aorta, which is a major blood vessel in the heart. This is particularly important for children and young adults who have a condition called Marfan syndrome, which can cause the aorta to become larger over time. By potentially slowing down this process, valsartan may help reduce the risk of serious heart problems in the future.

What is already known about the treatment

Valsartan – Valsartan is taken orally in the form of tablets and is currently being studied for its effectiveness in slowing the enlargement of the aorta in children and young adults with Marfan-type heritable thoracic aortic diseases. It is a well-known medication in medical literature, primarily used to treat high blood pressure and heart failure. The main therapeutic indication in this trial is to manage conditions like Marfan Syndrome and related rare diseases that affect the aorta. Valsartan works by blocking certain natural substances that tighten blood vessels, allowing blood to flow more smoothly and the heart to pump more efficiently. It belongs to a class of drugs known as angiotensin II receptor blockers (ARBs).

Investigated diseases

  • Marfan Syndrome

    Marfan Syndrome is a genetic disorder that affects the body's connective tissue, which provides support to various structures. It often results in elongated limbs, fingers, and toes, and can affect the heart, eyes, blood vessels, and skeleton. The condition progresses as connective tissue becomes weaker, leading to complications such as aortic enlargement. Over time, the aorta may continue to dilate, increasing the risk of further complications. The syndrome can also cause issues with the eyes, such as lens dislocation, and skeletal abnormalities like scoliosis. The progression varies among individuals, with some experiencing more severe symptoms than others.

  • Loeys-Dietz Syndrome

    Loeys-Dietz Syndrome is a genetic disorder that affects connective tissue, leading to abnormalities in the cardiovascular, skeletal, and skin systems. It is characterized by arterial tortuosity, widely spaced eyes, and a bifid uvula or cleft palate. The disease progresses with the weakening of arterial walls, which can lead to aneurysms and dissections. Skeletal manifestations may include scoliosis and joint laxity. Skin may show easy bruising and abnormal scarring. The progression of symptoms can vary significantly among individuals.

  • Vascular Ehlers-Danlos Syndrome

    Vascular Ehlers-Danlos Syndrome is a genetic disorder affecting connective tissue, primarily impacting the blood vessels, skin, and internal organs. It is characterized by fragile blood vessels, thin skin, and a tendency for bruising. As the disease progresses, the risk of arterial rupture increases due to the fragility of the blood vessels. The skin may become translucent, and there may be a risk of organ rupture. Joint hypermobility is less common compared to other types of Ehlers-Danlos Syndrome. The progression of symptoms can vary widely among individuals.

  • Arterial Tortuosity Syndrome

    Arterial Tortuosity Syndrome is a rare genetic disorder that affects the connective tissue, leading to elongation and twisting of the arteries. This condition can cause complications in the cardiovascular system, including aneurysms and stenosis. As the disease progresses, the arteries may become more tortuous, increasing the risk of vascular complications. It can also affect the skin and joints, leading to hyperextensible skin and joint laxity. The progression of symptoms can vary, with some individuals experiencing more severe vascular issues. The condition is typically present from birth and can affect multiple systems.

  • Shprintzen-Goldberg Syndrome

    Shprintzen-Goldberg Syndrome is a rare genetic disorder that affects connective tissue, leading to craniofacial, skeletal, and cardiovascular abnormalities. It is characterized by features such as craniosynostosis, scoliosis, and arachnodactyly. As the disease progresses, individuals may experience developmental delays and intellectual disabilities. Cardiovascular issues may include aortic root dilation and mitral valve prolapse. The syndrome can also affect the skin, leading to laxity and easy bruising. The progression and severity of symptoms can vary among individuals.

  • Neonatal Marfan Syndrome

    Neonatal Marfan Syndrome is a severe form of Marfan Syndrome that presents at birth or in early infancy. It is characterized by features such as long limbs, arachnodactyly, and joint contractures. The disease progresses rapidly, with significant cardiovascular involvement, including aortic dilation and mitral valve prolapse. Skeletal abnormalities may include scoliosis and chest deformities. The condition can also affect the eyes, leading to lens dislocation. The progression of symptoms is typically more severe compared to classic Marfan Syndrome.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-515059-39-00Protocol codeNBK154/2/2021Estimated enrolment180 patientsSponsorMedical University Of Gdansk

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