The main goal of this study is to evaluate the effects of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of Major Clinical Events (MCEs).
Who can participate
Age range17 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria for Main Study:
* Males and females, from 0 (including newborns) to \< 18 years of age at time of randomization
* Confirmed diagnosis of LC-FAOD
* Have a caregiver(s) willing and able to assist in all applicable study requirements
* Have a legally authorized representative willing and able to provide written informed consent after the nature of the study has been explained and prior to any research-related procedures, and the study participant to be able to provide age-appropriate written assent
* Have ANY ONE of the following significant clinical manifestations of LC-FAOD:
* At least 2 in the prior year, or 3 in the prior 2 years, of severe major episodes of metabolic decompensation (eg, hypoglycemia, rhabdomyolysis, or exacerbation of cardiomyopathy, requiring ER/urgent care unit visits or hospitalizations)
* Recurrent symptomatic hypoglycemia (clinical symptoms of hypoglycemia) requiring intervention
* Susceptibility to hypoglycemia after short periods of fasting (less than 4 to 12 hours, depending on age)
* Evidence of functional cardiomyopathy requiring ongoing medical management or clinical manifestation of heart failure
* Sibling(s) with the same pathogenic variant who presented with MCEs
* Participant with pathogenic variants that are known or suspected to be associated with absent or severely reduced enzyme activity or with severe disease manifestations.
* From the period following informed consent to 5 days after the last dose of stu…
What they're measuring
1
Annualized Event Rate of Major Clinical Events (MCEs)