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Marfan
Study Basics
Marfan is a Trial
of Beta Blocker (BB) Therapy (Atenolol) vs. Angiotensin II
Receptor Blocker (ARB) Therapy (Losartan) in Individuals with
Marfan Syndrome (MFS). The target number of participants is
604. The accrual period is approximately 6 years with recruitment
to begin in the Fall of 2006.
Study Design
A Phase III,  , double-blind, multi-center trial to compare the effect
of BB therapy to that of ARB therapy on rates of aortic growth
and progression of aortic regurgitation in individuals with
MFS. Patients are followed for three years after randomization.
Primary Endpoint
Rate of change
in aortic root body surface area adjusted Z-score.
Secondary Endpoints
- Frequency of
combined endpoint: aortic dissection, aortic root surgery
or death
- Rate of change
of mitral regurgitation
- Rate of progression
of left ventricular dilation and dysfunction
- Rate of change
in central aortic stiffness indices
- Long bone overgrowth
and weight gain
- Comparison
of adverse drug reactions between groups
Inclusion Criteria
- 6 months to
25 years of age
- Diagnosis of
MFS according to Ghent criteria
- Aortic root
Z-score >3.0
Exclusion Criteria
- Prior aortic
surgery
- Aortic root
dimension at the sinuses of Valsalva >5 cm.
- Planned aortic
surgery within 6 months of enrollment
- Aortic dissection
- Shprintzen-Goldberg
syndrome
- Loeys-Dietz
syndrome
- Therapeutic
rather than prophylactic use of ACE-I, BB, or calcium channel
blocker (e.g. for arrhythmia, ventricular dysfunction or
valve regurgitation)
- History of
angioedema while taking ACE-I or BB
- Intolerance
to losartan or other ARB that resulted in termination of
therapy
- Intolerance
to atenolol or other BB that resulted in termination of
therapy
- Renal dysfunction
- Asthma of sufficient
severity to preclude use of a BB
- Diabetes mellitus
- Pregnancy or
planned pregnancy within 36 months of enrollment
- Inability to
complete study procedures including history of poor acoustic
windows (inability to obtain accurate measurement of aortic
root)
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