Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy
1 other identifier
interventional
52
1 country
6
Brief Summary
The study is to demonstrate non-inferiority of spironolactone vs. eplerenone in preserving cardiac and pulmonary function in patients with preserved LV ejection fraction. Males with Duchenne muscular dystrophy (DMD) confirmed clinically and by mutation analysis will be enrolled. Subjects will be randomized to either eplerenone or spironolactone. Subjects will use a drug diary to record daily compliance of taking the study medication as well as any concerns they may have during the study period. Subjects will undergo cardiac magnetic resonance imaging (CMR) and pulmonary function tests (PFT) at baseline and then again at 12 months post enrollment. Subjects will also complete a quality of life questionnaire at baseline and 12 months. Degree of elbow contracture will be measured using a goniometer at baseline and 12 months.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Mar 2015
Typical duration for phase_3
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
First Submitted
Initial submission to the registry
January 27, 2015
CompletedFirst Posted
Study publicly available on registry
February 3, 2015
CompletedStudy Start
First participant enrolled
March 20, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2018
CompletedResults Posted
Study results publicly available
September 24, 2019
CompletedOctober 7, 2019
September 1, 2019
3.1 years
January 27, 2015
September 3, 2019
September 23, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
Left Ventricular Strain
a sensitive measure of heart muscle function
12 months
Study Arms (2)
Eplerenone
ACTIVE COMPARATOREplerenone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Inspra. Eplerenone is a potassium-sparing diuretic.
Spironolactone
ACTIVE COMPARATORSpironolactone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Aldactone. Spironolactone is a potassium-sparing diuretic.
Interventions
26 Subjects will take Eplerenone, one 50mg capsule by mouth once daily for 12 months.
26 Subjects will take Spironolactone, one 50mg capsule by mouth once daily for 12 months.
Eligibility Criteria
You may qualify if:
- Boys age ≥7 years with DMD confirmed clinically and by mutation analysis able to undergo cardiac magnetic resonance (CMR) without sedation
- LV EF ≥45% (+/-5%) by clinically-acquired echocardiography, nuclear scan or cardiac MRI done within 2 weeks of enrollment
You may not qualify if:
- Non-MR compatible implants
- Severe claustrophobia
- Gadolinium contrast allergy
- Kidney disease
- Prior use of or allergy to aldosterone antagonist
- Use of other investigational therapy.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Ohio State Universitylead
- University of California, Los Angelescollaborator
- University of Utahcollaborator
- University of Colorado, Denvercollaborator
- University of Kansas Medical Centercollaborator
- Vanderbilt University Medical Centercollaborator
Study Sites (6)
Mattel Children's Hospital and David Geffen School of Medicine at UCLA
Los Angeles, California, 90095-1743, United States
University of Colorado
Aurora, Colorado, 80045, United States
University of Kansas Medical Center
Kansas City, Kansas, 66103, United States
The Ohio State University Medical Center
Columbus, Ohio, 43210, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37212, United States
University of Utah
Salt Lake City, Utah, 84113, United States
Related Publications (1)
Raman SV, Hor KN, Mazur W, Cardona A, He X, Halnon N, Markham L, Soslow JH, Puchalski MD, Auerbach SR, Truong U, Smart S, McCarthy B, Saeed IM, Statland JM, Kissel JT, Cripe LH. Stabilization of Early Duchenne Cardiomyopathy With Aldosterone Inhibition: Results of the Multicenter AIDMD Trial. J Am Heart Assoc. 2019 Oct;8(19):e013501. doi: 10.1161/JAHA.119.013501. Epub 2019 Sep 24.
PMID: 31549577DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Dr. Subha Raman
- Organization
- Ohio State University
Study Officials
- PRINCIPAL INVESTIGATOR
Subha V Raman, MD
Ohio State University
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 3
- 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
- Professor of Medicine
Study Record Dates
First Submitted
January 27, 2015
First Posted
February 3, 2015
Study Start
March 20, 2015
Primary Completion
May 1, 2018
Study Completion
May 1, 2018
Last Updated
October 7, 2019
Results First Posted
September 24, 2019
Record last verified: 2019-09
Data Sharing
- IPD Sharing
- Will not share