Expanded Access to Triheptanoin | NCT03773770

Studies
Expanded Access to Triheptanoin

NCT03773770

Sponsor

Ultragenyx Pharmaceutical Inc

Status

Available

Last Update Posted

Apr 13, 2026Actual

Enrollment

Not provided

Phase

Not provided

Conditions

Long Chain Fatty Acid Oxidation Disorders

Interventions

Triheptanoin

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Apr 2026

Overall Recruitment Status or Expanded Access Status

Available

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Not provided

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

Dec 10, 2018

First Submission Date that Met QC Criteria

Dec 10, 2018

First Posted Date

Dec 12, 2018Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 6, 2026

Last Update Posted Date

Apr 13, 2026Actual

Description Module

Brief Summary

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

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.

Conditions Module

Conditions

Long Chain Fatty Acid Oxidation Disorders

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

Design Module

Study Type

Expanded Access

Number of References to an Expanded Access Study

Expanded Access Types

Individual Patients

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

No data available

No data is available for this block.

Biospecimen

No data available

No data is available for this block.

Enrollment

Not provided

Name	Type	Description	Arm Group Labels	Other Names
Triheptanoin	Drug	Liquid for oral (PO) or enteral tube administration		UX007

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Not provided

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Early Access	Contact	1-415-483-8800		EarlyAccess@ultragenyx.com

Overall Officials

Name	Affiliation	Role
Medical Director	Ultragenyx Pharmaceutical Inc	Study Director

Locations

Not provided

References Module

Citations

Not provided

Label	URL
Access to Investigational Therapies / Requesting Access	View source
Dojolvi Patient and Prescribing Information Page	View source

ID	Term
C536353	VLCAD deficiency
C566945	Trifunctional Protein Deficiency With Myopathy And Neuropathy
C562812	Carnitine-Acylcarnitine Translocase Deficiency