Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT01886378 · ClinicalTrials.gov registry record · Phase 2
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
A Phase 2 study of Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) and Carnitine Palmitoyltransferase (CPT II) Deficiency, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 2
- Development phase
- 29
- Enrollment target
- 10
- Study locations
NCT01886378: Completed Phase 2 study of Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) and Carnitine Palmitoyltransferase (CPT II) Deficiency, sponsored by Ultragenyx Pharmaceutical.
NCT01886378 is a Phase 2 study of Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) and Carnitine Palmitoyltransferase (CPT II) Deficiency that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 29 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (78% lower). The trial reports 10 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01886378, a Phase 2 study of Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) and Carnitine Palmitoyltransferase (CPT II) Deficiency, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 29 participants
- Enrollment target
- 10
- Study locations
Study 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.
Primary Outcome
To evaluate the impact 24 weeks of treatment with UX007 has on exercise intolerance, the change from Baseline in time adjusted-AUC (AUC/time) for workload during 40-minute cycle ergometry tests at Week 24 were assessed using the generalized estimation equation (GEE) model. A cycle ergometer can measure the work performed by an individual over time during physical exercise, the work was measured every 10 minutes from 0 to 40 minutes at Baseline and Week 24. The GEE model included the change from
Conditions Studied
Interventions
- DRUG UX007
Study Locations (10)
Other
- Birmingham Children's Hospital - Birmingham
- National Hospital for Neurology and Neurosurgery - London
- Great Ormond Street Hospital - London
District of Columbia
- Children's National Medical Center - Washington D.C.
Florida
- University of Southern Florida - Tampa
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Massachusetts
- Boston Children's Hospital - Boston
Pennsylvania
- Children's Hospital of Pittsburgh of UPMC - Pittsburgh
Tennessee
- Vanderbilt Medical Center - Nashville
Utah
- University of Utah - Salt Lake City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 29 participants |
| Start Date | 2014-02-06 |
| Est. Completion | 2016-08-25 |
| Phase | Phase 2 |
What the finished NCT01886378 record still lists
NCT01886378 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 29 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (78% lower).
The record links to 5 conditions, with Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) appearing as the primary indexed condition, and to 1 intervention - of which UX007 is the first listed.
NCT01886378 lists 10 locations in 8 states (Other, District of Columbia, Florida).
Frequently Asked Questions
What is clinical trial NCT01886378 about?
NCT01886378 is a clinical study titled "A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)". 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.
What is the current status of trial NCT01886378?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 29 participants. The study started on 2014-02-06. Estimated completion is 2016-08-25.
What conditions does trial NCT01886378 study?
This clinical trial studies the following 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.
What interventions are being tested in trial NCT01886378?
The interventions under investigation include: UX007 (DRUG).
Who is sponsoring clinical trial NCT01886378?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01886378 being conducted?
This trial has 10 study locations across District of Columbia, Florida, Illinois, Massachusetts, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT01886378's enrollment target sits among peer trials
29 1669th of 2000 higher than 324 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02429557 · 29 participants · Phase 1
Hemodynamic Mechanisms of Abdominal Compression in the Treatment of Orthostatic Hypotension in Autonomic Failure
- NCT02568878 · 29 participants · Phase 3
Creatine for Depressed Male and Female Methamphetamine Users
- NCT03014401 · 29 participants · NA
The Effect of Adipose-Derived Stem Cells for Knee Osteoarthritis
- NCT03128996 · 29 participants · Phase 1
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI