Expanded Access to Triheptanoin
NCT03773770 · Status: AVAILABLE · Type: EXPANDED_ACCESS
Last updated 2026-04-13
Summary
Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.
Conditions
- Long Chain Fatty Acid Oxidation Disorders
Interventions
- DRUG
-
Triheptanoin
Liquid for oral (PO) or enteral tube administration
Sponsors & Collaborators
- lead INDUSTRY
Principal Investigators
-
Medical Director · Ultragenyx Pharmaceutical Inc
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
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