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Showing 1–24 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Intervention: triheptanoin Clear all
Completed Phase 2 Interventional Results available

Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D

NCT03301532
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 16, 2024 · Synced Sep 5, 2026, 2:19 PM EDT
Active, not recruiting No phase listed Observational

Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

NCT04632953
View directory record Official record
Conditions
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2035
U.S. locations
13
States / cities
Phoenix, Arizona • San Francisco, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Sep 5, 2026, 2:19 PM EDT
Terminated Phase 2 Interventional Results available

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

NCT02599961
View directory record Official record
Conditions
Glucose Transporter Type 1 Deficiency Syndrome
Interventions
UX007
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Year and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
5
States / cities
Aurora, Colorado • Miami, Florida • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2020 · Synced Sep 5, 2026, 2:19 PM EDT
Completed Phase 2 Interventional Results available

Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder

NCT01379625
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency, Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency
Interventions
Triheptanoin
Drug
Lead sponsor
Oregon Health and Science University
Other
Eligibility
7 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
2
States / cities
Portland, Oregon • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 22, 2017 · Synced Sep 5, 2026, 2:19 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

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

NCT02018315
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 30, 2019 · Synced Sep 5, 2026, 2:19 PM EDT
Withdrawn Phase 2 Interventional

Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

NCT06067802
View directory record Official record
Conditions
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Interventions
Triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
16 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Sep 5, 2026, 2:19 PM EDT
Recruiting Phase 1 Interventional

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

NCT06340685
View directory record Official record
Conditions
Pyruvate Dehydrogenase Complex Deficiency
Interventions
Triheptanoin
Drug
Lead sponsor
Jirair Krikor Bedoyan
Other
Eligibility
1 Year to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 16, 2026 · Synced Sep 5, 2026, 2:19 PM EDT
Completed Phase 2 Interventional Results available

Diet Treatment Glucose Transporter Type 1 Deficiency (G1D)

NCT03181399
View directory record Official record
Conditions
GLUT1DS1, Epilepsy, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Metabolism Disorders, Glucose Transport Defect, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter Protein Type 1 Deficiency Syndrome
Interventions
Triheptanoin
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
24 Months to 35 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Aug 6, 2025 · Synced Sep 5, 2026, 2:19 PM EDT
No Longer Available No phase listed Expanded access

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

NCT01461304
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 9, 2021 · Synced Sep 5, 2026, 2:19 PM EDT
Completed Phase 2 Interventional Results available

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

NCT01993186
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 18, 2020 · Synced Sep 5, 2026, 2:19 PM EDT
Completed Phase 1 Interventional

Treatment Development of Triheptanoin (G1D)

NCT03041363
View directory record Official record
Conditions
Epilepsy, GLUT1DS1, Glut1 Deficiency Syndrome 1, Autosomal Recessive, Glucose Metabolism Disorders, Glucose Transport Defect, Glucose Transporter Type 1 Deficiency Syndrome, Glucose Transporter Protein Type 1 Deficiency Syndrome
Interventions
Triheptanoin
Drug
Lead sponsor
Juan Pascual
Other
Eligibility
2 Years to 35 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 21, 2022 · Synced Sep 5, 2026, 2:19 PM EDT
Completed Phase 2 Interventional Results available

An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome

NCT02036853
View directory record Official record
Conditions
Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
Interventions
Triheptanoin
Drug
Lead sponsor
Adrian Lacy
Other
Eligibility
1 Year to 50 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Fort Worth, Texas
Source: ClinicalTrials.gov public record
Updated Jan 27, 2021 · Synced Sep 5, 2026, 2:19 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Study of the Acute Effects of Triheptanoin in Heart Failure

NCT01787851
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 13, 2018 · Synced Sep 5, 2026, 2:19 PM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

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

NCT03506425
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 23, 2019 · Synced Sep 5, 2026, 2:19 PM EDT
Completed Phase 2 Interventional Results available

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

NCT02214160
View directory record Official record
Conditions
Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency, Trifunctional Protein (TFP) Deficiency, Carnitine-acylcarnitine Translocase (CACT) Deficiency
Interventions
UX007
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
6 Months and older
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
9
States / cities
San Francisco, California • Washington D.C., District of Columbia • Tampa, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2023 · Synced Sep 5, 2026, 2:19 PM EDT
Not listed Phase 2 Interventional

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

NCT02696044
View directory record Official record
Conditions
Rett Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Center for Rare Neurological Diseases, Norcross, GA
Other
Eligibility
2 Years and older · Female only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Norcross, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 16, 2020 · Synced Sep 5, 2026, 2:19 PM EDT
Completed Phase 2 Interventional Results available

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

NCT01886378
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 10, 2021 · Synced Sep 5, 2026, 2:19 PM EDT
No Longer Available No phase listed Expanded access

Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

NCT02968953
View directory record Official record
Conditions
Glucose Transporter 1 Deficiency Syndrome
Interventions
Triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
3 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 7, 2019 · Synced Sep 5, 2026, 2:19 PM EDT
Completed Early Phase 1 Interventional

Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

NCT03665636
View directory record Official record
Conditions
Glycogen Storage Disease Type I
Interventions
Triheptanoin
Drug
Lead sponsor
Areeg El-Gharbawy
Other
Eligibility
1 Month to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 14, 2021 · Synced Sep 5, 2026, 2:19 PM EDT
Completed No phase listed Observational

Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program

NCT03768817
View directory record Official record
Conditions
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
20 participants
Timeline
2019 – 2020
U.S. locations
15
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2020 · Synced Sep 5, 2026, 2:19 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Triheptanoin (C7 Oil), a Food Supplement, for Glucose Transporter Type I Deficiency (G1D)

NCT02021526
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 14, 2019 · Synced Sep 5, 2026, 2:19 PM EDT
No Longer Available No phase listed Expanded access

Post Study Continuation of C7 for G1D

NCT02018302
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 17, 2019 · Synced Sep 5, 2026, 2:19 PM EDT
Withdrawn Phase 2 Interventional

Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

NCT07097311
View directory record Official record
Conditions
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Interventions
Triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
4 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Sep 5, 2026, 2:19 PM EDT