The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy

NCT ID: NCT00606775

Last Updated: 2008-02-05

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE4
• Total Enrollment: 60 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-12-31
• Study Completion Date: 2012-12-31

Brief Summary

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.

Detailed Description

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.

Conditions

Duchenne Muscular Dystrophy; Cardiomyopathies

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

Carvedilol

Group Type: EXPERIMENTAL

Carvedilol

Intervention Type: DRUG

2.5-5mg/day

Control

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Carvedilol

2.5-5mg/day

Intervention Type: DRUG

Other Intervention Names

Artist, Daich-Sankyo Co.Ltd

Eligibility Criteria

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

• Minimum Eligible Age: 8 Years
• Maximum Eligible Age: 45 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Nagoya University

OTHER

Sponsor Role: collaborator

Suzuka Hospital

OTHER_GOV

Sponsor Role: lead

Responsible Party

Department of Cardiology, Nagoya Universtiy Graduate School of Medicine

Principal Investigators

Takao Nishizawa, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Department of Cardiology, Nagoya University Graduate School of Medicine

Locations

Suzuka Hospial

Suzuka, Mie-ken, Japan

Site Status: RECRUITING

Countries

Japan

Central Contacts

Takao Nishizawa, MD,PhD

Role: CONTACT

nishizta@med.nagoya-u.ac.jp

+81-52-744-2150

Fumihiko Yasuma, MD,PhD

Role: CONTACT

yasuma@suzuka.go.jp

+81-59-378-1321

Facility Contacts

Takao Nishizawa, MD. PhD

Role: primary

nishizta@med.nagoya-u.ac.jp

+81-52-744-2150

Fumihiko Yasuma, MD. PhD

Role: backup

yasuma@suzuka.go.jp

+81-593-78-0337

References

Sato Y, Yamada T, Taniguchi R, Nagai K, Makiyama T, Okada H, Kataoka K, Ito H, Matsumori A, Sasayama S, Takatsu Y. Persistently increased serum concentrations of cardiac troponin t in patients with idiopathic dilated cardiomyopathy are predictive of adverse outcomes. Circulation. 2001 Jan 23;103(3):369-74. doi: 10.1161/01.cir.103.3.369.

Reference Type: BACKGROUND

PMID: 11157687 (View on PubMed)

Yasuma F, Konagaya M, Sakai M, Kuru S, Kawamura T. A new lease on life for patients with Duchenne muscular dystrophy in Japan. Am J Med. 2004 Sep 1;117(5):363. doi: 10.1016/j.amjmed.2004.03.028. No abstract available.

Reference Type: BACKGROUND

PMID: 15336589 (View on PubMed)

Hunsaker RH, Fulkerson PK, Barry FJ, Lewis RP, Leier CV, Unverferth DV. Cardiac function in Duchenne's muscular dystrophy. Results of 10-year follow-up study and noninvasive tests. Am J Med. 1982 Aug;73(2):235-8. doi: 10.1016/0002-9343(82)90184-x.

Reference Type: BACKGROUND

PMID: 7114081 (View on PubMed)

Jefferies JL, Eidem BW, Belmont JW, Craigen WJ, Ware SM, Fernbach SD, Neish SR, Smith EO, Towbin JA. Genetic predictors and remodeling of dilated cardiomyopathy in muscular dystrophy. Circulation. 2005 Nov 1;112(18):2799-804. doi: 10.1161/CIRCULATIONAHA.104.528281. Epub 2005 Oct 24.

Reference Type: BACKGROUND

PMID: 16246949 (View on PubMed)

Ishikawa Y, Bach JR, Minami R. Cardioprotection for Duchenne's muscular dystrophy. Am Heart J. 1999 May;137(5):895-902. doi: 10.1016/s0002-8703(99)70414-x.

Reference Type: BACKGROUND

PMID: 10220639 (View on PubMed)

Other Identifiers

TN1966220

Identifier Type: -

Identifier Source: org_study_id