ClinicalTrials.gg
AvailableNCT03773770Updated Jul 31, 2026

Expanded Access to Triheptanoin

An expanded access record providing Triheptanoin in Long Chain Fatty Acid Oxidation Disorders, sponsored by Ultragenyx Pharmaceutical Inc. Available. Per ClinicalTrials.gov, last updated 2026-07-31.

Sponsored by Ultragenyx Pharmaceutical Inc · Expanded access

Study type
Expanded access
Access type
Individual patients
Sex
All
01

Study summary

Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.

Read the detailed description

Long Chain Fatty Acid Oxidation Disorders (LC-FAOD) and other Non-FAOD conditions, where applicable: Expanded access may be available outside of the US in countries prior to approval by the local regulatory agencies.

For full details, please visit the links provided below.

02

Conditions studied

  • Long Chain Fatty Acid Oxidation Disorders

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Keywords

  • Expanded Access
  • Compassionate Use
  • Carnitine Palmitoyltransferase Deficiency
  • CPT I
  • CPT II
  • Very Long Chain acyl-CoA Dehydrogenase Deficiency
  • VLCAD
  • Long Chain 3-hydroxy-acyl-CoA Dehydrogenase Deficiency
  • LHCAD
  • Trifunctional Protein Deficiency
  • TFP
  • Carnitine-acylcarnitine Translocase Deficiency
  • CACT
  • LC-FAOD
03

In context

VLCAD deficiency

26 studies on the registry are indexed under VLCAD deficiency; 6 are open to participants now.

Browse VLCAD deficiency studies →

Lead sponsor

Ultragenyx Pharmaceutical Inc is the lead sponsor of 63 studies on the registry; 8 are open to participants now.

Of its 16 completed or terminated interventional studies of FDA-regulated products, 12 (75%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All

Eligibility criteria

- The participant must not be eligible for a UX007 clinical trial

05

Access details

Study type
Expanded access
Access type
Individual patients

Available treatment

  • Liquid for oral (PO) or enteral tube administration

    Also known as: UX007

06

Where to request access

No study locations are listed for this record.

07

References and documents

08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 31, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT03773770
Responsible party
Sponsor
First posted
Dec 12, 2018
Last update
Jul 31, 2026

Study contacts

Early Access
Contact
EarlyAccess@ultragenyx.com
Medical Director
study director · Ultragenyx Pharmaceutical Inc
View the source record on ClinicalTrials.gov ↗

Requesting access

Expanded access is arranged between your doctor and the company. Ask your care team to contact the provider listed on this record.

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Discussion

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