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Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies

NCT02214160 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 94

Last updated 2023-08-01

Study results available
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Summary

The primary objective of this study is to evaluate the long-term safety and efficacy of UX007 in participants with LC-FAOD. The secondary objectives of this study are to evaluate the effect of UX007 on energy metabolism in LC-FAOD and evaluate the impact of UX007 on clinical events associated with LC-FAOD.

Conditions

  • Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency
  • Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency
  • Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency
  • Trifunctional Protein (TFP) Deficiency
  • Carnitine-acylcarnitine Translocase (CACT) Deficiency

Interventions

DRUG

UX007

Administered orally (PO) with food or by gastrostomy tube, at the target dose range of 25-35% of total calories.

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-12-09
Primary Completion
2020-12-03
Completion
2020-12-03
FDA Drug
Yes

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 NCT02214160 on ClinicalTrials.gov