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ClinicalTrials.gov public records Last synced Sep 26, 2026, 7:16 PM EDT

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Showing 1–9 of 9 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)

NCT02018315
Conditions
Glucose Transporter Type 1 Deficiency Syndrome, GLUT1 Deficiency Syndrome
Interventions
Triheptanoin
Drug
Lead sponsor
Juan Pascual
Other
Eligibility
1 Month to 20 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
Dallas, Texas
No Longer Available No phase listed Expanded access

Post Study Continuation of C7 for G1D

NCT02018302
Conditions
Glut1 Deficiency Syndrome, Glucose Transporter Type 1 Deficiency Syndrome
Interventions
Triheptanoin
Drug
Lead sponsor
Juan Pascual
Other
Eligibility
1 Month to 28 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Dallas, Texas
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
Interventions
UX007
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
6 Months and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
7
States / cities
Washington D.C., District of Columbia • Tampa, Florida • Chicago, Illinois + 4 more
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)

NCT03506425
Conditions
ALS
Interventions
Triheptanoin
Drug
Lead sponsor
Richard Bedlack, M.D., Ph.D.
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Interventions
UX007, Placebo
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Year to 100 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
8
States / cities
Aurora, Colorado • Miami, Florida • Orlando, Florida + 4 more
Conditions
Congestive Heart Failure, Non-ischemic Cardiomyopathy
Interventions
Triheptanoin oil
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Dallas, Texas
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Glucose Transporter Type 1 Deficiency Syndrome, Glut1 Deficiency Syndrome
Interventions
Triheptanoin
Drug
Lead sponsor
Juan Pascual
Other
Eligibility
30 Months to 55 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Dallas, Texas
Conditions
GLUT1DS1
Interventions
Triheptanoin
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
30 Months to 35 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Dallas, Texas