Nebivolol Versus Losartan Versus Nebivolol+Losartan Against Aortic Root Dilation in Genotyped Marfan Patients

NCT ID: NCT00683124

Last Updated: 2011-07-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE3

Total Enrollment

291 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-07-31

Study Completion Date

2013-07-31

Brief Summary

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The major clinical problems in patients with Marfan Syndrome (MFS) are aortic root dilation (ARD), dissection and rupture. Although the available treatments (beta-blockers, BBs) improve the evolution of the disease, they do not protect MFS patients from progression of ARD and dissection. A key molecule that negatively influences cell growth, differentiation, survival and death in MFS is TGFb which is antagonised by existing drugs employed in the clinical practice, the Angiotensin II receptor blockers (ARB).

Detailed Description

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Marfan Syndrome is a rare disease (1:5000)(MIM#154700) caused by mutations of the Fibrillin 1 (FBN1) gene. The major clinical problem is aortic aneurysm with risk of dissection when root diameter is 5 cm.

The investigators designed a clinical trial in which a new generation Beta-Blocker Nebivolol with expected effects on shear stress, heart rate and potential anti-stiffness benefits is compared to Losartan, and Angiotensin receptor blocker anti TGF-beta effects, and to the association of both molecules in patients with Marfan Syndrome. Nebivolol is a patented drug that differs chemically, pharmacologically and therapeutically from all other BBs. Nebivolol shows the highest selectivity for ß1 receptors among the currently available BBs, influences the arterial stiffness through an agonistic effect on ß2-receptors and preserves the arterial compliance. We expect a significantly lower progression of the Aortic Root Dilation in the arm of Nebivolol plus Losartan vs. single drug (primary end-point).The investigators further expect: decrease of arterial stiffness higher in the arm treated with both drugs than in solely Nebivolol or Losartan; a decrease of serum levels of active TGFb in both Losartan arms, a drug \& age-dependant variation of the expression of the mutated FBN1 gene. As for other end-points, the potential results are the improvement of valve function, hard events \& delay of surgical timing for the aortic root. The enrolment period will last 12 months, while the overall follow-up period will be of 4 years. An interim analysis for the primary outcome is programmed at month 24.

Conditions

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Marfan Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Investigators Outcome Assessors

Study Groups

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Losartan

Losartan administered as maximal tolerated dosage, not to exceed the maximal theorical dosage of 100 mg/day for adults and 1,6 mg/kg/die for children minor than 16 years.

Group Type EXPERIMENTAL

Losartan

Intervention Type DRUG

Losartan is administered orally as pills. It is given preferentially once a day or, if not well tolerated because of hypotension, the total daily dosage is given in two administrations

Nebivolol

Nebivolol administered as maximal tolerated dosage, not to exceed the maximal theorical dosage of 10 mg/day for adults and 0,16 mg/kg/die for children minor than 16 years.

Group Type EXPERIMENTAL

Nebivolol

Intervention Type DRUG

Nebivolol is administered orally as pills. It is given preferentially once a day in the morning or, if not well tolerated because of hypotension, the total daily dosage is given in two administrations

Losartan+Nebivolol

Losartan administered as maximal tolerated dosage, not to exceed the maximal theorical dosage of 100 mg/day for adults and 1,6 mg/kg/die for children minor than 16 years.

Nebivolol administered as maximal tolerated dosage, not to exceed the maximal theorical dosage of 10 mg/day for adults and 0,16 mg/kg/die for children minor than 16 years.

Group Type EXPERIMENTAL

Losartan and nebivolol

Intervention Type DRUG

Nebivolol is administered orally as pills. It is given preferentially once a day in the morning or, if not well tolerated because of hypotension, the total daily dosage is given in two administrations.

Losartan is administered orally as pills. It is given preferentially once a day or, if not well tolerated because of hypotension, the total daily dosage is given in two administrations

Interventions

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Losartan and nebivolol

Nebivolol is administered orally as pills. It is given preferentially once a day in the morning or, if not well tolerated because of hypotension, the total daily dosage is given in two administrations.

Losartan is administered orally as pills. It is given preferentially once a day or, if not well tolerated because of hypotension, the total daily dosage is given in two administrations

Intervention Type DRUG

Losartan

Losartan is administered orally as pills. It is given preferentially once a day or, if not well tolerated because of hypotension, the total daily dosage is given in two administrations

Intervention Type DRUG

Nebivolol

Nebivolol is administered orally as pills. It is given preferentially once a day in the morning or, if not well tolerated because of hypotension, the total daily dosage is given in two administrations

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of MFS: Ghent criteria and genetically proven defect of the FBN1 gene
* Age: 12 months to 55 years
* BSA-adjusted aortic z score = or \>2 measured at the level of the sinuses of Valsalva at baseline according to Roman's method, or absolute aortic root diameter \>38mm for females and \>40 mm for males

Exclusion Criteria

* Prior aortic surgery and/or dissection
* Aortic root diameter at the level of the sinuses of Valsalva 5 cm
* Planned aortic surgery within 6 months of enrollment for a rate of ARD progression\>5 mm/year even in pts with ARD less than 5 cm
* Clinical or molecular diagnosis of non-MFS connective tissue diseases sharing some features with MFS (Shprintzen-Goldberg syndrome or Loeys-Dietz syndromes)
* Un-renounceable therapeutic (systemic hypertension, arrhythmia, ventricular dysfunction, valve regurgitation) use of drugs such as ACE inhibitors, BBs, or calcium-channel blockers
* Known side-effects while taking an ARB or a BB
* Intolerance to ARB that resulted in termination of therapy
* Intolerance to BB that resulted in termination of therapy
* Renal dysfunction (creatinine level more than upper limit of age-related normal values)
* Diabetes mellitus
* Pregnancy or planned pregnancy within 48 months of enrollment
* Technical limitations for the imaging studies including poor acoustic windows with limits the accurate measurement of aortic root
* Asthma.
Minimum Eligible Age

12 Months

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Merck Sharp & Dohme LLC

INDUSTRY

Sponsor Role collaborator

Menarini Group

INDUSTRY

Sponsor Role collaborator

Fondazione IRCCS Policlinico San Matteo di Pavia

OTHER

Sponsor Role lead

Responsible Party

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IRCCS Foundation San Matteo Hospital

Principal Investigators

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Eloisa Arbustini, MD,FESC,FACC

Role: PRINCIPAL_INVESTIGATOR

IRCCS Foundation San Matteo Hospital, Pavia

Luigi Tavazzi, MD,FESC,FACC

Role: STUDY_CHAIR

IRCCS Foundation San Matteo Hospital, Pavia

Locations

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IRCCS Foundation San Matteo Hospital

Pavia, , Italy

Site Status RECRUITING

Countries

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Italy

Central Contacts

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Eloisa Arbustini, MD,FESC,FACC

Role: CONTACT

+390382501206

Fabiana I Gambarin, MD

Role: CONTACT

+390382501206

Facility Contacts

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Eloisa Arbustini, MD,FESC,FACC

Role: primary

+390382501206

Fabiana I Gambarin, MD

Role: backup

+390382501206

References

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Munger JS, Harpel JG, Gleizes PE, Mazzieri R, Nunes I, Rifkin DB. Latent transforming growth factor-beta: structural features and mechanisms of activation. Kidney Int. 1997 May;51(5):1376-82. doi: 10.1038/ki.1997.188.

Reference Type BACKGROUND
PMID: 9150447 (View on PubMed)

Hao J, Wang B, Jones SC, Jassal DS, Dixon IM. Interaction between angiotensin II and Smad proteins in fibroblasts in failing heart and in vitro. Am J Physiol Heart Circ Physiol. 2000 Dec;279(6):H3020-30. doi: 10.1152/ajpheart.2000.279.6.H3020.

Reference Type BACKGROUND
PMID: 11087260 (View on PubMed)

Border WA, Noble NA. Interactions of transforming growth factor-beta and angiotensin II in renal fibrosis. Hypertension. 1998 Jan;31(1 Pt 2):181-8. doi: 10.1161/01.hyp.31.1.181.

Reference Type BACKGROUND
PMID: 9453300 (View on PubMed)

Lim DS, Lutucuta S, Bachireddy P, Youker K, Evans A, Entman M, Roberts R, Marian AJ. Angiotensin II blockade reverses myocardial fibrosis in a transgenic mouse model of human hypertrophic cardiomyopathy. Circulation. 2001 Feb 13;103(6):789-91. doi: 10.1161/01.cir.103.6.789.

Reference Type BACKGROUND
PMID: 11171784 (View on PubMed)

Pauwels PJ, Gommeren W, Van Lommen G, Janssen PA, Leysen JE. The receptor binding profile of the new antihypertensive agent nebivolol and its stereoisomers compared with various beta-adrenergic blockers. Mol Pharmacol. 1988 Dec;34(6):843-51.

Reference Type BACKGROUND
PMID: 2462161 (View on PubMed)

Ignarro LJ. Experimental evidences of nitric oxide-dependent vasodilatory activity of nebivolol, a third-generation beta-blocker. Blood Press Suppl. 2004 Oct;1:2-16.

Reference Type BACKGROUND
PMID: 15587107 (View on PubMed)

McEniery CM, Schmitt M, Qasem A, Webb DJ, Avolio AP, Wilkinson IB, Cockcroft JR. Nebivolol increases arterial distensibility in vivo. Hypertension. 2004 Sep;44(3):305-10. doi: 10.1161/01.HYP.0000137983.45556.6e. Epub 2004 Jul 19.

Reference Type BACKGROUND
PMID: 15262912 (View on PubMed)

Ware JE Jr, Sherbourne CD. The MOS 36-item short-form health survey (SF-36). I. Conceptual framework and item selection. Med Care. 1992 Jun;30(6):473-83.

Reference Type BACKGROUND
PMID: 1593914 (View on PubMed)

Van Bortel LM, Bulpitt CJ, Fici F. Quality of life and antihypertensive effect with nebivolol and losartan. Am J Hypertens. 2005 Aug;18(8):1060-6. doi: 10.1016/j.amjhyper.2005.03.733.

Reference Type BACKGROUND
PMID: 16296572 (View on PubMed)

Mangrella M, Rossi F, Fici F, Rossi F. Pharmacology of nebivolol. Pharmacol Res. 1998 Dec;38(6):419-31. doi: 10.1006/phrs.1998.0387.

Reference Type BACKGROUND
PMID: 9990650 (View on PubMed)

Roman MJ, Devereux RB, Kramer-Fox R, O'Loughlin J. Two-dimensional echocardiographic aortic root dimensions in normal children and adults. Am J Cardiol. 1989 Sep 1;64(8):507-12. doi: 10.1016/0002-9149(89)90430-x.

Reference Type BACKGROUND
PMID: 2773795 (View on PubMed)

Habashi JP, Judge DP, Holm TM, Cohn RD, Loeys BL, Cooper TK, Myers L, Klein EC, Liu G, Calvi C, Podowski M, Neptune ER, Halushka MK, Bedja D, Gabrielson K, Rifkin DB, Carta L, Ramirez F, Huso DL, Dietz HC. Losartan, an AT1 antagonist, prevents aortic aneurysm in a mouse model of Marfan syndrome. Science. 2006 Apr 7;312(5770):117-21. doi: 10.1126/science.1124287.

Reference Type RESULT
PMID: 16601194 (View on PubMed)

Selamet Tierney ES, Feingold B, Printz BF, Park SC, Graham D, Kleinman CS, Mahnke CB, Timchak DM, Neches WH, Gersony WM. Beta-blocker therapy does not alter the rate of aortic root dilation in pediatric patients with Marfan syndrome. J Pediatr. 2007 Jan;150(1):77-82. doi: 10.1016/j.jpeds.2006.09.003.

Reference Type RESULT
PMID: 17188619 (View on PubMed)

Ahimastos AA, Aggarwal A, D'Orsa KM, Formosa MF, White AJ, Savarirayan R, Dart AM, Kingwell BA. Effect of perindopril on large artery stiffness and aortic root diameter in patients with Marfan syndrome: a randomized controlled trial. JAMA. 2007 Oct 3;298(13):1539-47. doi: 10.1001/jama.298.13.1539.

Reference Type RESULT
PMID: 17911499 (View on PubMed)

Gambarin FI, Favalli V, Serio A, Regazzi M, Pasotti M, Klersy C, Dore R, Mannarino S, Vigano M, Odero A, Amato S, Tavazzi L, Arbustini E. Rationale and design of a trial evaluating the effects of losartan vs. nebivolol vs. the association of both on the progression of aortic root dilation in Marfan syndrome with FBN1 gene mutations. J Cardiovasc Med (Hagerstown). 2009 Apr;10(4):354-62. doi: 10.2459/JCM.0b013e3283232a45.

Reference Type DERIVED
PMID: 19430350 (View on PubMed)

Related Links

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http://www.marfansyndrome.eu

home page of the Polyspecialistic Group for Marfan Syndrome diagnosis and management

Other Identifiers

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EudraCT 2008-001462-81

Identifier Type: -

Identifier Source: secondary_id

FARM7KP2PX

Identifier Type: -

Identifier Source: org_study_id

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