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A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

NCT01886378 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 29

Last updated 2021-02-11

Study results available
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Summary

The primary objective of the study was to evaluate the impact of UX007 on acute clinical pathophysiology associated with LC-FAOD following 24 weeks of treatment.

Conditions

  • Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
  • Carnitine Palmitoyltransferase (CPT II) Deficiency
  • Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency
  • Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency
  • Trifunctional Protein (TFP) Deficiency

Interventions

DRUG

UX007

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-02-06
Primary Completion
2016-08-25
Completion
2016-08-25

Countries

  • United States
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01886378 on ClinicalTrials.gov