Identification of Carnitine-Responsive Cardiomyopathy
(C001 Trial)

Recruiting in Palo Alto (17 mi)

Overseen byIngrid Tein, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Waitlist Available

Sponsor: University Health Network, Toronto

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Research Team

Faghfoury Hannaneh, MD

Principal Investigator

University Health Network, Toronto, Ontario

Ingrid Tein, MD

Principal Investigator

The Hospital for Sick Children, Toronto, Ontario

Eligibility Criteria

Inclusion Criteria

An adult patient (>18 years) with a diagnosis of either hypertrophic or dilated cardiomyopathy, for which the underlying etiology of the cardiomyopathy is unknown.

Treatment Details

Interventions

Carnitine (Other)

Participant Groups

1Treatment groups

Experimental Treatment

Group I: CarnitineDeficientExperimental Treatment1 Intervention

Patients identified with primary and secondary carnitine deficiency in the cardiomyopathy population will be prescribed with carnitine supplements to assess cardiac muscle function and status.