Authorised Therapeutic confirmatory (Phase III) Marfan Syndrome Diseases related to Marfan syndrome (rare diseases with aneurysms of the thoracic aorta and dissection of the aorta), among others: 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, etc.

Evaluation of the Efficacy of Valsartan in Slowing Down Aortic Root Dilatation in Children and Young Adults with Marfan

EU CTIS ID: 2024-515059-39-00

What this study is testing

Evaluation of the efficacy of valsartan in slowing down aortic root dilatation in children and young adults with Marfan-type heritable thoracic aortic diseases

  • Therapeutic confirmatory (Phase III)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

  • 1. Age 1- 39 years. 2. Diagnosis of a syndrome classified under the HTAD group: • Marfan Syndrome • Diseases related to Marfan syndrome (rare diseases with aneurysms of the thoracic aorta and dissection of the aorta), among others: 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. 3. Aortic root dilatation (z-score ≥ 2). 4. Signed informed consent to participate in the study.

You likely can't join if

  • 1. Past aortic cardiac surgery. 2. Eligibility for aortic cardiac surgery at the time of examination. 3. Hemodynamically significant, severe or moderate aortic valve defect assessed by echocardiography. 4. Inability to obtain diagnostic echocardiographic images necessary for the evaluation of the aorta in a transthoracic echocardiographic examination - absence of the "Acoustic window". 5. Heart failure; defined as left ventricular ejection fraction <40%. 6. ARB therapy, unless 3 months have elapsed from the last dose taken before qualification. 7. Taking ACE inhibitors, unless 3 months have elapsed from the last dose taken before qualification. 8. Previous adverse reactions to valsartan or other ARB drugs. 9. Contraindications to valsartan (including hypersensitivity to the active substance or to any of the excipients, severe liver dysfunction, biliary cirrhosis, cholestasis, bilateral renal artery stenosis). 10. Breastfeeding, pregnancy or planning pregnancy in the next 12 months and for women of childbearing age, not on an effective method of contraception. 11. Known renal impairment as manifested by estimated creatinine clearance <30 ml/min in children and <10 ml/min in adults

The study team makes the final eligibility decision.

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 0-17 years, 18-64 years. The study team makes the final eligibility decision.

Explore other conditions

BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.