Cardiomyopathy in DMD: Lisinopril vs. Losartan
Compare Efficacy of the Angiotensin Converting Enzyme Inhibitor (ACEi) Lisinopril With Angiotensin II Receptor Antagonist Losartan (ARB) for the Cardiomyopathy of Duchenne Muscular Dystrophy
1 other identifier
interventional
23
1 country
6
Brief Summary
This trial is a double-blind randomized clinical trial of lisinopril versus losartan for the treatment of cardiomyopathy in Duchenne Muscular Dystrophy (DMD). Both drugs are known to be effective for the treatment of dilated cardiomyopathy. ACEi have been reported to delay the onset and progression of left ventricle dysfunction in children with DMD. Multiple studies show therapeutic efficacy of losartan in animals with cardiomyopathy related to muscular dystrophy and in patients with cardiomyopathy from diverse causes. ARBs are often reserved for patients in whom heart failure is not adequately treated or where side effects preclude the use of an ACEi. However, in DMD, losartan might be a better choice as a first line drug because of studies demonstrating a potential benefit for skeletal muscle in the mdx mouse. Considering that both skeletal and cardiac muscles are major contributors of the disability of DMD, a drug that could improve both heart and skeletal muscles simultaneously would need consideration as the drug of choice for the cardiomyopathic DMD patient.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for not_applicable
Started Mar 2009
Longer than P75 for not_applicable
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
March 1, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
September 1, 2013
CompletedFirst Submitted
Initial submission to the registry
October 29, 2013
CompletedFirst Posted
Study publicly available on registry
November 13, 2013
CompletedResults Posted
Study results publicly available
March 21, 2017
CompletedMarch 21, 2017
January 1, 2017
3.4 years
October 29, 2013
August 14, 2015
January 31, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Cardiac Ejection Fraction as Measured by Echocardiogram
Mean cardiac ejection fraction as measured by echocardiogram at 12 month study visit. Cardiac ejection fractions were measured using the biplane Simpson's rule using images obtained from the apical 4 chamber views of the heart.
12 month visit
Study Arms (2)
Lisinopril
ACTIVE COMPARATORLosartan
ACTIVE COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- Duchenne muscular dystrophy patients of all ages
- Null mutation of the dystrophin gene or muscle with \<5% dystrophin
- Doppler echocardiogram with ejection fraction (EF) \<55% within 30 days of enrollment
- Ability to cooperate for testing
- Glucocorticoid treatment acceptable including daily or weekend administration of prednisone or deflazacort
You may not qualify if:
- Patients with EF 55% or greater
- Patients with EF \<40% after washout
- Patients taking \>5 mg lisinopril, or \>25 mg losartan or \>5 mg enalapril
- Skeletal deformities or pulmonary anatomical variants that preclude consistent measures of Doppler echocardiography
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Nationwide Children's Hospitallead
- Boston Children's Hospitalcollaborator
- University of California, Daviscollaborator
- University of Kansas Medical Centercollaborator
- University of Minnesotacollaborator
- St. Louis Children's Hospitalcollaborator
Study Sites (6)
University of California Davis
Davis, California, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Minnesota
Saint Paul, Minnesota, United States
St. Louis Children's Hospital
Saint Louise, Missouri, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Related Publications (3)
Thrush PT, Allen HD, Viollet L, Mendell JR. Re-examination of the electrocardiogram in boys with Duchenne muscular dystrophy and correlation with its dilated cardiomyopathy. Am J Cardiol. 2009 Jan 15;103(2):262-5. doi: 10.1016/j.amjcard.2008.08.064. Epub 2008 Oct 30.
PMID: 19121448BACKGROUNDViollet L, Thrush PT, Flanigan KM, Mendell JR, Allen HD. Effects of angiotensin-converting enzyme inhibitors and/or beta blockers on the cardiomyopathy in Duchenne muscular dystrophy. Am J Cardiol. 2012 Jul 1;110(1):98-102. doi: 10.1016/j.amjcard.2012.02.064. Epub 2012 Mar 29.
PMID: 22463839RESULTAllen HD, Flanigan KM, Thrush PT, Dvorchik I, Yin H, Canter C, Connolly AM, Parrish M, McDonald CM, Braunlin E, Colan SD, Day J, Darras B, Mendell JR. A randomized, double-blind trial of lisinopril and losartan for the treatment of cardiomyopathy in duchenne muscular dystrophy. PLoS Curr. 2013 Dec 12;5:ecurrents.md.2cc69a1dae4be7dfe2bcb420024ea865. doi: 10.1371/currents.md.2cc69a1dae4be7dfe2bcb420024ea865.
PMID: 24459612DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Dr. Jerry R. Mendell
- Organization
- Nationwide Children's Hopsital
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Director, Center for Gene Therapy
Study Record Dates
First Submitted
October 29, 2013
First Posted
November 13, 2013
Study Start
March 1, 2009
Primary Completion
August 1, 2012
Study Completion
September 1, 2013
Last Updated
March 21, 2017
Results First Posted
March 21, 2017
Record last verified: 2017-01