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CarnitineDeficient for Carnitine Deficiency (C001 Trial)

Phase 4
Waitlist Available
Led By Faghfoury Hannaneh, MD
Research Sponsored by University Health Network, Toronto
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
* An adult patient (\>18 years) with a diagnosis of either hypertrophic or dilated cardiomyopathy, for which the underlying etiology of the cardiomyopathy is unknown.
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, every 6m for up to 2 years
Awards & highlights
Approved for 10 Other Conditions
All Individual Drugs Already Approved
Pivotal Trial
Drug Has Already Been Approved
No Placebo-Only Group

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.

Eligible Conditions
  • Carnitine Deficiency

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, every 6m for up to 2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, every 6m for up to 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Serum Carnitine Concentration
Secondary study objectives
B-Natriuretic Peptide (BNP)
Echocardiographic Measures

Awards & Highlights

Approved for 10 Other Conditions
This treatment demonstrated efficacy for 10 other conditions.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Drug Has Already Been Approved
The FDA has already approved this drug, and is just seeking more data.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Levocarnitine
FDA approved

Find a Location

Who is running the clinical trial?

University Health Network, TorontoLead Sponsor
1,520 Previous Clinical Trials
503,214 Total Patients Enrolled
The Physicians' Services Incorporated FoundationOTHER
163 Previous Clinical Trials
28,397 Total Patients Enrolled
Faghfoury Hannaneh, MDPrincipal InvestigatorUniversity Health Network, Toronto, Ontario
~2 spots leftby Nov 2025