NCT03773770|Unknown
Expanded Access to Triheptanoin
1 other identifier
UX007-EAP
Study Type
expanded_access
Target
N/A
Locations
0 countries
Sites
N/A
Timeline
RegisteredDec 2018
Brief Summary
Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.
Trial Health
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 10, 2018
Completed2 days until next milestone
First Posted
Study publicly available on registry
December 12, 2018
CompletedLast Updated
April 13, 2026
Status Verified
April 1, 2026
First QC Date
December 10, 2018
Last Update Submit
April 6, 2026
Conditions
Keywords
Expanded AccessCompassionate UseCarnitine Palmitoyltransferase DeficiencyCPT ICPT IIVery Long Chain acyl-CoA Dehydrogenase DeficiencyVLCADLong Chain 3-hydroxy-acyl-CoA Dehydrogenase DeficiencyLHCADTrifunctional Protein DeficiencyTFPCarnitine-acylcarnitine Translocase DeficiencyCACTLC-FAOD
Interventions
Eligibility Criteria
Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
\- The participant must not be eligible for a UX007 clinical trial
Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.
Sponsors & Collaborators
Related Links
MeSH Terms
Conditions
VLCAD deficiencyTrifunctional Protein Deficiency With Myopathy And NeuropathyCarnitine-Acylcarnitine Translocase Deficiency
Interventions
triheptanoin
Study Officials
- STUDY DIRECTOR
Medical Director
Ultragenyx Pharmaceutical Inc
Central Study Contacts
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 10, 2018
First Posted
December 12, 2018
Last Updated
April 13, 2026
Record last verified: 2026-04