Official Title: “Carvedilol for the Prevention of Minor Cardiac Damage and Cardiac Function in Duchenne Muscular Dystrophy”

Purpose This cardiac dysfunction in patients with Duchenne muscular dystrophy is associated with minor cardiac damage as indicated by elevation of plasma cardiac troponin I (cTnI). The purpose of this study is to investigate whether the administration of Carvedilol can suppress the minor cardiac damage and prevent deterioration of cardiac function.

Study Type: Interventional

Study Design: Prevention, Randomized, Open Label, Active Control, Parallel Assignment, Safety/Efficacy Study

Study Primary Completion Date: December 2008

The life span in patients with Duchenne muscular dystrophy has been extending due to the development of artificial respiratory devices. According to that, the ratio of cardiac dysfunction as a cause of death has been increasing. This cardiac dysfunction was associated with minor cardiac damage as indicated by elevation of plasma cardiac troponin I (cTnI).

Furthermore, and the detection rate of cTnI plasma as revealed to be correlated with the deterioration speed of LV dysfunction assessed by serial echocardiography measurements.

Accordingly, if this minor cardiac damage is suppressed, it is postulated that the progression of cardiac dysfunction can be stopped. In the cases with ventricular arrhythmia and tachycardia, we found plasma cTnI became undetectable after administration of beta-blocker. Accordingly, we investigate whether administration of beta-blocker, carvedilol can persistently suppress the minor cardiac damage and lead to suppress the deterioration of LV function. Note that his study preventive study for preserved to moderate LV dysfunction and is not intended to the beta-blocker treatment for severe LV dysfunction. Because we assume that the mechanism of elevation of cTnI is different; spontaneous in preserved to mild LV dysfunction in patients but LV wall stress in severe LV dysfunction in patients with Duchenne muscular dystrophy.

Intervention(s) in this Clinical Trial

• Drug: Carvedilol
  • 2.5-5mg/day

Arms, Groups and Cohorts in this Clinical Trial

• Experimental: Carvedilol
• No Intervention: Control

Primary Measures

• The suppression of minor cardiac damage indicated as elevation of plasma cTnI
  • Time Frame: 2 years
    Safety Issue?: Yes

Secondary Measures

• Left ventricular function deterioration assessed by echocardiography In-hospital mortality for cardiac dysfunction In-hospital mortality for any cause Overall mortality
  • Time Frame: 5 years
    Safety Issue?: No

Inclusion Criteria:

• Male patients with Duchenne muscular dystrophy are required to meet the following criteria:
• 1. Aged ８ to 45 years
• 2. Positive plasma cardiac troponin I (0.06ng/mL) at least 4 blood measurement in every 3 month.
• 3. Left ventricular ejection fraction >30% by echocardiography assessment
• 4. Written informed consent

Exclusion Criteria:

Patients with the following conditions will be excluded from the study:

• 1. Left ventricular ejection fraction <30%
• 2. No plasma cTnI elevation
• 3. beta-blocker is already administered without measurement of plasma cTnI
• 4. Contraindication against treatment with β blockers
• 5. Any other serious disease that could potentially complicate the management and follow-up protocols

Gender Eligibility for this Clinical Trial: Male

Minimum Age for this Clinical Trial: 8 Years

Maximum Age for this Clinical Trial: 45 Years

Are Healthy Volunteers Accepted for this Clinical Trial?: No

Lead Sponsor: Suzuka Hospital

Overall Clinical Trial Officials and Contacts

Takao Nishizawa, MD, PhD Principal Investigator Department of Cardiology, Nagoya University Graduate School of Medicine  

Overall Contact: Takao Nishizawa, MD,PhD +81-52-744-2150 nishizta@med.nagoya-u.ac.jp

Information obtained from ClinicalTrials.gov on July 02, 2009

Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT00606775

Study ID Number: TN1966220

ClinicalTrials.gov Identifier: NCT00606775

Health Authority: Japan: Institutional Review Board

The institutional review is disclosed in Japanese.The title is "The prospective randomised controlled trial for the efficacy and safety of carvedilol in patients with Duchenne muscular dystrophy".