Status:
COMPLETED
Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
Lead Sponsor:
Ultragenyx Pharmaceutical Inc
Conditions:
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Eligibility:
All Genders
Brief 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.
Detailed 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. Participat...
Eligibility Criteria
Inclusion
- 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
- Unwilling to sign informed consent or assent to release of medical records
Key Trial Info
Start Date :
January 30 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 8 2020
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT03768817
Start Date
January 30 2019
End Date
June 8 2020
Last Update
August 28 2020
Active Locations (15)
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1
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
2
Kaiser Permanente
Los Angeles, California, United States, 90027
3
Children's Hospital of Orange County
Orange, California, United States, 92868
4
Stanford University
Palo Alto, California, United States, 94304