A Randomized, Double-blind, Multicenter Study to Determine the Effect of Triheptanoin Compared with Even-chain, Medium-chain Triglycerides (MCT) on Major Clinical Events (MCEs) in Pediatric Patients with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Conditions

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Brief summary

Annualized event rate of MCEs

Detailed description

Annualized duration of MCEs, Annualized hypoglycemic event-rate captured as MCEs and HCEs, CGI-C scale score, Left ventricular ejection fraction, left ventricular systolic volume, and left ventricular wall mass, Annualized frequency and duration of rhabdomyolysis-MCEs, Annualized frequency and duration of cardiomyopathy-MCEs, Annualized duration of hypoglycemic-MCEs, Change from baseline in scores for: Caregiver-reported PedsQL 4.0 Generic Core Scale (physical health summary, psychosocial health summary, and total scores) (2 years of age and older) OR PedsQL Infant Scale (physical health summary, psychosocial health summary, and total scores) (ages 1 to < 24 months), Survival time, Annualized hospitalization days, Number of missed school or learning opportunity days, Frequency, severity, and relationship to study drug of TEAEs, serious TEAEs, and AESIs, Incidence of TEAEs and serious TEAEs leading to dose modifications, dose reductions, treatment interruptions, discontinuations from study drug, and discontinuations from the study, Plasma concentration levels of heptanoate and betahydroxypentanoate (BHP), Acceptability and Palatability Survey scores of triheptanoin mixed with oral liquids, Change from baseline to 6 months in hepatic PDFF%, assessed by 1HMRS in subjects enrolled in the Liver Substudy

Related papers

No related papers found yet.

Interventions

DRUGFAT EMULSIONS

DRUGTriheptanoin

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Annualized event rate of MCEs	—

Secondary

Measure	Time frame
Annualized duration of MCEs, Annualized hypoglycemic event-rate captured as MCEs and HCEs, CGI-C scale score, Left ventricular ejection fraction, left ventricular systolic volume, and left ventricular wall mass, Annualized frequency and duration of rhabdomyolysis-MCEs, Annualized frequency and duration of cardiomyopathy-MCEs, Annualized duration of hypoglycemic-MCEs, Change from baseline in scores for: Caregiver-reported PedsQL 4.0 Generic Core Scale (physical health summary, psychosocial health summary, and total scores) (2 years of age and older) OR PedsQL Infant Scale (physical health summary, psychosocial health summary, and total scores) (ages 1 to < 24 months), Survival time, Annualized hospitalization days, Number of missed school or learning opportunity days, Frequency, severity, and relationship to study drug of TEAEs, serious TEAEs, and AESIs, Incidence of TEAEs and serious TEAEs leading to dose modifications, dose reductions, treatment interruptions, discontinuations from st	—

Measure

Time frame

Annualized duration of MCEs, Annualized hypoglycemic event-rate captured as MCEs and HCEs, CGI-C scale score, Left ventricular ejection fraction, left ventricular systolic volume, and left ventricular wall mass, Annualized frequency and duration of rhabdomyolysis-MCEs, Annualized frequency and duration of cardiomyopathy-MCEs, Annualized duration of hypoglycemic-MCEs, Change from baseline in scores for: Caregiver-reported PedsQL 4.0 Generic Core Scale (physical health summary, psychosocial health summary, and total scores) (2 years of age and older) OR PedsQL Infant Scale (physical health summary, psychosocial health summary, and total scores) (ages 1 to < 24 months), Survival time, Annualized hospitalization days, Number of missed school or learning opportunity days, Frequency, severity, and relationship to study drug of TEAEs, serious TEAEs, and AESIs, Incidence of TEAEs and serious TEAEs leading to dose modifications, dose reductions, treatment interruptions, discontinuations from st

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Countries

Czechia, Germany, Poland, Spain

Outcome results

None listed