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| Sponsor: | National Heart, Lung, and Blood Institute (NHLBI) |
|---|---|
| Collaborators: |
FDA Office of Orphan Products Development The National Marfan Foundation |
| Information provided by: | National Heart, Lung, and Blood Institute (NHLBI) |
| ClinicalTrials.gov Identifier: | NCT00429364 |
Purpose
Marfan syndrome is a hereditary connective tissue disorder. Many individuals with this condition die because of the associated heart and blood vessel abnormalities. This study will compare the effectiveness of two medications, losartan and atenolol, at slowing aortic root enlargement in individuals with Marfan syndrome.
| Condition | Intervention | Phase |
|---|---|---|
|
Marfan Syndrome |
Drug: Losartan Potassium Drug: Atenolol |
Phase III |
| Study Type: | Interventional |
| Study Design: | Treatment, Randomized, Single Blind (Outcomes Assessor), Active Control, Parallel Assignment, Safety/Efficacy Study |
| Official Title: | Trial of Beta Blocker Therapy (Atenolol) Versus Angiotensin II Receptor Blocker Therapy (Losartan) in Individuals With Marfan Syndrome (A Trial Conducted by the Pediatric Heart Network) |
| Estimated Enrollment: | 604 |
| Study Start Date: | January 2007 |
| Estimated Study Completion Date: | December 2013 |
| Estimated Primary Completion Date: | January 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
1: Active Comparator
Atenolol
|
Drug: Atenolol
Atenolol .5 - 4 mg/kg
|
|
2: Active Comparator
Losartan
|
Drug: Losartan Potassium
Losartan .3 - 1.4 mg/kg
|
Marfan syndrome is an inheritable disorder that affects the body's connective tissue. An abnormal protein results in connective tissue that is weaker than normal. Because connective tissue is found throughout the body, Marfan syndrome can affect many body systems, including the skeleton, eyes, nervous system, skin, lungs, heart, and blood vessels. Overall, heart and blood vessel abnormalities are the leading cause of death in individuals with Marfan syndrome. A common blood vessel abnormality associated with this disease involves the aorta, which is the large artery that carries blood away from the heart to the rest of the body. The aortic root, the portion of the aorta that is attached to the heart, may enlarge and tear or even rupture. A tear or rupture is considered a life-threatening emergency. Recent studies have shown that the medication losartan may reduce aortic root growth and improve heart function. The purpose of this study is to compare the effectiveness of losartan versus atenolol at slowing aortic root growth in individuals with Marfan syndrome.
This 3-year study will enroll individuals with Marfan syndrome. Participants will be randomly assigned to receive either losartan or atenolol on a daily basis. All participants will initially receive a low dose of their assigned medication. This dose will be gradually increased every 3 to 4 weeks until the maximum tolerated dose is reached. A continuous electrocardiogram (ECG) that monitors heart rate and activity in 24-hour intervals will be used to determine the proper dose increase for each participant. Participants will then receive the maximum tolerated dose for the remainder of the study. Study visits will occur at baseline and Months 6, 12, 24, and 36. Each study visit will include a physical examination, a medical history review, an ECG, an echocardiogram, and questionnaires. Additionally, at the baseline study visit blood will be collected for laboratory testing.
Eligibility| Ages Eligible for Study: | 6 Months to 25 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Contact: Gloria Klein, MS, RD | 617-923-7747 ext 323 | gklein@neriscience.com |
Show 26 Study Locations| Principal Investigator: | Ron Lacro, MD | Boston Children's Hospital |
More Information
| Responsible Party: | NERI ( Lynn Sleeper, PI ) |
| Study ID Numbers: | 461, U01 HL68270 |
| Study First Received: | January 29, 2007 |
| Last Updated: | July 24, 2009 |
| ClinicalTrials.gov Identifier: | NCT00429364 History of Changes |
| Health Authority: | United States: Food and Drug Administration; Belgium: Directorate general for the protection of Public health: Medicines; Canada: Health Canada |
|
Aortic Root Dissection Aortic Root Dilation Pediatric Heart Network |
|
Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Adrenergic Agents Physiological Effects of Drugs Bone Diseases Musculoskeletal Abnormalities Limb Deformities, Congenital Pathologic Processes Musculoskeletal Diseases Syndrome Therapeutic Uses Abnormalities, Multiple Bone Diseases, Developmental Connective Tissue Diseases Adrenergic beta-Antagonists |
Cardiovascular Diseases Anti-Arrhythmia Agents Congenital Abnormalities Losartan Sympatholytics Heart Diseases Disease Cardiovascular Abnormalities Cardiovascular Agents Marfan Syndrome Antihypertensive Agents Pharmacologic Actions Angiotensin II Type 1 Receptor Blockers Genetic Diseases, Inborn Autonomic Agents |