Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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A Retrospective Chart Review Study to Assess the Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program

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

Intervention / Treatment
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* Other: No Intervention


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female patients of any age with confirmed diagnosis of one of the LC-FAOD disorders including: mitochondrial trifunctional protein (TFP) deficiency, carnitine palmitoyltransferase deficiencies (CPT I and CPT II), very long chain acyl-CoA dehydrogenase (VLCAD) deficiency, long-chain 3-hydroxy-acyl-CoA dehydrogenase (LCHAD) deficiency, and carnitine acylcarnitine translocase (CATR) deficiency Treatment initiation with triheptanoin was via eIND before 01 September 2018 Treated with triheptanoin in the USA Willing and able to provide written, signed informed consent, or where appropriate for subjects under the age of 18, or provide written assent and written informed consent by a legally authorized representative after the nature of the study has been explained and prior to any research-related procedures. To obtain and review medical records of deceased individuals, informed consent from next of kin or appropriate legal entity will be obtained, as applicable. Exclusion Criteria: Unwilling to sign informed consent or assent to release of medical records
Sexes Eligible for Study
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All

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patients treated with triheptanoin<br>Patients with LC-FAOD treated with triheptanoin before 01 September 2018 under eIND | Other: No Intervention<br>* This is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Duration of Hospitalization for Trigger Event |  | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment | 
| Disposition on Discharge of Trigger Event |  | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment | 
| Duration of Important Interventions for Trigger Event |  | 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 |  | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Duration of Hospitalization for Other Major Clinical Events |  | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment | 
| Disposition on Discharge of Other Major Clinical Events |  | 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 |  | 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 |  | Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Triheptanoin