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

PHASE2CompletedINTERVENTIONAL
Enrollment

36

Participants

Timeline

Start Date

February 28, 2014

Primary Completion Date

September 20, 2017

Study Completion Date

September 20, 2017

Conditions
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Interventions
DRUG

UX007

oral liquid

DRUG

Placebo

oral liquid

Trial Locations (14)

3084

Melbourne Brain Centre, Heidelberg

10032

Columbia University - Department of Neurology, New York

Columbia University Medical Center, New York

32819

Neurology & Epilepsy Research Center, Orlando

33155

Miami Children's Research Institute, Miami

75935

Service de Neurologie Pédiatrique et des Maladies Métaboliques - INSERM U1141 Hôpital Robert Debré - APHP, Paris

76104

Cook Children's Hospital, Fort Worth

77030

University of Texas Neurometabolic Clinic, Houston

80045

Children's Hospital Colorado - University of Colorado, Denver, School of Medicine, Aurora

98105

Seattle Children's Hospital, Seattle

Unknown

Sheba University Medical Center, Tel Aviv

"Unita' Operativa Neurologia Pediatrica e Malattie Muscolari Istituto Giannina Gaslini", Genova

Hospital Sant Joan de Deu, Barcelona

Newcastle University, Newcastle upon Tyne

All Listed Sponsors
lead

Ultragenyx Pharmaceutical Inc

INDUSTRY

NCT01993186 - Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) | Biotech Hunter | Biotech Hunter