Skip to main content
OpenTrials
Completed

NCT Number: NCT03768817

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

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

Sex eligibility

All sexes

Study type

Observational

Primary location

Children's Hospital of Los Angeles, Los Angeles, California, United States

Loading trial locations.

About this study

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.

Who can participate

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

No intervention

Other

This is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol.

Primary outcomes

  1. Duration of Hospitalization for Trigger Event

    Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment

  2. Disposition on Discharge of Trigger Event

    Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment

  3. Duration of Important Interventions for Trigger Event

    Time frame: Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment

  4. 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

Secondary outcomes

  1. 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

  2. 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

  3. 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

  4. 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

Sponsors and collaborators

Lead sponsor

Ultragenyx Pharmaceutical Inc

Industry

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2020
Study completion
2020
First posted
Dec 7, 2018
Registry last updated
Aug 28, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.