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

Completed

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

In brief

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.

Key facts

Study ID

NCT03768817

Run by

Ultragenyx Pharmaceutical Inc

People needed

20

Starts

2019-01-30

Expected to finish

2020-06-08

Last updated by the study team

2020-08-28

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Unwilling to sign informed consent or assent to release of medical records

Where it is running

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

Full record on ClinicalTrials.gov

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