Identification of Carnitine-Responsive Cardiomyopathy
NCT01904396 · Status: UNKNOWN · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 30
Last updated 2013-07-22
Summary
There are some adults with skeletal muscle weakness (called "myopathy") and heart muscle weakness (called "cardiomyopathy") who have low blood levels of a compound called carnitine as a cause of their problems. Carnitine is very important to energy production in muscles. In fact, there are reports of some people with carnitine deficiency who have developed myopathy and cardiomyopathy that was completely reversed with carnitine treatment. The main objective of our project is to determine the number of patients who have carnitine deficiency as a cause of their myopathy and cardiomyopathy. The investigators will be measuring carnitine levels in 1000 patients with cardiomyopathy and will describe the specific features in all the study patients to see if there are any trends that may help us predict which patients with muscle weakness are at risk of developing low carnitine levels. The investigators will be treating patients with low carnitine levels with carnitine and observing them to see if their cardiomyopathy and their muscle weakness improve. Knowing the exact percentage of myopathy and cardiomyopathy patients with carnitine deficiency may allow for screening of patients in a cheap and targeted way to treat the serious complication of this condition, including heart failure and sudden death.
Conditions
- Carnitine Deficiency
Interventions
- DRUG
-
Carnitine
Patients who are found to be carnitine deficient will be started on carnitine replacement and their heart function will be monitored on carnitine.
Sponsors & Collaborators
-
The Physicians' Services Incorporated Foundation
collaborator OTHER -
University Health Network, Toronto
lead OTHER
Principal Investigators
-
Faghfoury Hannaneh, MD · University Health Network, Toronto, Ontario
-
Ingrid Tein, MD · The Hospital for Sick Children, Toronto, Ontario
Study Design
- Allocation
- NA
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2013-08-31
- Primary Completion
- 2016-08-31
- Completion
- 2016-12-31
Countries
- Canada
Study Locations
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