Duchenne Muscular DystrophyPhase 4NCT00606775
The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy
A Phase 4 study testing Carvedilol in people with Duchenne Muscular Dystrophy and Cardiomyopathies, run by Suzuka Hospital at 1 site in Japan.
Trial 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). Read more about this study
- Who is it for?
- Ages 8 to 45 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy, Cardiomyopathies
- What is being tested?
- Carvedilol
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteJapan
- Study dates
- Dec 2007to Dec 2012
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 45 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male patients with Duchenne muscular dystrophy are required to meet the following criteria:
- Aged ８ to 45 years
- Positive plasma cardiac troponin I (0.06ng/mL) at least 4 blood measurement in every 3 month.
- Left ventricular ejection fraction >30% by echocardiography assessment
- Written informed consent
Who cannot join
- Patients with the following conditions will be excluded from the study:
- Left ventricular ejection fraction <30%
- No plasma cTnI elevation
- beta-blocker is already administered without measurement of plasma cTnI
- Contraindication against treatment with β blockers
- Any other serious disease that could potentially complicate the management and follow-up protocols
Trial locations and contacts
1 site in 1 country.
Japan1 site, 1 recruiting
- Recruiting
Suzuka Hospial
Suzuka, Mie-ken, 513-8501
Takao Nishizawa, MD. PhD, +81-52-744-2150, nishizta@med.nagoya-u.ac.jp
Fumihiko Yasuma, MD. PhD, +81-593-78-0337, yasuma@suzuka.go.jp
About this study
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.
Read the 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
- Being tested
- Carvedilol
- Keywords
- Adrenergic beta-Antagonists, Duchenne Muscular Dystrophy, Cardiomyopathies, Troponin I
- Medical subject headings
- Muscular Dystrophy, Duchenne, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CarvedilolDrug
Also called Artist, Daich-Sankyo Co.Ltd
2.5-5mg/day
| Study group | Role | What they receive |
|---|---|---|
| Carvedilol | Experimental | Not described Receives: Drug: Carvedilol |
| Control | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
The suppression of minor cardiac damage indicated as elevation of plasma cTnI
Measured: 2 years
Secondary outcomes (1)
Left ventricular function deterioration assessed by echocardiography In-hospital mortality for cardiac dysfunction In-hospital mortality for any cause Overall mortality
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2008 (estimated)
- Study startDec 2007
- Primary completionDec 2008 (estimated)
- Study completionDec 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Suzuka Hospital
- Sponsor type
- Other government
- Collaborators
- Nagoya University
- Investigators
- Takao Nishizawa, MD, PhD, Principal investigator, Department of Cardiology, Nagoya University Graduate School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00606775
- Sponsor's protocol ID
- TN1966220
- Status last verified
- Dec 2007
- First submitted
- 22 Jan 2008
- Last updated
- 5 Feb 2008
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 11157687Yasuma 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.
PubMed 15336589Hunsaker 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.
PubMed 7114081Jefferies 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.
PubMed 16246949Ishikawa 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.
PubMed 10220639
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Carvedilol for the Prevention of Minor Cardiac Damage and Cardiac Function in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT00606775
- Acronym / sponsor ID
- TN1966220
- Conditions
- Duchenne Muscular Dystrophy, Cardiomyopathies
- Keywords
- Adrenergic beta-Antagonists, Duchenne Muscular Dystrophy, Cardiomyopathies, Troponin I
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Suzuka HospitalOther government
- Collaborators
- Nagoya University
- Study start
- Dec 2007
- Primary completion
- Dec 2008 (estimated)
- Study completion
- Dec 2012 (estimated)
- First posted
- 5 Feb 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Carvedilol | Experimental | Drug: Carvedilol |
| Control | No intervention | – |
Who may qualify
- Ages
- 8 to 45 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Male patients with Duchenne muscular dystrophy are required to meet the following criteria:
- Aged ８ to 45 years
- Positive plasma cardiac troponin I (0.06ng/mL) at least 4 blood measurement in every 3 month.
- Left ventricular ejection fraction >30% by echocardiography assessment
- Written informed consent
Key exclusion criteria (6 listed)
- Left ventricular ejection fraction <30%
- No plasma cTnI elevation
- beta-blocker is already administered without measurement of plasma cTnI
- Contraindication against treatment with β blockers
- Any other serious disease that could potentially complicate the management and follow-up protocols
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CarvedilolAlso: Artist, Daich-Sankyo Co.Ltd2.5-5mg/day | Drug | Carvedilol |
Primary outcome
The suppression of minor cardiac damage indicated as elevation of plasma cTnI
Time frame: 2 years
1 secondary outcome
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2008.
- Background: PubMed 11157687Sato 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.
- Background: PubMed 15336589Yasuma 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.
- Background: PubMed 7114081Hunsaker 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.
- Background: PubMed 16246949Jefferies 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.
- Background: PubMed 10220639Ishikawa 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Takao Nishizawa, MD, PhD principal investigator
Department of Cardiology, Nagoya University Graduate School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Carvedilol in people with Duchenne Muscular Dystrophy and Cardiomyopathies, run by Suzuka Hospital at 1 site in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Feb 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Takao Nishizawa, MD,PhD (+81-52-744-2150, nishizta@med.nagoya-u.ac.jp), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The suppression of minor cardiac damage indicated as elevation of plasma cTnI (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00606775, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2008.