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

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

Official 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03768817
• Other study ID #: UX007-CL003
• Status: Completed
• Start date: January 30, 2019
• Est. completion date: June 8, 2020

Study information

• Verified date: August 2020
• Source: Ultragenyx Pharmaceutical Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: June 8, 2020
• Est. primary completion date: June 8, 2020
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

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

Locations

Country	Name	City	State
United States	Emory University	Atlanta	Georgia
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Duke University	Durham	North Carolina
United States	Children's Hospital of Los Angeles	Los Angeles	California
United States	Kaiser Permanente	Los Angeles	California
United States	University of Minnesota Masonic Children's Hospital	Minneapolis	Minnesota
United States	Yale University	New Haven	Connecticut
United States	Children's Hospital of Orange County	Orange	California
United States	Stanford University	Palo Alto	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon
United States	Seattle Children's Hospital	Seattle	Washington
United States	University of South Florida	Tampa	Florida
United States	Children's National Hospital	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Duration of Hospitalization for Trigger Event		Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Primary	Disposition on Discharge of Trigger Event		Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Primary	Duration of Important Interventions for Trigger Event		Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Primary	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	Duration of Hospitalization for Other Major Clinical Events		Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Secondary	Disposition on Discharge of Other Major Clinical Events		Up to 48 Weeks Prior to and 48 Weeks After the Start of Triheptanoin Treatment
Secondary	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
Secondary	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