Valsartan for Marfan syndrome|Loeys-Dietz syndrome|Vascular Ehlers-Danlos syndrome|Arterial tortuosity syndrome|Shprintzen-Goldberg syndrome|Neonatal Marfan syndrome|Familial thoracic aortic aneurysm and dissection|Bicuspid aortic valve (familial BAV)

Inclusion criteria

• {"criterion_text":"- 1. Age 1- 39 years."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 3. Aortic root dilatation (z-score ≥ 2)."}
• {"criterion_text":"- 4. Signed informed consent to participate in the study."}

Exclusion criteria

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

Primary endpoints

• {"endpoint_text":"- Absolute annual difference in aortic t diameter measured by transthoracic echocardiography and expressed in mm/yearroo","definition_or_measurement_approach":"Measured by transthoracic echocardiography and expressed in mm/year."}

Secondary endpoints

• {"endpoint_text":"- annual difference in the aortic root diameter expressed in z-scores, indexed to sex and body surface area (BSA), measured by transthoracic echocardiography and expressed as z-score/year, - the absolute annual difference in aortic root diameter expressed in millimetres as assessed by angio- CT","definition_or_measurement_approach":"Includes z-score change indexed to sex and BSA measured by transthoracic echocardiography (z-score/year) and absolute annual difference assessed by angio-CT (mm/year). Secondary objectives also include measurements by transesophageal echocardiography for other aortic segments, subgroup analyses by genetic HTAD type, major aortic events, adverse events, and comparison of Holter blood pressure values."}

Poland

Earliest CTIS Part Ii Submission Date

02-06-2024

Latest Decision Or Authorization Date

30-06-2025

Processing Time Days

393

Number Of Sites

4

Number Of Participants

180

Primary sponsor

Full Name

Medical University Of Gdansk

Organisation Type

Educational Institution

Country Of Registered Address

Poland