Cardiomyopathy in DMD: Lisinopril vs. Losartan

This study has been completed.
Sponsor:
Collaborators:
Children's Hospital Boston
University of California, Davis
Unverisity of Kansas Medical Center
University of Minnesota - Clinical and Translational Science Institute
St. Louis Children's Hospital
Information provided by (Responsible Party):
Jerry R. Mendell, Nationwide Children's Hospital
ClinicalTrials.gov Identifier:
NCT01982695
First received: October 29, 2013
Last updated: November 5, 2013
Last verified: November 2013

October 29, 2013
November 5, 2013
March 2009
August 2012   (final data collection date for primary outcome measure)
Cardiac ejection fraction as measured by echocardiogram [ Time Frame: Every 4 months for 2 years ] [ Designated as safety issue: Yes ]
Participants will have a baseline visit and then followed every four months for the duration study, anticipated for a total of 2 years.
Same as current
Complete list of historical versions of study NCT01982695 on ClinicalTrials.gov Archive Site
  • Skeletal muscle strength [ Time Frame: Every 12 months for 2 years ] [ Designated as safety issue: No ]
    Muscle strength will be measured by hand held dynamometer. Participants will have a measurement collected at their baseline visit and then again at their 1 and 2 year visits.
  • Skeletal Muscle function [ Time Frame: Every 12 months for 2 years ] [ Designated as safety issue: No ]
    Skeletal muscle function will be measured by Brooke Upper Extremity Functional Rating Scale, Nine-Hole-Peg test, and 6-minute walk test (6MWT). Participants will have a measurement collected at their baseline visit and then again at their 1 and 2 year visits.
Same as current
  • Pulmonary function testing [ Time Frame: Every 12 months for 2 years ] [ Designated as safety issue: No ]
    Forced vital capacity (FVC) will be measured using a spirometer. Participants will have a measurement collected at their baseline visit and then again at their 1 and 2 year visits.
  • Changes in activities of daily living [ Time Frame: Every 12 months for 2 years ] [ Designated as safety issue: No ]
    Measured using the Egen Klassification (EK) Scale. Participants will have a measurement collected at their baseline visit and then again at their 1 and 2 year visits.
  • Health related quality of life [ Time Frame: Every 12 months for 2 years ] [ Designated as safety issue: No ]
    Measured using the PedsQL. Participants will have a measurement collected at their baseline visit and then again at their 1 and 2 year visits.
Same as current
 
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

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.

Not Provided
Interventional
Not Provided
Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
  • Duchenne Muscular Dystrophy (DMD)
  • Cardiomyopathy
  • Drug: Losartan
  • Drug: Lisinopril
  • Active Comparator: Lisinopril
    Intervention: Drug: Lisinopril
  • Active Comparator: Losartan
    Intervention: Drug: Losartan

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
17
September 2013
August 2012   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
Male
Not Provided
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01982695
IRB12-00149
No
Jerry R. Mendell, Nationwide Children's Hospital
Nationwide Children's Hospital
  • Children's Hospital Boston
  • University of California, Davis
  • Unverisity of Kansas Medical Center
  • University of Minnesota - Clinical and Translational Science Institute
  • St. Louis Children's Hospital
Not Provided
Nationwide Children's Hospital
November 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP