The primary objective of the study is to evaluate the clinical outcomes for the trigger event of patients who receive triheptanoin in the emergency Investigational New Drug (eIND) program.
The study is a retrospective chart review of patients in the USA with LC-FAOD who received triheptanoin via eIND request from their metabolic or treating physician before 01 September 2018. Participating study sites will provide the medical records of subjects who meet eligibility criteria, and relevant data regarding the trigger event (the reason the patient was placed on triheptanoin) and key details about hospitalizations during the study period will be extracted.
Study Type
OBSERVATIONAL
Enrollment
20
This is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol.
Children's Hospital of Los Angeles
Los Angeles, California, United States
Kaiser Permanente
Los Angeles, California, United States
Duration of Hospitalization for Trigger Event
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Disposition on Discharge of Trigger Event
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Duration of Important Interventions for Trigger Event
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Number of Critical Medical Assessments Related to LC-FAOD for Trigger Event
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Duration of Hospitalization for Other Major Clinical Events
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Disposition on Discharge of Other Major Clinical Events
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Number of Important Medical Interventions for Other Major Clinical Events
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Number of Critical Medical Assessments Related to LC-FAOD for Other Major Clinical Events
Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
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