UX007

DRUG5 trials

Sponsors

Ultragenyx Pharmaceutical Inc

Conditions

Carnitine Palmitoyltransferase (CPT I or CPT II) DeficiencyCarnitine Palmitoyltransferase (CPT II) DeficiencyCarnitine-acylcarnitine Translocase (CACT) DeficiencyGlucose Transporter Type 1 Deficiency SyndromeGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) DeficiencyLong-chain Fatty Acid Oxidation Disorders (LC-FAOD)Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency

Phase 2

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

CompletedNCT01886378

Ultragenyx Pharmaceutical IncCarnitine Palmitoyltransferase (CPT II) Deficiency, Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency, Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) +2

Start: 2014-02-06End: 2016-08-25Updated: 2021-02-11

Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

CompletedNCT01993186

Ultragenyx Pharmaceutical IncGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Start: 2014-02-28End: 2017-09-20Updated: 2020-06-19

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

CompletedNCT02214160

Ultragenyx Pharmaceutical IncCarnitine-acylcarnitine Translocase (CACT) Deficiency, Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency +2

Start: 2014-12-09End: 2020-12-03Updated: 2023-08-01

Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)

TerminatedNCT02599961

Ultragenyx Pharmaceutical IncGlucose Transporter Type 1 Deficiency Syndrome

Start: 2015-09-10End: 2019-10-22Updated: 2020-06-11

Phase 3

Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

TerminatedNCT02960217

Ultragenyx Pharmaceutical IncGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Start: 2017-04-19End: 2019-10-09Updated: 2020-06-16

Related Papers

Development and content validity of the Glut1DS Movement Disorder Diary

Future Rare Diseases2025-02-22

Triheptanoin for the treatment of long‐chain fatty acid oxidation disorders: Final results of an open‐label, long‐term extension study

Journal of Inherited Metabolic Disease2023-06-0717 citations

Dietary management and major clinical events in patients with long-chain fatty acid oxidation disorders enrolled in a phase 2 triheptanoin study.

2020-12-259 citations

PP531 Quality Of Life Of Patients With Long-Chain Fatty Acid Oxidation Disorders Before And During Treatment With Triheptanoin

International Journal of Technology Assessment in Health Care2020-12-013 citations