NCT02214160 - Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

December 9, 2014

Primary Completion Date

December 3, 2020

Study Completion Date

December 3, 2020

Conditions

Carnitine Palmitoyltransferase (CPT I or CPT II) DeficiencyVery Long Chain Acyl-CoA Dehydrogenase (VLCAD) DeficiencyLong-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) DeficiencyTrifunctional Protein (TFP) DeficiencyCarnitine-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.

Trial Locations (11)

15224

Children's Hospital of Pittsburgh of UPMC, Pittsburgh

20010

Children's National Health System, Washington D.C.

33606

University of South Florida, Tampa

37232

Vanderbilt Medical Center, Nashville

60611

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

84108

University of Utah, Salt Lake City

94143

University of California San Francisco, San Francisco

98105

Seattle Children's Hospital, Seattle

02115

Boston Children's Hospital, Boston

WC1N 3BP

National Hospital for Neurology and Neurosurgery, London

WC1N 3JH

Great Ormond Street Hospital, London

All Listed Sponsors

lead

Ultragenyx Pharmaceutical Inc

INDUSTRY