Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
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
1 other identifier
observational
20
1 country
15
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Jan 2019
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 5, 2018
CompletedFirst Posted
Study publicly available on registry
December 7, 2018
CompletedStudy Start
First participant enrolled
January 30, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 8, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
June 8, 2020
CompletedAugust 28, 2020
August 1, 2020
1.4 years
December 5, 2018
August 26, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (4)
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 Outcomes (4)
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
Study Arms (1)
Patients treated with triheptanoin
Patients with LC-FAOD treated with triheptanoin before 01 September 2018 under eIND
Interventions
This is a non-interventional retrospective medical record review study. No study investigational product will be administered in this protocol.
Eligibility Criteria
Medical records of patients in the USA with LC-FAOD who received triheptanoin via eIND request from their metabolic or treating physician before 01 September 2018.
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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (15)
Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
Kaiser Permanente
Los Angeles, California, 90027, United States
Children's Hospital of Orange County
Orange, California, 92868, United States
Stanford University
Palo Alto, California, 94304, United States
Yale University
New Haven, Connecticut, 06511, United States
Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
University of South Florida
Tampa, Florida, 33606, United States
Emory University
Atlanta, Georgia, 30322, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, 55455, United States
Duke University
Durham, North Carolina, 27710, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Study Officials
- STUDY DIRECTOR
Medical Director
Ultragenyx Pharmaceutical Inc
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- RETROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 5, 2018
First Posted
December 7, 2018
Study Start
January 30, 2019
Primary Completion
June 8, 2020
Study Completion
June 8, 2020
Last Updated
August 28, 2020
Record last verified: 2020-08