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.
Conditions
Interventions
Trade Name: Valsacor
Product Name: Valsartanum 40 mg / Placebo 1
Pharmaceutical Form: Coated tablet
Pharmaceutical form of the placebo: Coated tablet
Route of administration of the placebo: Oral use
Sponsors
Medical University of Gdansk
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Individuals who meet all of the following criteria will be eligible for the study: 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. Are the trial subjects under 18? yes Number of subjects for this age range: 180 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 140 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: A person who meets any of the following criteria may not be included in the study: 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: absolute annual difference in aortic root diameter measured by transthoracic echocardiography and expressed in mm/year;Secondary Objective: - 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, - absolute annual difference in the diameter of the aortic annulus, sinotubular junction, distal ascending aorta, aortic arch, thoracic aorta, and abdominal aorta assessed by transesophageal echocardiography and expressed in mm/year, - annual difference in the diameter of the aortic annulus, sinotubular junction, distal ascending aorta, aortic arch, thoracic aorta and abdominal aorta expressed in z-score (sex-indexed and BSA), assessed by transesophageal echocardiography and expressed as z-score/year, etc. ;Primary end point(s): absolute annual difference in aortic root diameter measured by transthoracic echocardiography and expressed in mm/year | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - 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, - absolute annual difference in the diameter of the aortic annulus, sinotubular junction, distal ascending aorta, aortic arch, thoracic aorta, and abdominal aorta assessed by transesophageal echocardiography and expressed in mm/year, - annual difference in the diameter of the aortic annulus, sinotubular junction, distal ascending aorta, aortic arch, thoracic aorta and abdominal aorta expressed in z-score (sex-indexed and BSA), assessed by transesophageal echocardiography and expressed as z-score/year, - difference in aortic root diameter in subgroups of patients with different types of HTAD, identified by genetic testing, - acute aortic syndromes: aortic dissection, aortic perforation, intramural hematoma, penetrating ulcer), aortic dilatation requiring surgery, death from cardiovascular causes, - adverse events related to / potentially related to the administered pharmacotherapy (serious adverse events - SAE, adverse events – AE), - comparison of systolic and diastolic blood pressure values based on Holter blood pressure measurements. | — |
Countries
Poland
Contacts
Public ContactCentrum Wsparcia Badan Klinicznych
Piotr Widlak
Outcome results
None listed